[House Hearing, 110 Congress]
[From the U.S. Government Publishing Office]
STEM CELL SCIENCE: THE FOUNDATION FOR FUTURE CURES
=======================================================================
HEARING
BEFORE THE
SUBCOMMITTEE ON HEALTH
OF THE
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED TENTH CONGRESS
SECOND SESSION
__________
MAY 8, 2008
__________
Serial No. 110-115
Printed for the use of the Committee on Energy and Commerce
energycommerce.house.gov
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COMMITTEE ON ENERGY AND COMMERCE
JOHN D. DINGELL, Michigan,
Chairman
HENRY A. WAXMAN, California
EDWARD J. MARKEY, Massachusetts
RICK BOUCHER, Virginia
EDOLPHUS TOWNS, New York
FRANK PALLONE, Jr., New Jersey
BART GORDON, Tennessee
BOBBY L. RUSH, Illinois
ANNA G. ESHOO, California
BART STUPAK, Michigan
ELIOT L. ENGEL, New York
ALBERT R. WYNN, Maryland
GENE GREEN, Texas
DIANA DeGETTE, Colorado
Vice Chairman
LOIS CAPPS, California
MIKE DOYLE, Pennsylvania
JANE HARMAN, California
TOM ALLEN, Maine
JAN SCHAKOWSKY, Illinois
HILDA L. SOLIS, California
CHARLES A. GONZALEZ, Texas
JAY INSLEE, Washington
TAMMY BALDWIN, Wisconsin
MIKE ROSS, Arkansas
DARLENE HOOLEY, Oregon
ANTHONY D. WEINER, New York
JIM MATHESON, Utah
G.K. BUTTERFIELD, North Carolina
CHARLIE MELANCON, Louisiana
JOHN BARROW, Georgia
BARON P. HILL, Indiana JOE BARTON, Texas
Ranking Member
RALPH M. HALL, Texas
FRED UPTON, Michigan
CLIFF STEARNS, Florida
NATHAN DEAL, Georgia
ED WHITFIELD, Kentucky
BARBARA CUBIN, Wyoming
JOHN SHIMKUS, Illinois
HEATHER WILSON, New Mexico
JOHN B. SHADEGG, Arizona
CHARLES W. ``CHIP'' PICKERING,
Mississippi
VITO FOSSELLA, New York
STEVE BUYER, Indiana
GEORGE RADANOVICH, California
JOSEPH R. PITTS, Pennsylvania
MARY BONO MACK, California
GREG WALDEN, Oregon
LEE TERRY, Nebraska
MIKE FERGUSON, New Jersey
MIKE ROGERS, Michigan
SUE WILKINS MYRICK, North Carolina
JOHN SULLIVAN, Oklahoma
TIM MURPHY, Pennsylvania
MICHAEL C. BURGESS, Texas
MARSHA BLACKBURN, Tennessee
_________________________________________________________________
Professional Staff
Dennis B. Fitzgibbons, Chief of
Staff
Gregg A. Rothschild, Chief Counsel
Sharon E. Davis, Chief Clerk
David Cavicke, Minority Staff
Director
(ii)
Subcommittee on Health
FRANK PALLONE, Jr., New Jersey, Chairman
HENRY A. WAXMAN, California NATHAN DEAL, Georgia,
EDOLPHUS TOWNS, New York Ranking Member
BART GORDON, Tennessee RALPH M. HALL, Texas
ANNA G. ESHOO, California BARBARA CUBIN, Wyoming
GENE GREEN, Texas HEATHER WILSON, New Mexico
Vice Chairman JOHN B. SHADEGG, Arizona
DIANA DeGETTE, Colorado STEVE BUYER, Indiana
LOIS CAPPS, California JOSEPH R. PITTS, Pennsylvania
TOM ALLEN, Maine MIKE FERGUSON, New Jersey
TAMMY BALDWIN, Wisconsin MIKE ROGERS, Michigan
ELIOT L. ENGEL, New York SUE WILKINS MYRICK, North Carolina
JAN SCHAKOWSKY, Illinois JOHN SULLIVAN, Oklahoma
HILDA L. SOLIS, California TIM MURPHY, Pennsylvania
MIKE ROSS, Arkansas MICHAEL C. BURGESS, Texas
DARLENE HOOLEY, Oregon MARSHA BLACKBURN, Tennessee
ANTHONY D. WEINER, New York JOE BARTON, Texas (ex officio)
JIM MATHESON, Utah
JOHN D. DINGELL, Michigan (ex
officio)
C O N T E N T S
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Page
Hon. Frank Pallone, Jr., a Representative in Congress from the
State of New Jersey, opening statement......................... 1
Hon. Nathan Deal, a Representative in Congress from the State
ofGeorgia, opening statement................................... 3
Hon. Henry A. Waxman, a Representative in Congress from the State
of California, opening statement............................... 4
Hon. Joseph R Pitts, a Representative in Congress from the
Commonwealth of Pennsylvania, opening statement................ 5
Hon. John D. Dingell, a Representative in Congress from the State
of Michigan, opening statement................................. 6
Hon. Tim Murphy, a Representative in Congress from the
Commonwealth of Pennsylvania, opening statement................ 8
Hon. Diana D. DeGette, a Representative in Congress from the
State of Colorado, opening statement........................... 8
Prepared statement........................................... 10
Hon. Marsha Blackburn, a Representative in Congress from the
State of Tennessee, opening statement.......................... 11
Hon. Tammy Baldwin, a Representative in Congress from the State
of Wisconsin, opening statement................................ 12
Hon. Mike Ferguson, a Representative in Congress from the State
of New Jersey, opening statement............................... 13
Hon. Gene Green, a Representative in Congress from the State of
Texas, opening statement....................................... 14
Hon. Anna G. Eshoo, a Representative in Congress from the State
of California, prepared statement.............................. 107
Hon. Barbara Cubin, a Representative in Congress from the State
of Wyoming, prepared statement................................. 107
Hon. Lois Capps, a Representative in Congress from the State of
California, prepared statement................................. 108
Hon. Edolphus Towns, a Representative in Congress from the State
of New York, prepared statement................................ 109
Hon. John Sullivan, a Representative in Congress from the State
of New Oklahoma, prepared statement............................ 110
Witnesses
Elias A. Zerhouni, M.D., Director, National Institutes of Health. 15
Prepared statement........................................... 18
Submitted questions.......................................... 112
John D. Gearhart, Ph.D., C. Michael Armstrong professor of
medicine, Institute for Cell Engineering, Johns Hopkins
University..................................................... 55
Prepared statement........................................... 57
Answers to submitted questions \1\...........................
Amit N. Patel, M.D., M.S., director of cardiac cell therapy, The
Heart, Lung and Esophageal Institute, UPMC Presbyterian,
McGowan Institute of Regenerative Medicine..................... 62
Prepared statement........................................... 65
Answers to submitted questions............................... 121
Douglas T. Rice, Spokane Valley, Washington...................... 69
Prepared statement........................................... 71
Answers to submitted questions............................... 125
George Q. Daley, M.D., Ph.D., president, International Society
for Stem Cell Research; and associate professor of pediatrics,
Children's Hospital Boston..................................... 75
Prepared statement........................................... 76
Answers to submitted questions............................... 127
Weyman Johnson, Jr., J.D., chairman, National Multiple Sclerosis
Society........................................................ 78
Prepared statement........................................... 79
Answers to submitted questions............................... 133
Joseph R. Bertino, M.D., interim director and chief scientific
officer, The Cancer Institute of New Jersey.................... 82
Prepared statement........................................... 84
Answers to submitted questions............................... 185
John K. Fraser, Ph.D., principal scientist, Cytori Therapeutics.. 88
Prepared statement........................................... 90
Answers to submitted questions............................... 136
Submitted Material
``Autologous Nonmyeloablative Hematopoietic Stem Cell
Transplantation in Newly Diagnosed Type 1 Diabetes Mellitus,''
Journal of the American Medical Association, April 11, 2007.... 40
``Stem Cell Vindication,'' Charles Krauthammer, Washington Post,
November 30, 2007.............................................. 49
----------
\1\ Mr. Gearhart did not answer submitted questions for the
record.
.................................................................
STEM CELL SCIENCE: THE FOUNDATION FOR FUTURE CURES
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THURSDAY, MAY 8, 2008
House of Representatives,
Subcommittee on Health,
Committee on Energy and Commerce,
Washington, DC.
The subcommittee met, pursuant to call, at 10:00 a.m., in
room 2322 of the Rayburn House Office Building, Hon. Frank
Pallone, Jr. (chairman) presiding.
Members present: Representatives Pallone, Waxman, Towns,
Green, DeGette, Capps, Baldwin, Dingell (ex officio), Deal,
Hall, Pitts, Ferguson, Myrick, Sullivan, Murphy, Burgess,
Blackburn, and Barton (ex officio).
Staff present: Jessica McNiece, Katherine Martin, Melissa
Sidman, Chad Grant, and Robert Clark.
Mr. Pallone. I call the meeting of the subcommittee to
order.
First of all, let me say good morning to everybody, and
explain that today the subcommittee is meeting to hear about
stem cell science and the potential it holds, and I will
recognize myself for an opening statement initially.
OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE
IN CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Pallone. In terms of the potential for stem cell
science to develop new treatments, therapies, and cures for a
myriad of diseases, conditions, and disabilities, there is
obviously a lot of potential and could impact so many people in
their lives. There are few areas of scientific inquiry that
hold the same level of promise to revolutionize the practice of
medicine. Stem cells offer the possibility of replacing damaged
or diseased cells inside the body with healthy ones. They could
make it possible to strengthen failing heart muscle, regenerate
severed spinal cord nerves, replace damaged brain cells, and
cure many other currently incurable disorders.
Through my service on the subcommittee, I have had the
opportunity to meet and hear from people from communities
across the country and they have come to share their stories or
the stories of their loved ones; just as an example, a young
child with diabetes who requires daily medical attention, an
adult who has left her job to care for a father whose mind has
been ravaged by the effects of Alzheimer's disease, a husband
who watched his wife's motor function deteriorate with the
onset of Parkinson's disease. Their stories vary tremendously
and range from the heartbreaking to the harrowing yet they all
share one common theme, and that is the message of hope, hope
that someday stem cell research will unlock the door and reveal
a new discovery that will cure them of their ailments.
I believe it is our obligation as legislators to enact a
Federal policy that will help advance all types of stem cell
research and provide the opportunity for such discoveries to
take place. Unfortunately, the current Federal policy on stem
cell research is falling short of that goal. The President's
2001 Executive order limits the use of Federal funds for
research on the few lines of stem cells that had already been
harvested. At the time he said that, stem cell research offered
great promise. Almost 7 years later, it is clear to me that the
President's policy has placed arbitrary constraints on stem
cell research and has put patients in great peril.
Since the President issued his Executive order, we have
undoubtedly lost valuable time and resources that could have
been devoted to advancing stem cell research. While there have
been important advancements in certain fields such as stem
cells harvested from cord blood and adult stem cells, the
scientific community appears to be in agreement that it is
embryonic stem cell research that holds the greatest promise
for the development of new cures and treatments. Unfortunately,
the Administration's current policy on embryonic stem cell
research has tied the hands of researchers, impeding scientific
progress and inhibiting America's ability to compete with
scientists around the world. Thankfully, the private sector and
individual States have decided to forge ahead, paving the way
without any Federal funding.
In 2005, my home State of New Jersey became the first State
to provide for the public funding of embryonic stem cell
research. Since then, plans for construction have begun on a
new state-of-the-art facility that will house the Stem Cell
Institute of New Jersey, a joint initiative undertaken by the
University of Medicine and Dentistry of New Jersey and Rutgers,
the State University of New Jersey, and I want to welcome Dr.
Bertino, the interim director of the Stem Cell Institute of New
Jersey, who will be testifying on our second panel today.
But New Jersey is not the only State taking the lead. A
number of other States have either enacted their own measures
that would fund various forms of stem cell research or have
bills pending before their legislatures. While I am thankful
for these efforts, I believe that in order to truly propel the
advancement of stem cell research, we need a Federal policy
that builds upon the advancements being funded in the private
sector and at the State level.
Last year, the House and Senate passed such a policy with
overwhelming bipartisan majorities. The Stem Cell Research
Enhancement Act, sponsored by Ms. DeGette, would have allowed
Federal funding for stem cell research to be conducted on
embryos that would otherwise have been discarded from fertility
clinics and with the consent of the embryos' donors.
Unfortunately, this commonsense policy was met swiftly with the
President's veto pen, the very first of his presidency. I know
this is a controversial issue for many Americans, including
many members who serve on this subcommittee, and I can respect
that. However, I still have trouble understanding the
opposition that exists to such a commonsense approach that
would allow for the progression of stem cell science in what I
view as a careful, ethical, and respectful fashion.
The fact is that Americans want stem cell science to
advance. An overwhelming majority of Americans support
embryonic stem cell research and their representatives in
Congress do so as well, and they want us as legislators to do
everything we can to help unlock the potential of embryonic
stem cells in the quickest fashion possible and bring new life-
saving therapies to the patients who need them.
With millions of Americans dying each year from diseases
that might be cured by stem cell therapies, we can't wait any
longer. The time has come to enact a new Federal policy, and I
know that Ms. DeGette in particular is concerned about that.
She asked that we have this hearing today.
Mr. Pallone. I now recognize our ranking member, Mr. Deal,
for 5 minutes for an opening statement.
OPENING STATEMENT OF HON. NATHAN DEAL, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF GEORGIA
Mr. Deal. Thank you, Mr. Chairman.
With individuals with degenerative life-altering diseases
or life-changing events resulting in paralysis, the
possibilities presented by embryonic stem cell research
represent a glimmer of hope to heal a loved one or reverse the
damage caused by debilitating disease. For others, this issue
seems just as personal as they struggle to reconcile the
possibilities presented by research and science with their own
personal convictions about the sanctity of any human life. It
is at this intersection where we find ourselves this morning.
My hope is that we could explore the possibilities presented by
all types of stem cell research and willingly confront the
ethical and scientific questions raised by this issue.
To my knowledge, adult stem cell research, which does not
raise the ethical questions surrounding the destruction of a
human embryo, has resulted in many new and exciting
discoveries. I would hope that our witnesses could further
elaborate on the potential of research conducted with adult
stem cells and other cells that are capable of producing all or
almost all of the cell types of the developing body. We must
consider whether we should be taking funding away from the
areas of research which have been proven to work and the
promising adult stem cell therapies which have already improved
patient health. Specifically, I hope our witnesses can tell us
about the existing track record of adult stem cell research as
compared to embryonic stem cell research.
I think the question we should be trying to answer here is
whether or not there is a middle ground which allows scientists
to continue their cutting-edge research while respecting the
sanctity of every human life. Hopefully our witnesses today can
describe the variety of research being done with all types of
stem cells today. It would be very useful to learn more about
the future of embryonic stem cell research and the time frame
in which researchers expect to develop these treatments, which
are often cited by supporters of embryonic stem cell research.
I think this should be a good hearing on the issue and
certainly one that warrants our complete attention, and I thank
all of our witnesses for coming and I look forward to your
testimony. I yield back.
Mr. Pallone. I yield 5 minutes to the gentleman from
California--I am sorry--3 minutes to Mr. Waxman.
OPENING STATEMENT OF HON. HENRY A. WAXMAN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF CALIFORNIA
Mr. Waxman. Thank you, Mr. Chairman, for recognizing me and
for holding this hearing today.
Stem cell research is truly exciting scientific research.
Stem cells, both embryonic and adult, hold great potential. For
example, we will hear today about how adult stem cells may be
used to treat potentially deadly heart conditions and embryonic
cells have the potential to become any cell in the body. There
is great hope that these cells will help us understand more
about such devastating diseases as Parkinson's and diabetes and
perhaps some day lead to treatments. And in a fascinating
advance announced last year, several labs have been able to
reprogram adult cells to develop into multiple kinds of cells,
much as embryonic stem cells can.
What I think will become clear as we hear from scientific
experts today is it doesn't make any sense to pit one type of
stem cell research against another. Each line of research holds
distinct promise. They function differently as research models
and may function differently as potential routes to therapies.
It makes sense to encourage the growth of all of these types of
research, not to sit here and argue about which is more
promising than another and why.
Unfortunately, all too frequently, discussions of stem cell
issues are based more on politics than on science. As we have
seen in too many areas, from stacked advisory committees to the
deletion of accurate scientific information from government Web
sites, the science around stem cells has at times been
distorted to justify a particular political or ethical view. We
are given inaccurate accounts of the availability of embryonic
stem cell lines derived from the President's moratorium and in
certain cases, misleading claims about adult stem cells have
been used to argue that there is no scientific need whatsoever
for embryonic stem cell research. Of course, ethical,
political, and other considerations affect policy decisions,
but distorting science is wrong.
I think we are going to hear from a number of experts who
will tell us that there is a consensus among scientists that we
should support embryonic stem cell research. New methods of
creating stem cells are promising. Without funding embryonic
stem cell research, we are guaranteed to learn nothing from it.
We will leave the field behind in the United States and we will
lose the opportunity to develop a meaningful Federal framework
of oversight and ethical guidelines.
I hope today's hearing creates a better understanding in
Congress and America of why support for all kinds of stem cell
research continues to be so important.
Mr. Pallone. Thank you, Mr. Waxman.
Mr. Pitts.
OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA
Mr. Pitts. Thank you, Mr. Chairman. I would like to thank
you for convening this hearing today to discuss the future of
stem cells, and I am grateful for this rare platform to
highlight the incredible developments in stem cell research
that are being used to successfully treat people for several
dozen different conditions. These conditions include heart
disease, juvenile diabetes, Parkinson's, liver failure, lupus,
sickle cell anemia, and spinal cord injuries to name a few.
Over the last decade, there has been contentious debate
over the issue of taxpayer funding for stem cell research that
results in the destruction of a human embryo. At the center of
this debate has been the hope for treatment and cures for
patients across the world who suffer from a host of different
diseases. So I would like to talk about just that, the
patients.
We have here on the left a picture of three patients. The
first one here on the left is Amy Daniels. Amy was diagnosed
with systemic scleroderma, a rare autoimmune disease that
affects connective tissue in the body. Next to Amy is Barry
Gowdy, who suffered from multiple sclerosis. And last is Joe
Rosen, a patient with antiphospholipid syndrome, an autoimmune
disorder that causes blood clots. These three patients endured
vastly different experiences but share two things in common.
First, all three of them had lost hope that they could ever
live a normal life, and second, all three of them found hope in
the form of adult stem cell treatments, which have successfully
mitigated their symptoms.
Another patient is seated here with us today. In 2003,
Carol Franz was diagnosed with multiple myeloma. Myeloma is a
blood cancer that eats away at the bones. X-rays of Carol's
bones made them look like target practice. Faced with the
daunting fears of a deadly form of cancer, Carol found hope as
she was told about a treatment that could help her by using her
own stem cells, and now Carol sits before us having survived
two bouts with cancer after receiving two stem cell
transplants, and she wears a bright green tee shirt that says
``Survivor: adult stem cell transplant.'' And this mantra is
based not on ideology but on science. It is based on what
works. It is based on what saved Carol's life twice. Adult stem
cells are doing what we have all hoped for and wished for: they
are successfully treating patients.
I look forward to hearing the testimony of yet another
patient and witness on this panel, Doug Rice, who has been
treated for heart disease using adult stem cells.
Unfortunately, the political agenda for taxpayer-funded
research that destroys human embryos and has failed to treat
any patients has diverted the focus away from the success of
adult stem cells. In fact, it was just 1 year ago that Dr.
Richard Burt, along with Brazilian researcher Dr. Julio
Voltarelli, conducted a study that used stem cells from
patients' own bodies to successfully reverse type 1 juvenile
diabetes in 13 out of 15 patients over a several-year period.
It was regrettable that this remarkable research had to be
conducted in Brazil due to a lack of interest in the United
States.
Thankfully, last fall, the contentious and heated debate
surrounding stem cell research was quieted by a scientific
breakthrough which has shown the ability to create embryonic-
like stem cells. This research will face all of the same
hurdles as embryonic stem cells, including tumors and
rejection. However, it holds all the potential touted by
proponents of embryonic stem cell research but without any of
the ethical concerns. Dr. Rudolph Jaenisch of the Whitehead
Institute confirmed that, ``Biologically, there is no
difference'' between iPS and embryonic stem cells. Dr. James
Thomson, University of Wisconsin----
Mr. Pallone. Mr. Pitts, if you could just wrap it up. You
are a minute and 26 seconds over.
Mr. Pitts. I am sorry. Dr. Thomson, the pioneer of
embryonic destructive stem cell research, was one of the
scientists to discover this new method and he described
significant advantages of iPS cells because they don't pose the
same ethical challenges as destroying embryos, cloning or
harvesting eggs. So the topic of this hearing is the future of
stem cells.
Thank you, Mr. Chairman, for holding this important
hearing.
Mr. Pallone. Let me just mention to members that we are
going to have, I believe, five votes in another 15 minutes but
we will continue and try to get a couple more opening
statements in before then and then we will come back.
I now recognize the chairman of the full committee, Mr.
Dingell.
OPENING STATEMENT OF HON. JOHN D. DINGELL, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF MICHIGAN
Mr. Dingell. Mr. Chairman, I thank you for your courtesy. I
thank you for the recognition and I commend you for this
hearing this morning.
Stem cell research holds great promise for a better
understanding and treatment of a broad range of debilitating
and deadly diseases and conditions including Parkinson's
disease, cancer, Alzheimer's disease, diabetes, and multiple
sclerosis, amongst others, yet a significant problem is created
by politics and the promise is being somewhat imperiled or
indeed seriously threatened by politics.
This committee is engaged in a practice that is very
important: oversight, the gathering of information to
understand what our national policies should be and what our
actions should be here in the Congress in the way of
legislation, what we should do in the way of expenditure of
monies and national efforts to achieve great national purposes.
Scientists, it should be observed, work with two kinds of
stem cells: adult stem cells and embryonic stem cells. Current
science indicates that adult and embryonic stem cells differ in
significant ways and therefore we need to examine both. Yet
despite well-documented benefits of embryonic stem cell
research and pleas from the scientific community, the
Administration has regrettably adopted research restrictions
that inhibit the ability of scientists to fully explore the
potential of embryonic stem cells. In this Congress, the House
and Senate have sent the President not once but twice
bipartisan legislation that would limit and lift these
restrictions, and both times the President has vetoed this
legislation.
Researchers in my own State of Michigan have been doubly
hamstrung by Federal constraints and by State limitations. The
University of Michigan has an impressive Life Sciences
Institute, focusing on stem cell research and a prominent
University Center for Stem Cell Biology. In 2003, under the
capable leadership of Dr. Max Wicha, who directs the
Comprehensive Cancer Center at the University of Michigan,
scientists there discovered breast cancer stem cells, and last
year found stem cells in pancreatic cancer. These are
especially noteworthy and impressive accomplishments and give
us knowledge and warnings that are important to us in our
concerns about these matters. Given the limited funding
available to the university with State and Federal dollars
unavailable for research, the university scrambles to support
this groundbreaking research with private funds.
I do not profess to know which stem cell lines are most
valuable or which ones offer the most promise or which can give
the greatest hope to those living with debilitating conditions
and diseases. I defer to the experts on such questions such as
Dr. Zerhouni, the director of NIH, who is here today, and
Doctor, by the way, welcome to you. Your comments in 2007, I
will quote: ``It is in the best interests of our scientists,
our science, and our country that we find ways and that the
Nation find a way to go full speed across adult and embryonic
stem cells equally.'' From my standpoint, it is clear today
that the American science will be better served and the Nation
better served if we let our scientists have access to more cell
lines.
I defer to the Institute of Medicine, IOM, which stated in
2002, and I quote, ``Studies of both embryonic and adult human
stem cells will be required to most efficiently advance the
scientific and therapeutic potential of regenerative
medicine.''
Research on both adult and embryonic human stem cells
should be pursued. None of us can guarantee to those suffering
from Parkinson's disease, spinal cord injuries or multiple
sclerosis or any other condition that embryonic stem cell
research will bring success but we can assure and we can
guarantee that if we don't and if we let politics, not science,
guide our efforts, we are consigning ourselves to failure and
to suffering.
I thank the chairman, Mr. Pallone, for holding today's
hearing, and I commend our colleague, Ms. DeGette, for her
dedication and commitment on this issue. Finally again, I thank
our friend, the NIH director, Dr. Zerhouni, for rearranging his
schedule to be here with us today. I look forward to the
testimony of our expert witnesses on the current state of stem
cell research and science, and I thank you, Mr. Chairman; I
thank my colleagues and I thank our witnesses.
Mr. Pallone. Thank you, Chairman Dingell.
I would like to take one more opening statement but let me
just mention, we have five votes, 15 and then four fives, 10
minutes of debate on a motion to recommit, a 15-minute vote on
that and then another five, so we are probably talking close to
an hour once we go into recess. But I would like to have Mr.
Murphy recognized for an opening statement and then after that
we will go vote.
OPENING STATEMENT OF HON. TIM MURPHY, A REPRESENTATIVE IN
CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA
Mr. Murphy. Thank you, Mr. Chairman, and let me begin by
welcoming one of our witnesses here today, Dr. Patel, who is
Director of Cardiovascular Cell Therapies at the University of
Pittsburgh Medical Center. I look forward to hearing his
testimony.
While we are talking about science and research, I think it
is important to understand that ethics cannot be diminished by
relabeling it as political and dismissing the value of ethical
review through polling or politics. The life of a human embryo
is not insignificant and not immaterial to scientific research,
and one cannot perform scientific medical research without
including medical ethics. A couple years ago, at the time that
Congress was voting on embryonic stem cell research, a study
came out out of South Korea, Seoul International University, I
believe, and many were so eager to find the results they wanted
to see that they failed to see that the results were not what
was really found.
We need to continue stem cell research but to also review
its scientific merit and outcome and to always, always review
each finding under the lamp of careful scientific and ethical
scrutiny.
The Federal Government does not prohibit any private
individual or business from carrying out embryonic stem cell
research but we have chosen to hold off taxpayers' dollars for
this, and it is not just a matter of deciding on a poll. We
have to acknowledge that years from now, perhaps this very
subcommittee will be debating and holding hearings on what we
may now consider as the unthinkable: cloning replicas of
ourselves to be used as organ gardens waiting to be harvested.
Indeed, that may come in the future. But let us understand when
it comes to stem cell research, dozens and dozens of great
scientific breakthroughs have come from using adult stem cells,
placenta, umbilical cord, muscle, skin, other issues, and that
is important, but the number of studies that have come out that
have shown significant scientific results from embryonic stem
cells is zero.
So I hope that this panel will look at these issues as ones
that are important to review and that we cannot, no matter how
hard we might use tactics to dismiss it as political, we cannot
dismiss ourselves from the obligation of carefully, carefully
reviewing each thing we do. Life does have value, saving lives
has value, and scientific research cannot be made distinct from
ethical oversight of that same research.
I yield back.
Mr. Pallone. I think we have time for one more, so I
recognize Ms. DeGette for an opening statement.
Ms. DeGette. Thank you, Mr. Chairman. I would ask unanimous
consent to put my full opening statement in the record.
Mr. Pallone. Without objection, so ordered.
OPENING STATEMENT OF HON. DIANA D. DEGETTE, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF COLORADO
Ms. DeGette. Thank you, and I want to thank you for holding
this very first ever hearing on stem cell research in the
Energy and Commerce Committee. It is an incredibly important
topic, and I want to thank Dr. Zerhouni for coming and
rearranging his schedule today.
It is particularly important that we have this hearing
because over the past year we have had many developments in the
field of cell-based scientific research. We saw breakthroughs
and accomplishments that could not have been predicted even
months before they happened: insulin-producing islet cells
created from embryonic stem cells, induced pluripotent stem
cells developed from adult skin cells, and primate embryonic
stem cells generated through somatic cell nuclear transfer. All
of this proves that one can rarely predict the outcomes of
scientific research and it underscores what the other members
have been saying, that it is crucial to make the investment in
all ethical forms of research to begin with.
That is what we are going to explore during this hearing:
where we are now and where we are going with stem cell
research. Every time there has been some new discovery in some
other type of research besides embryonic stem cells, the Bush
Administration and opponents of this research try to claim it
is a substitute for embryonic stem cell research, yet as every
researcher tells me, all of these forms of cell-based research
are complementary and they all aid future developments of cures
for patients, which we see so eloquently here in the front row.
It simply does not make sense to remove one avenue of research
from the equation, especially one that is relatively well
developed. We should continue pursuing all forms of ethical
research.
It makes me particularly angry when people try to claim
that adult stem cells can substitute cures for diseases for
which adult stem cells have shown no clinical promise
whatsoever. I know that these wonderful patients who are here
today who have been cured by adult stem cells, mostly for
blood-related diseases, would never say that somebody with
diabetes or somebody with Parkinson's or somebody with nerve
damage or somebody with macular degeneration, all diseases for
which embryonic stem cell research has shown promise and adult
stem cells have shown no clinical promise, no one would say
those people should not be cured, and that is the whole issue
here today. I pray every day that my 14-year-old daughter will
be cured of diabetes and I frankly don't care if she is cured
by embryonic stem cell research or adult stem cell research or
ethical somatic cell nuclear transfer. I don't really care and
I don't think the rest of the parents in this country care
either.
But what we do need to do as a government is we need to
take our responsibility seriously and we need to say we are
going to expand this research in an ethical way, we are going
to make a national commitment to doing it, and we are not going
to play politics with it. That is why I want to introduce and
congratulate my friend, Mike Castle, who has snuck into the
back of the room, who has been my compadre and fellow fighter
on this issue. Mike and I are developing new legislation which
I hope this hearing will help us begin to get evidence for, and
what we believe our new legislation should do is obviously lift
the ban on Federal funding for research on embryonic stem cell
lines developed after August 2001, construct a framework for
ethical oversight of all cell-based research developed by the
National Institutes of Health and with the NIH as a key player,
and make the national commitment to this research that we
should have had for the last 10 years. We expect to be
introducing this legislation soon and are looking forward to
input from the experts in the field.
And just one last note, Mr. Chairman. Absent in this whole
discussion today and absent in the Bush Administration's
national discussion is the fact that there is no Federal
ethical oversight over the research that is going on either
among the States with the limited Federal dollars that are
available right now or perhaps most disturbing to me, with
private entities that are doing the research. We need to both
make the commitment to all ethical cell-based research but we
also need to make the commitment to ethical oversight because
some of this research is on the edge of bioethics and we need
to make sure that we get it right for the patients of tomorrow.
With that, Mr. Chairman, I appreciate your comity and I
yield back.
[The prepared statement of Ms. DeGette follows:]
Statement of Diana DeGette
Mr. Chairman, I want to thank you for holding today's
hearing on the future of stem cell research. Over the past year
there have been many important developments in the field of
cell-based scientific research. We saw breakthroughs and
accomplishments that couldn't have been predicted even months
before they happened--insulin producing islet cells created
from embryonic stem cells, induced pluripotent stem cells (IPS)
developed from adult skin cells, and primate embryonic stem
cells generated through somatic cell nuclear transfer (SCNT).
All of this proves that one can rarely predict the outcomes of
scientific research and underscores why it is crucial to make
the investment in all ethical forms of research to begin with.
This is what we are going to explore during this hearing: where
we are now and where we are going with stem cell research.
Everytime there has been a new discovery in some type of
research besides embryonic stem cells, the Bush Administration
tries to claim that it is a substitute for embryonic stem cell
research. Yet, in actuality the numerous types of cell-based
research are all complementary--they aide future developments
or provide the background necessary for some yet-to-be-
discovered breakthrough. It simply does not make sense to
remove one avenue of research from the equation--we should
continue pursuing all forms of ethical research and see where
the science takes us.
It is important that we still pursue embryonic stem cell
research, for example, since it remains the most promising
avenue of research for certain debilitating diseases like
diabetes, Parkinson's, and Multiple Sclerosis. However, there
is still plenty to learn about both embryonic and induced
pluripotent stem cells. Embryonic stem cells, as the vast
majority of scientists agree, are currently the gold-standard
for stem-cell research, and are the basis upon which to measure
the success of IPS cells. The goal of IPS cell research is to
make them mimic embryonic stem cells. But, how are we ever
going to know whether the IPS cells are acting like embryonic
stem cells if we haven't done enough research on embryonic
cells to even know what we are looking for?
None of the recent progress in the adult stem cell field
would have even been possible without the original embryonic
stem cell research. Looking forward, we simply do not know
where the advances will come from for each of the many diseases
that we need to address-we do not know which will come from
embryonic stem cell research and which will come from IPS
research. We need to support both embryonic stem cell research
and IPS research and let the science decide which is more
promising over the long-run.
We do not yet fully know what the recent IPS stem cell
breakthrough means in terms of application. It seems as though
it will likely prove to be a significant scientific advance.
However, we do not yet know whether it will prove to be a
significant medical advance. For example, IPS cells currently
remain far too dangerous for actual treatment, and we do not
know whether they will ever be safe for humans. Cutting off
funding for other promising avenues of research in the meantime
would be about the most short-sighted things we can do. When we
develop new tools, we don't throw out the old ones that still
serve a valuable and unique purpose. Why should it be any
different when it comes to medical research?
Although we are making great progress in the field of stem
cell research, it has not progressed as far as it might have
had the Administration instituted a cohesive federal policy for
ethical oversight of stem cell research, rather than simply
banning the use of federal funding for research on embryonic
stem cell lines developed after August 9, 2001. Progress has
been even further hindered because of inadequate resources for
all research at NIH.
With all the new research coming down the pipeline, much of
which we have yet to even imagine, it is clear to me that we
need a comprehensive, ethical oversight framework for all cell-
based research, as well as a national commitment to a robust
research program in the United States.
So, in light of these issues, I have been working to
develop new stem cell legislation with my dear friend Mr.
Castle, who was kind enough to join us here today. We know that
NIH is best-suited to overseeing and coordinating all forms of
ethical stem cell research. It is best positioned to ensure
that all research meets high ethical standards, as it has long
experience overseeing cutting edge research and establishing
regulations that ensure the research is done ethically. So, the
new legislation will:
Construct a framework for ethical oversight of all
cell-based research, with NIH as a key player;
Ban certain unethical activities,
Lift the ban on federal funding for research on
embryonic stem cell lines developed after August, 2001.
Input from the experts in the fields is key to crafting
quality legislation, which is also part of the reason we are
holding this hearing. I look forward to a vigorous discussion
here today with our witnesses about where the science is
currently, where the science is likely to go in the future, and
what we, as federal lawmakers, should do in order to best
support and promote all the promising new research that our
scientists are working on.
Thank you, Mr. Chairman. I yield back the balance of my
time.
----------
Mr. Pallone. Thank you.
The subcommittee will stand in recess until the votes are
completed, about an hour, maybe a little less.
[Recess.]
Mr. Pallone. The subcommittee will reconvene. We were I
guess longer than we expected. We left off with Congresswoman
DeGette, and next I recognize the gentlewoman from Tennessee,
Ms. Blackburn.
OPENING STATEMENT OF HON. MARSHA BLACKBURN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TENNESSEE
Ms. Blackburn. Thank you, Mr. Chairman. I thank our
witnesses for their patience today. As the chairman said, we
were a little longer than we had anticipated being, but we do
appreciate that you are here. We are looking forward to what
you have to say.
We all know that embryonic stem cell research continues to
be a controversial issue. In my opinion, it does implicate
ethical and moral standards within scientific progress and has
the potential to offend millions of our constituents. It is my
understanding that no journals have shown any treatment trials
in human beings to have been successful using embryonic stem
cells but there has been successful stem cell research, most
definitely yes, from adult stem cells. In almost all cases,
adult stem cells are equivalent or superior to embryonic stem
cells and there are plenty of sources of adult stem cells,
amniotic and placental fluid, cord blood, bone marrow--and none
of these sources require any destruction of precious human
embryos.
But many organizations continue to push for funding for
embryonic stem cell research, claiming that it is the holy
grail for cures of many diseases. One particular disease that
is touted for support of embryonic stem cell research is
diabetes, but since 2002, published studies in stem cells in
diabetes journals concluded that trials using these cells
showed no cures, and most of the time the treatments resulted
in tumors, and I hope we will hear a little more about that.
Significant progress, however, has been made on treating
diabetes with adult stem cells, and since 2003, studies in the
same journal showed adult stem cells successfully treated
diabetes in mice, and when human trials conducted in Brazil and
Europe began to use adult stem cells for treatment, many of the
patients were insulin free after the stem cell transplant. The
Federal Government should not be funding research that is
showing no results and forcing Americans to pay for research
that requires the destruction of human embryos, research that
offends their moral and ethical sensibilities. Adult stem cells
have a proven track record, and the NIH should be focusing, in
my opinion, much of their research effort on this. I urge my
colleagues to consider what is laid before us today, to ask
good questions and to inquire about science that actually works
and shows results.
Thank you, Mr. Chairman. I yield back the balance of my
time.
Mr. Pallone. Thank you.
Next is the gentlewoman from Wisconsin, Ms. Baldwin,
recognized for an opening statement.
OPENING STATEMENT OF HON. TAMMY BALDWIN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF WISCONSIN
Ms. Baldwin. Thank you, Mr. Chairman. I really appreciate
the fact that you are holding this very important hearing
today.
I am a strong supporter of embryonic stem cell research. I
am fortunate to represent the University of Wisconsin, Madison
campus, where Dr. Jamie Thomson and his team were the first to
derive and culture human embryonic stem cells in a lab, and I
have had the opportunity to tour Dr. Thomson's lab and review
the work that happens in that lab, and the field is truly
groundbreaking.
Embryonic stem cells open the possibility of dramatic new
medical treatments, transplantation therapies, and cures, but
at 9 p.m. on August 9, 2001, the hope and promise of this
embryonic stem cell research was greatly curtailed by this
Administration's restrictions on the Federal research dollars
for embryonic stem cells. The President's policy that limits
Federal funding for embryonic stem cell research to those stem
cell lines that were created before a certain time and date is
arbitrary and irrational, and it needlessly ties the hands of
our scientists as they search for cures and treatments to
diseases and conditions like diabetes, Parkinson's disease,
Alzheimer's disease, and spinal cord injury. It also sends a
very negative message to young, upcoming scientists that this
is not the field to enter if you hope to secure Federal grant
funding to support your research efforts.
But despite the President turning his back on the promise
of embryonic stem cell research, I am pleased that many States,
universities and private research foundations have stepped in
to fill that role and the research has continued. Late last
year, the same Dr. Thomson that I referenced earlier announced
that he had discovered a way to reprogram skin cells into stem
cells that seem to act like embryonic stem cells. While this
development is very exciting, we must continue to support
embryonic stem cell research and explore all the possibilities
that this science holds. Whether we are talking about embryonic
stem cells, adult stem cells, cord blood stem cells, or these
new reprogrammed cells, we must explore all avenues of
research. We owe it to the millions of Americans who suffer
from diabetes, Parkinson's disease, paralysis, and countless
other conditions to realize the potential of all of this
research.
And I just want to close by associating myself with
Congresswoman DeGette's frustration, she said anger, over the
confusion between adult and embryonic stem cells and the
arguments that have been proffered. These stem cells have
different properties. I can't say with scientific accuracy that
it is like comparing apples to oranges but I can say that we
need to clarify the properties and why we need to pursue both
lines of research, and I hope that our expert witnesses will
help educate the members of Congress on this committee on the
different properties that those stem cells have.
Thank you, Mr. Chairman, and I yield back.
Mr. Pallone. Thank you.
Mr. Ferguson of New Jersey.
OPENING STATEMENT OF HON. MIKE FERGUSON, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF NEW JERSEY
Mr. Ferguson. Thank you, Mr. Chairman. Thanks for holding
this hearing.
I am sure many people are aware, as we have heard already,
that there have been great strides that scientists have been
making in the past several years in stem cell research, in
treating and even curing patients that have life-altering
diseases. Research has produced very exciting developments such
as the development that Ms. Baldwin was talking about, the
induced pluripotent stem, or iPS, cells, which are derived from
nonpluripotent cells by inserting genes to create the
pluripotent stem cell. In 2006, Shinya Yamanaka of Kyoto
University published the first article concerning iPS cells in
mice, and 16 months later, his group and a group led by, as was
said, Dr. James Thomson at the University of Wisconsin-Madison,
reported the creation of human iPS cells.
But I have to say, as Dr. Thomson himself has said, if
human embryonic--I quote, ``If human embryonic stem cell
research does not make you at least a little bit uncomfortable,
you have not thought about it enough.'' He is right. And
fortunately, there are better alternatives. There are more
promising alternatives. There are alternatives that are showing
treatments and progress in human beings today. Carol France is
sitting in front of us. She suffers from multiple myeloma. Five
years ago, my mother died from multiple myeloma. When she was
first diagnosed at age 52, she was told she probably had a year
to live. She lived 6 years because she had a similar treatment
that is extending Carol's life today. One of our children was
able to--when my mother was first diagnosed, she had no
grandchildren. Three of our kids were born in the 6 years that
her life was extended because of this stem cell treatment, not
an embryonic stem cell treatment where there are no treatments,
no humans that are benefiting from that today, but a treatment
that is benefiting Carol and countless other people today, not
just in cancers, but yes, there is progress in Parkinson's
disease. Yes, there is progress in diabetes as was shown in the
Brazilian study. It is true.
So when we are looking at where we spend scarce taxpayer
dollars on Federal research, let us look at what is working,
where the promise is, and not spinning our wheels going
elsewhere. You know, I think citizens are rightly concerned
about where their tax dollars are going, and in fact, my home
State, the chairman and my home State of New Jersey, just last
year, in New Jersey, embryonic stem cell research is done
privately. We don't even have a law against human cloning in
New Jersey so we are pretty so-called progressive State when it
comes to scientific research. But last year, voters in our
State rejected a $450 million embryonic stem cell research
center. Now, in the State of New Jersey, a ballot test hasn't
been defeated in 17 years, and in fact, there was another
ballot question on the ballot at the same time that would have
funded something else that passed. This is the only one in 17
years that failed. I think voters and citizens as they look at
the scientific evidence, I think as they look at the progress
and they see the great progress of adult stem cell research and
the people that it is benefiting today and they look at the
alternatives, I think they are seeing that our--the question is
not what is legal, the question is, where should we be spending
taxpayer money? Where are we going to get the most bang from
our buck? And I think people are beginning to see more and more
clearly, particularly because of the research of Dr. Thomson
and others, that there are very promising, very ethical
opportunities for this research and we don't have to go down a
route that frankly has a lot of the ethical baggage that
embryonic stem cell research has.
Thank you, Mr. Chairman. I yield back.
Mr. Pallone. Thank you, Mr. Ferguson.
Our vice chair, Mr. Green, recognized for an opening
statement.
OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF TEXAS
Mr. Green. Thank you, Mr. Chairman. I am shocked that New
Jersey doesn't have a ban on human cloning.
Mr. Ferguson. Me too.
Mr. Green. My concern is that some of my colleagues may
want to put that ban in effect in Texas and we would have no
Texans that sound like me here.
But be that as it may, there is not anyone in this room or
in our country who has a friend or family member or a neighbor
that hasn't suffered from diabetes, Alzheimer's, or Parkinson's
disease or a spinal cord injury, and how difficult that
struggle is. But the issue, and you hear it today, the
diversity of opinion is we can do what we need to do with adult
stem cell research and there has been some great strides, but
there is a substantial difference between adult stem cells and
embryonic stem cells, and that is why we need both. We don't
need to say we can only do it with adult, and that is what
frustrating about this debate.
Embryonic stem cells can actually divide indefinitely and
evolve into any cell type in our body, and that is the big
difference. We need to research it all and not just
artificially say we are not going to do something, and that is
what is frustrating. I have seen poll after poll the last
number of years since the President set his criteria that 70 to
80 percent of the people support embryonic stem cell research,
just because why we would put our head in the ground when we
shouldn't--when there is some potential for that. And I would
hope the next Congress, if not this one, would pass the
legislation again because it has been overwhelmingly passed in
the House and the Senate, obviously not enough to override a
veto, but hopefully we will pass it during the next Congress,
if not this one.
Mr. Chairman, I would like to ask my statement be placed in
the record, and thank our witnesses for their patience for all
our votes we had on the floor.
[Mr. Green did not submit a prepared statement for the
record.]
Mr. Pallone. Without objection, so ordered, and I think we
have completed our opening statements, so we will turn to our
witness, who has been waiting patiently here for 2 hours or so.
First of all, welcome. Dr. Elias Zerhouni is director of
the National Institutes of Health, and we appreciate your being
here today. We have 5-minute opening statements. They become
part of the hearing record, and you may in the discretion of
the Committee submit additional statements in writing for
inclusion in the record, and I now recognize you for 5 minutes.
Thank you for being here.
STATEMENT OF ELIAS A. ZERHOUNI, M.D., DIRECTOR, NATIONAL
INSTITUTES OF HEALTH
Dr. Zerhouni. Well, thank you, Mr. Chairman. It is worth
waiting 2 hours to discuss stem cell research, and thank you,
members of the subcommittee.
I am really pleased to appear before you today to testify
about the current state of stem cell research science and its
significance, its current prospects, and its likely future. But
let me start by saying that from the scientific standpoint,
this is one of the most important, if not the most important,
areas of medical research today. It has the potential to not
only treat millions of individuals but also allow us to
discover some of the fundamental findings and discoveries that
we need to make in this century if we are going to be effective
as a society in lessening the burden of disease.
The central issue which I would like to go over in my oral
statement and submit my total written statement for the record
is the significance of this research from the standpoint of
science. Why is it important that stem cell research be pursued
very aggressively? I have a panel that I would like to share
with you and I think we have distributed copies of that to each
member. But let me just tell you what the real mystery is for
us as doctors or scientists. It is the mystery of how DNA,
which is exactly the same in every one cell of our body, goes
from what we call a totipotent cell with the exact same DNA, to
then form a complete organism with over 260 different cell
types in what we know as ourselves. This is a fundamental
mystery that we need to unravel in this century. Why? Because
we know also that DNA has been sequenced. We know how the DNA
code is written. We know all the letters of the DNA code. What
we do not know is how it is played, how it is programmed. So we
know the hardware of how cells do this; we don't know the
software. And the whole field of stem cell research cannot be
separated from our standpoint into components of adult or
intermediate because they are all part of the same continuum,
and let me explain that for you. Clearly, when a totipotent
cell evolves, it plays a program, a program of molecular
factors that are timed to change the characteristics of the DNA
and how the DNA is played out. That then leads to a pluripotent
cell. That pluripotent cell has a very interesting
characteristic. It can self-renew. It can stay, in other words,
idle until it goes forward in development and then can create
through a second set of programs a program to create three
precursors of our body systems. One is a line called the
endodermal line. The internal organs, the guts, for example,
arise from that line. The second is the mesodermal mid layer
which really gives rise to muscles and bones and heart and
blood. And then there is the ectodermal line, the epilayer, the
outer layer, which gives rise to the nervous system and all of
the neurons and all of the superficial layers of the skin.
Now, we know that we can evolve a pluripotent cell into one
of these, and this is the discovery that Jamie Thomson was
credited for, finding that in fact you can cultivate these
pluripotent cells, these embryonic stem cell lines, and then
program them in different directions. This is where the
research has been very active.
Now, as we also learned, this is not the only program that
is played. You still need to go from this line, from this cell,
for example, the mesodermal precursor, and then you go through
a different series of what we call adult stem cells. So you may
have adult stem cells through multiple programs, many of which
are completely unknown to us. We know some; we don't know many
of them. And then these will then give elements of the blood,
for example, the white blood cells or the muscle, the deep
layers of the skin, the skin fibroblast.
Now, why is it important to understand that when we talk
about adult stem cells, embryonic stem cells, committed
precursors, it is very important to understand that this is a
whole, that in fact, when we look at embryonic stem cell
research, what we are looking at is to look forward in the
programming from a totally unprogrammed cell to a fully
programmed cell. Now, adult stem cells are partially programmed
cells, which are able to evolve into different end points. Now,
the therapies in adult stem cells have been developed for over
40 years, and the first one to be developed was the idea of
replacing the bone marrow in patients who had blood cancers
like myeloma or leukemias and so on, and the idea was to
eradicate the cancer cells and then fish from the bone marrow
some of these stem cell precursors to replace the bone marrow
in a healthy way.
So for most of the past 40 years, we have used that therapy
to treat many cancers, and over the past 10 years there has
been another line of research, which is to replace the immune
system. We have many autoimmune diseases--multiple sclerosis,
type 1 diabetes, lupus, scleroderma--and so doctors have had
the idea of using the technique that was developed for cancer
to use it to treat autoimmune disease where your own immune
system goes awry and attacks your own tissues. So the idea
there is to change that immune system, actually destroy it with
radiation and chemotherapy, and replace it with a healthy bone
marrow precursor that would then replace that. So
fundamentally, if you think about the central issue, the
central issue is, how is the software of DNA organized? We know
the hardware; we don't now the software. How do we discover how
that is organized in health and disease is the central
scientific question.
Now, when you look at this, as you know, scientists have
been looking at all angles of this research, and two things
happened between 2001 and today as we were able to fund for the
first time embryonic stem cell research. Researchers tried to
look for what is it that makes a pluripotent cell a stem cell,
and what they started to describe are DNA factors, genes, that
were active at that time and then they defined culture
conditions which allowed those cells to expand. Now, the thing
that is very important to understand is that embryonic stem
cells can be expanded many times and adult stem cells, up to
today are not something that is frequent in the body and that
we can expand as well as we do embryonic stem cells. So
researchers have been thinking, can we create a new source of
pluripotent stem cells, and this is the discovery that Dr.
Yamanaka made, Dr. Thomson. Dr. Daley, who is one of your
witnesses today, also showed the same thing, and that is that
you can take a skin fibroblast and with these same factors that
were discovered during embryonic stem cell research, apply them
to a fully programmed cell, and lo and behold, you can
deprogram the cell, erase the program, the software that was
there and bring that cell back to what seems to be the exact
same potency as the stem cell. It looks very similar but we
know already they are not identical. But they have the same
potential of being reprogrammed into the first three
precursors. Now, here is another important issue, and that is
that if you were able to cross-program these cells from a blood
cell to a neural cell to a pancreatic cell, you would have made
a great breakthrough. To this date, we have absolutely no
evidence that once you have a precursor, you can reprogram it.
So in summary, what I would like to say is that from the
scientific standpoint, adult stem cell research, embryonic stem
cell research, and induced pluripotent stem cell research are
the faces of the same coin. They are intrinsically
interrelated. They are related to the fundamental program of
learning how to program, reprogram, deprogram DNA so that we
can use these cells for therapies.
So I will stop here, Mr. Chairman, and I would be happy to
take your questions.
[The prepared statement of Dr. Zerhouni follows:]
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Mr. Pallone. Thank you, Doctor, and we have questions now.
I will start and recognize myself for 5 minutes.
My colleague--this is just a quick one. My colleague,
Representative Blackburn, commented in her opening statement
that embryonic stem cells have not produced any results and
that adult stem cells have shown more promise. You know, can
you just respond to that? I mean, just in general.
Dr. Zerhouni. I think it is correct that if you look at
clinical applications, because we started in adult stem cells a
long time ago, 1956 was the first animal bone marrow
transplant--we have learned a lot more about this and how to
use that in many other diseases, primarily in two conditions:
cancer and autoimmune diseases. Most of the diseases that are
today helped by adult stem cells fit into these two categories.
So it is absolutely clear that it takes about 17 years for the
development of an idea to the first trial. We have had a lot
more time in adult stem cells, a lot more funding----
Mr. Pallone. But what about the promise of the embryonic?
In other words, she said they haven't produced any results but
is there still promise out there for embryonic?
Dr. Zerhouni. I think absolutely. I think that it is true
that if you look at the snapshot of today, that we have made
more clinical applications available. If you look at the
scientific question, as I described, discovering the program
that will make those things happen, it is very premature to say
that one has promise and the other one doesn't.
Mr. Pallone. Now, one of the witnesses--I hate to do this
when I ask you about something the next panel is going to say
before they have said it, but one of our witnesses on the next
panel, Dr. John Fraser, asserts, and I quote, ``that increasing
funding to embryonic stem cell research means a decrease in
funding to other stem cell research. Increasing funding to
embryonic stem cell research at the expense of funding adult
stem cell research means that valuable clinical opportunities
that are serving patients today and others that appear on the
cusp of doing so will be sacrificed for a technology and
approach that while scientifically interesting contains
enormous obstacles before responsible clinical application can
be contemplated.'' Did you want to comment on that as well?
Dr. Fraser. That has been removed from my testimony. That
is an old version that is not part of my testimony today.
Mr. Pallone. All right. Let me say for the record that I
appreciate what you said, but his comments are not part of the
record until he gets up here and testifies later. But if you
would just--all right. Let me--it is a little bizarre. You are
saying you didn't say this?
Dr. Fraser. I am saying that I amended--the document that I
sent was amended, and you have an older version.
Mr. Pallone. Oh, OK. Well, you can comment on the older
version then.
Dr. Zerhouni. I have to tell you, I think it is premature
to make statements as to the ultimate potential of one or
another. It is all interconnected. It is all the same problem.
I don't know where the breakthroughs are going to come from,
and if I don't know, then I don't want to close a door without
thinking about the consequences of doing that. There are ways
of doing it ethically, and I think we need to really think
about those. There is no doubt that our scientists are just as
concerned as anybody else in finding solutions that are
ethical, but I think we can't just completely shut a door with
the knowledge that we have today. As the director of NIH, we do
not know enough to know where to stop, when to stop one kind of
research or another.
Mr. Pallone. OK. If we could just stop the clock a minute,
I just don't want the reporter to have difficulty. We have
never had that before in my experience where somebody talked
who wasn't part of the panel. Are you able to handle that?
The Reporter. Yes, sir.
Mr. Pallone. OK. So you have his comment, both of his
comments?
The Reporter. Yes, sir.
Mr. Pallone. Then let me ask you, let us go back, Dr.
Zerhouni. Can you explain to me the significance of this date,
August 9, 2001, that the President has chosen? You know, he
says no Federal funding for research on stem cell lines derived
after August 9, 2001. What is the significance of the date? I
mean, does it relate in any way to research or the scientific
evidence?
Dr. Zerhouni. I remember that the Federal Government could
not fund any research deriving embryonic stem cells because of
the Dickey-Wicker amendment. There is an amendment on the books
which prevented NIH to fund any embryonic stem cell research
deriving embryonic stem cells. The President made a decision to
allow research to proceed and be funded for cell lines that had
already been derived so that there would be no further
destruction of embryos. That is what I understand the logic of
the decision to be. I wasn't involved in the decision. But the
2001 date was a date which the President made a decision to
fund what was developed prior to this, including Dr. Thomson's
lines and so on and many others, but not any further.
Mr. Pallone. But there wasn't any scientific significance
to the date?
Dr. Zerhouni. No, I don't think that the decision was based
on purely scientific considerations.
Mr. Pallone. OK. Now, do you believe that NIH is in danger
of falling behind other countries with respect to biomedical
research due to the restrictions that are based on that August
9, 2001, date?
Dr. Zerhouni. It is very difficult to state categorically
one way or the other. There is no doubt that about 50 percent
of all the research that is published is currently published
with results that are coming from NIH funding of this research.
But there is no doubt that the rest of the world is also
advancing. Fifty percent is published by the rest of the world.
So I don't think that it would be--it is hard to predict but I
don't think it would be in our best interests, if you will, to
not continue to proceed in understanding the DNA programming,
reprogramming issue that I think is core to biology in the 21st
century.
Mr. Pallone. Thank you.
Mr. Pitts.
Mr. Pitts. Thank you, Mr. Chairman.
Thank you, Dr. Zerhouni, for your testimony. First, how
much NIH funding has gone toward human adult stem cell clinical
trials beyond bone marrow transplants?
Dr. Zerhouni. Total funding for human non-embryonic stem
cells is $203 million.
Mr. Pitts. Does that include the bone marrow transplants?
Dr. Zerhouni. I would think it does on all applications of
adult stem cells.
Mr. Pitts. How much funding has NIH provided for human
embryonic stem cell research and animal embryonic stem cell
research?
Dr. Zerhouni. On a yearly basis, $203 million is a yearly
number. We have been funding human non-embryonic stem cells at
about $203 million, human embryonic stem cells at about $41
million a year, and non-human embryonic stem cells probably
$150 million but I will check that number for you.
Mr. Pitts. How much NIH funding has gone toward the new
human iPS research?
Dr. Zerhouni. The new iPS research, if you looked at many
of the funding, for example, Dr. Daley, who is here, was funded
by NIH as a Pioneer Award winner from the NIH, but the total
before the discoveries were made is about $4 million. But we
have, as you know, launched a program to encourage this area of
research. and we are currently looking at proposals. It is a
recent discovery, so you couldn't fund it as much until it was
discovered.
Mr. Pitts. Now, how many vials of stem cells does NIH have
available?
Dr. Zerhouni. I don't know the exact number but I can tell
you that we have shipped about 1,400 vials of human embryonic
stem cells from our stem cell bank. I don't know how many are
available in the stem cell bank.
Mr. Pitts. Have you ever turned down requests for a sample
of the approved lines due to lack of availability?
Dr. Zerhouni. I am not aware of that, but I know that
scientists will tell you that there are lines that they wish
not to use because there have been changes in the quality of
those lines. So they tend to use fewer lines than all 21 lines
because some of them don't necessarily function as they wish.
Mr. Pitts. Of the approved lines, how many have not yet
been developed for research?
Dr. Zerhouni. So we had initially 71 unique derivations,
and about 21 have been developed and expanded and are available
for research. About the same number were attempted to be
developed but failed. The failure rate is quite high in
developing these lines. And there are about 25 or 30 which have
not been developed, have not been expanded for various reasons.
Mr. Pitts. Is it possible that some of those lines were not
developed on mouse feeder cells?
Dr. Zerhouni. It is possible. Most of the--all the lines we
have currently expanded have been developed on mouse feeder
cells, which was the technology at the time.
Mr. Pitts. Do you have any idea of how many were not
developed on mouse feeder cells?
Dr. Zerhouni. I think we know that the Goteborg University
in Sweden has 16 derivations which have not been developed at
all and are attempted to be developed on human--on non-mouse
feeder cells.
Mr. Pitts. Now, you have stated before that the Bush-
approved human embryonic stem cells are contaminated. However,
Dr. James Thomson has stated that these cells can be washed and
the contamination is not a problem. Are you aware of the study
published by Dr. Thomson?
Dr. Zerhouni. So we looked at that several years--I don't
know I declared that but we did look at this very carefully,
and we have pointed out in testimony as well as in written
statements that the fact that something is grown in mouse
feeder cells makes applications much more difficult and FDA
approval more difficult but not impossible. We do have other
products like vaccines that have been developed in that way. So
our testimony does not say it cannot be done, but it is a lot
more difficult to do.
Mr. Pitts. Now, you said you weren't aware of any patient
being successfully treated with embryonic stem cells. When is
the soonest that you would anticipate clinical applications
using embryonic stem cells?
Dr. Zerhouni. The one current clinical application at the
FDA is one by a company, Geron I think is the name, G-e-r-o-n,
for using human embryonic stem cells for spinal cord injuries.
That is the only one that is near clinical application, has not
yet been approved by FDA for trials.
Mr. Pitts. And how would treatments be affected by their
propensity for tumor formation?
Dr. Zerhouni. That is a problem you need to resolve before
you can implant human embryonic stem cells. This is why most of
the researchers working with human embryonic stem cells need to
continue to work on these programs so that they can move the
cell to a point where it will no longer develop a tumor.
Mr. Pitts. Thank you, Mr. Chairman. I think my time is up.
It is hard to see.
Mr. Pallone. You still have another 25 seconds if you want
to use them.
Mr. Pitts. Well, I will ask one more. Why did NIH not fund
clinical trials for Harvard researcher Denise Faustman even
though she reversed diabetes in mice and was FDA approved to
start trials?
Dr. Zerhouni. I am a little stumped on this one. I don't
know the details of this particular researcher and the
particular trial. so I will get back to you on the record for
that.
Mr. Pitts. All right. Thank you, Mr. Chairman.
Mr. Pallone. Ms. DeGette.
Ms. DeGette. Thank you very much, Mr. Chairman.
Dr. Zerhouni, I want to ask you, you said in your opening
statement that this type of research, the general category of
cell-based research, is one of the most important, if not the
most important, forms of research we can do going forward in
the future. What is the entire NIH budget?
Dr. Zerhouni. About $29 billion.
Ms. DeGette. Twenty-nine billion dollars. What is the total
budget for the cell-based research including embryonic and non-
embryonic?
Dr. Zerhouni. About $655 million a year.
Ms. DeGette. Six hundred and fifty-five million dollars a
year. So I think probably if Congress were willing to authorize
and appropriate a substantially higher research budget for all
of these types of research, the NIH could probably find some
people who would--some researchers who would be willing to take
those grants and to make them into some promising discoveries,
don't you?
Dr. Zerhouni. Definitely.
Ms. DeGette. Do you think the NIH would need to have more
research to really make this kind of----
Dr. Zerhouni. If we could have more resources, we could
accelerate this research much faster.
Ms. DeGette. And if we accelerated the research faster
without predicting specific advances, what kinds of things do
you think could happen?
Dr. Zerhouni. Well, clearly, as I said, the scientific
community is making rapid progress in understanding these
factors, these molecular programming factors. Every week, every
2 weeks, we get a report of scientists, for example, developing
a very potent capable line, both in humans as well as in animal
systems. The question though is going to be, how fast can you
do this. Now, this deprogramming advance, this breakthrough,
happened because we learned of the factors that were in this
first program. Now, we are going to learn more and go forward
into this route, as we can fund scientists to do that.
Ms. DeGette. Will the current embryonic stem cell lines,
the 21, give or take a little, lines that still are allowed to
be used with Federal dollars by federally-funded labs be
sufficient to sustain this type of future research?
Dr. Zerhouni. Scientists will tell you that they need
access to more cell lines that are earlier in their history.
What happens is, as you cultivate a cell line, over time it
accumulates changes, both genetic changes and software changes,
program changes, which makes a lot of scientists say I would
rather have a cell which is early in this development right
here so I can understand the----
Ms. DeGette. Not to cut you off but what you are saying is
these cell lines that existed as of August 2001 are now getting
old from a research standpoint and the researchers would like
to have newer stem cell lines?
Dr. Zerhouni. Many researchers can use them, they are using
them, but many cannot.
Ms. DeGette. Now, the way it works, both with approved
lines at the NIH and also private researchers is, they take
cell lines that are developed from embryos which were created
for in vitro fertilization clinics and not used by the patients
and then slated to be thrown away, correct? I mean, these
embryos are----
Dr. Zerhouni. I would assume that is true, but I don't know
all the details of every case.
Ms. DeGette. OK. I will ask the researchers. I wanted to
ask you if you are familiar with this Brazilian diabetes study
that some have referred to today, and whether or not in fact
that study showed U.S. researchers that diabetes was curable by
adult stem cells.
Dr. Zerhouni. I am familiar with that study, and this is
the study that I think Congressman Pitts was mentioning.
Actually the study was conceived by a researcher at
Northwestern University and the idea there was this: type 1
diabetes is probably an autoimmune disease where your own
immune system is destroying your own cells. So again, along the
line of what I described where you use bone marrow stem cells,
adult stem cells to replace the immune system. The idea then
was, why don't we use the treatment that was developed for
cancer patients into young type 1 diabetes patients to prevent
the destruction of their stem cells. When that science was
reviewed by our ethics experts and by experts in bone marrow
transplants, it was felt that this would be unethical because
the mortality rate is 5 percent in these diseases. Now, you can
take that risk when you are dealing with a cancer that has a
life expectancy of a year, like leukemia, but the problem is,
type 1 diabetes is manageable today. We have patients who live
almost normal lives. So the risk-benefit ratio as assessed by
the ethical boards, the institutional review board said this
isn't something that should be started in children, we should
start it in adults perhaps or with a different risk ratio and
not go forward with----
Ms. DeGette. So that was never really in clinical trials in
the United States, correct?
Dr. Zerhouni. Not that I know of because of the ethics
issues.
Ms. DeGette. I have one last question. Right now does the
NIH have ethical oversight over the embryonic stem cell
research that is conducted at the State level or by private
firms?
Dr. Zerhouni. Well, as you know, we can only use Federal
funds for the approved uses of embryonic stem cells so we
cannot really have that oversight responsibility. I think that
this is something that I wish common ground could be found over
time. I think NIH has always played the harmonizing role and
prevented in fact unethical uses as well as promoted the good
use of science, so I would say that no, we do not, and I wish
we did.
Ms. DeGette. And do you think that the NIH would have the
capability of developing such ethical oversight over cell-based
research?
Dr. Zerhouni. Definitely I think NIH should have an
enhanced role in that. I think we have shown over the years
that we can do this. We have regulated, for example, gene
therapy through the Recombinant DNAAdvisory Committee for over
30 years. It has worked very well. And the same thing is true
now with biosecurity issues. I think we have the talent and
frankly, I don't know of any other organization in the world
that could do a better job than NIH.
Ms. DeGette. Thank you very much, and thank you for joining
us, Dr. Zerhouni.
Dr. Zerhouni. Thank you.
Mr. Pallone. Mr. Deal.
Mr. Deal. Thank you.
Dr. Zerhouni, let me first of all begin by thanking you for
the excellent job you do in managing and directing NIH. I think
political party affiliations and politics aside, I think
everybody feels comfortable with your leadership and your
knowledge of issues as you expressed on one of the more
difficult issue that all of us are confronted with, this one
that this hearing is about today, and I continue to be
impressed by your leadership and thank you for that.
Dr. Zerhouni. Thank you.
Mr. Deal. Let me ask you about one aspect. I wasn't here
but I was listening to you over my computer in my office as I
was doing some other things, and one of the things that is
interesting, at least to me, and I wish you would expound upon
it a little more, and that is the new human iPS research. Would
you expound on that a little bit more? What is the degree of
enthusiasm about this at NIH? Is it something that really has
great potential, do you think?
Dr. Zerhouni. I think it is one of the biggest
breakthroughs in stem cell research in recent years. We are
very excited about it. We want to explore it. Because the idea
that you can take a cell that has gone through full programming
and then using four factors, you can deprogram it to be able to
do other things, that is a venue that is extraordinarily
exciting. We are putting out requests for proposals. I know we
have received 29 proposals just in the first submission, the
majority of which are on iPS cells. And remember that iPS cells
are not just to replace cells in your body. They are also tools
to make progress in other areas. For example, if you have a
patient with a disease and you developed a pluripotent cell
from that patient, think about what you can do to discover new
treatments, new drugs, new therapies. Pharmaceutical companies
are very excited about this potential. You could reduce the
toxicity of drugs that today hurt patients because of heart
toxicity or liver toxicity. So you could create liver cells or
you can create heart cells and test the drug in vitro and
prevent the toxicity. So there are many more uses than just the
typical we are going to replace neurons or we are going to
replace diabetic cells, much more exciting than--and I have to
commend the scientists. Remember that what they did is, they
learned from embryonic stem cells and immediately applied it in
a way that will allow us to all go forward without the concerns
that many of us have about this research.
Mr. Deal. Well, thank you. I can see you have enthusiasm on
this.
Dr. Zerhouni. I surely do.
Mr. Deal. I think rightfully so, apparently. Although you
do not control all of the research that is being done,
especially on embryonic stem cell research, could you give us
some idea from your perspective the magnitude of research that
is being done that is not NIH-funded in this entire area?
Dr. Zerhouni. If you are referring to embryonic stem cell
research alone, we feel that, because of initiatives in several
States that the rest of the country spends more than we do at
NIH for the $40 million that we spend. If you look at the
totality though of what we do in this entire spectrum that I
described, which is really a continuum, it is all sides of the
same coin. When we look at that, we spend $655 million total,
which is higher than any other actor out there. California just
this week announced a $225 million investment in this type of
research. So I would say that if you look at non-Federal
sources, it probably equals the Federal investment, but I can't
really tell you because I don't know what is happening in
industry or in private entities.
Mr. Deal. Well, Mr. Chairman, I am going to yield back my
time, but again, thank you very much, Dr. Zerhouni, for being
here.
Mr. Pallone. Thank you, Mr. Deal.
Ms. Capps is recognized for questions.
Ms. Capps. Thank you very much for your patience with our
proceedings in the House, and I want to first of all say since
I didn't get to make an opening statement, how proud I am of
the National Institutes of Health. I am bragging about all
the--people know all the things that are wrong about Congress
and I say but there is at least one good thing that is
happening that really impacts lives in this country but it is
also our biggest gift to the world that we are able to do all
of that. That happens on the campus and other places as well.
I have a lot of questions I would like to ask you but I
want to start with one that was touched upon in the opening
remarks, and someone else may have asked you this. One of the
things we are clearly missing in the national policy on stem
cell research is a standard that we need that can be provided
for us in the way of ethical standards, an ethical framework.
We have now seen in my State of California and other States and
other private entities a lot of push forward because of the
lack of support from the Federal government. That is in one of
the best natures of our country as well. But what is clearly
missing from all of this from my perspective, but I would like
to learn from you, how do you regard the importance of an
ethical framework to guide both private and public research and
endeavors into all stem cell research?
Dr. Zerhouni. Without harmonious and coherent oversight,
which historically NIH has provided and is the best
organization in the world to provide, you can see a world where
different standards are going to be used. FDA will have real
trouble finding out whether the research in California is more
valid than the research in Washington or somewhere else. It
will slow down progress for all stem cell research, not just
embryonic stem cell research, because we need to characterize
exactly what those cells do. There is the risk of tumor
development. We need to control that. You cannot do that well
at the speed you need to do it. It is hard enough when it is
well overseen. It is, in my view, very shortsighted not to
oversee it at the Federal level.
Ms. Capps. So if we were able to pass legislation that
authorized Federal involvement, it wouldn't just be funding for
research through NIH, it would also be to provide that ethical
framework and guidelines for all of the research that is going
on?
Dr. Zerhouni. I think some common ground has to be found. I
really believe it is in the best interests of our millions of
patients and the best interest of our country to act in unison
when it comes to ethical oversight of any area of medical
research.
Ms. Capps. OK. In whatever time I have left, and you may
have touched on this before, but if there has been anything
left out, there were efforts underway before 2001 and advances
have been outside the Federal government's purview, both
through States and through private enterprise. What is missing
apart from that ethical framework? What could be the
contribution of providing funds for research specifically
through the Federal government? What would you do?
Dr. Zerhouni. There was no Federal funding of human
embryonic stem cell research before 2001.
Ms. Capps. Oh, I know that.
Dr. Zerhouni. And so we have had this 6 years experience of
how to do this. I think what in my view would be very important
is to get over in some fashion or another in a good way the
issue of providing scientists with avenues of exploration with
strong safeguards, strong ability for us to prevent some of the
rightly scary scenarios that could develop. So we need to have
that now because it wasn't that important in 2001 since the
science wasn't advanced, but I can tell you, it is advancing at
an enormous speed, and I think we owe it to ourselves to create
a new framework to oversee this research over time. Now, it
could be that you can separate funding from oversight. I mean,
there are many ways that can be done, but we cannot just say
stop this and do this and no oversight.
Ms. Capps. So in this vacuum, you say that some dangerous
or unintended consequence could be developing, putting some of
our citizens at risk?
Dr. Zerhouni. Well, let me just be frank here.
Ms. Capps. Yes.
Dr. Zerhouni. I get e-mails from clinics in various
countries that do not have the oversight structure we do about
promising treatments for stroke patients in the Dominican
Republic, other treatments in countries that just don't have
the oversight infrastructure we have. I am very concerned. As a
physician I am concerned. I know the despair of patients who
need treatment, and that can be abused and used. We have this
in cancer therapies, and we are seeing it in stem cell
therapies. Why would we let our citizens go in an unregulated,
not-overseen environment with the risks we know about this
research and say, go ahead, it is much better there than it is
here? It is not correct to say that, and I am very worried that
there will be people harmed by this.
Ms. Capps. So there is a moral component to this in terms
of our leadership, and these are our citizens, many of them who
are flocking to places because they have been promised certain
things?
Dr. Zerhouni. Absolutely. I mean, look, hope is hope, and
as you know, we need to really understand that.
Ms. Capps. Thank you.
Mr. Pallone. Thank you.
Our ranking member, Mr. Barton, recognized for questions.
Mr. Barton. Thank you, Mr. Chairman. I will be very brief.
I want to welcome you, Dr. Zerhouni. It is good to see you.
I haven't seen you in person in a while though we have talked
by telephone several times. I know the purpose of today's
hearing is an update on stem cell research, but I can't pass up
the opportunity to ask you to give us a brief review of the NIH
reform bill that this committee passed on a bipartisan basis at
the end of the last Congress. Could you kind of tell us where
that is and what, if anything, we need to do to help you
implement it?
Dr. Zerhouni. Well, first of all, let me thank you, Mr.
Chairman. I think you have accomplished what NIH needed to have
for many, many years. As you know, there had not been a
reauthorization of NIH for many years, and you have been able
to do this with your colleagues on a bipartisan basis and I am
very, very pleased and proud of the fact that both sides came
together in authorizing the NIH Reform Act of 2006.
The main impact of the reauthorization, in my view, is that
it has institutionalized the concept that as science is
becoming more complex, as science is also converging between
different Institutes, the NIH Reform Act has allowed us to have
cross-collaborations with a Common Fund so that no one is being
taxed, if you will, for doing the right thing across diseases.
Now, we know, as you just heard, that many diseases, for
example, multiple sclerosis or diabetes, are treated with the
same approach because they are all autoimmune diseases. Well,
those diseases obviously are taken care of by multiple
Institutes. So I would say that the fact that also in the same
year, the bill passed in 2006 and the Joint Resolution of
Congress, the appropriators then decided to fund the Common
Fund as a separate entity so that Institutes will no longer
have to contribute to that, I thought that was a great
statement of support. We appreciate it, and I think that you
will see results of that on a going-forward basis that I think
you would be surprised at the change in the ability of NIH to
address cross-cutting issues that go beyond any one Institute's
mission or Institute's focus.
Mr. Barton. Well, I have asked Chairman Dingell to hold an
oversight hearing where we could go into detail on it, so
hopefully he will do that in the near future.
Ms. DeGette. Will the ranking member yield?
Mr. Barton. Yes.
Ms. DeGette. As the Vice Chairman of the Committee, I will
tell you, and a big supporter of that bill, I also want to
thank you, Mr. Barton, for that legislation. I think it has
been great. And I have also spoken with Mr. Dingell about doing
oversight hearings and I expect we will be doing that this
year.
Mr. Barton. Anyway, it is good to see you, Doctor, and we
will hopefully welcome you back soon to talk on some other
issues.
I yield back, Mr. Chairman.
Mr. Pallone. Thank you, Mr. Barton. I was going to say that
now that you asked that question, we didn't need to have the
hearing, but I guess--I am just kidding.
Next we have Ms. Baldwin recognized for questions.
Ms. Baldwin. Thank you, Mr. Chairman, and I want to add my
words of support for the incredible and unique role that the
NIH plays in the world and in the United States. Following on
Ranking Member Barton's comments, I feel like my constituents
are beneficiaries in so many different ways, whether it is the
results of the research that is funded, the funding that comes
into research universities like the University of Wisconsin-
Madison or in my own case having been raised by my
grandparents, my grandfather was an NIH-funded scientist at the
university, so I am a beneficiary in yet another unique way of
NIH funding.
I want to talk about a couple of things sort of pivoting
off the questions that you have gotten about ethical concerns
and needing to have a harmonious oversight process. There is
another role that NIH plays, which is priority setting through
the process of reviewing the grant proposals, and because a
part of the overall stem cell research that is not being
conducted through NIH, I am wondering what comments you might
have of the role that NIH plays in priority setting in this
overall endeavor.
Dr. Zerhouni. Well, again, I think as you have seen through
history, NIH since 1945 has basically been the tempo maker for
science in many ways. The first treatments, for example, for
leukemia that changed the mortality in children from 95 percent
to 5 percent were done because of that process, and we need to
continue to do that. So my sense is that the more we have an
open understanding of how to run this forward, given the fact
that it is getting closer to clinical applications, needs to be
enhanced.
Ms. Baldwin. Now, I mentioned in my opening statement one
of many concerns I have about the current funding policy for
embryonic stem cell research is the message it sends to young,
upcoming scientists in terms of what direction they should go
in, but I am additionally concerned about the consequence of
the current Federal policy because in many ways, it seems like
we are maintaining two separate structures. I know in many
research institutions, they have to build and equip two sets of
labs, one that conducts NIH-funded research, a parallel,
oftentimes a whole building is constructed and lab equipment is
acquired. Do you have any sense of what sort of costs are
involved in this sort of dual structure that is occurring all
over the country?
Dr. Zerhouni. In all fairness, NIH does not impose
separation of physical facilities. We have been extremely clear
that you cannot use Federal funds for unapproved uses but you
can account separately within your own laboratory for that. It
is difficult to do though. Most of our researchers say, you
know, I don't want to get in trouble, I would rather separate
the two completely.
Ms. Baldwin. That has certainly been my reflection in my
home community.
Dr. Zerhouni. Right. So it is an impediment to the
researchers, who really want to do risk management in the
institutions. From our standpoint, we are satisfied with the
accounting procedures that we have put in place, and we haven't
had a case where there has been a significant issue that we
have been concerned about. But I think at the end, the
institutions, our concern about that--in California, I know
that the first $225 million are actually dedicated to building
separate facilities. I know that this is a concern out there.
Ms. Baldwin. Lastly, we have had some discussion about how
much funding has been devoted to the new iPS findings. Going
forward, what sort of growth do you expect in terms of
contributing funding to that?
Dr. Zerhouni. I cannot be precise, but I can see
exponential growth in the field of induced pluripotent stem
cells for the reasons that I mentioned. One, it is much more
practical, easier to do. It also highlights different ways of
programming DNA, as I said at the beginning. It has multiple
uses other than just the clinical use, because right now these
cells are not ready for clinical use. They are generated using
viruses that carry these factors, so we have to do more
research on them to find a way to use them safely in the
environment. But my sense is that already we know of many
researchers--you are going to hear from Dr. Daley, who is a
leader in that field--many researchers, many applications, and
researchers who are currently funded by NIH, redirecting their
research to that area. So you will see--I think you will see
major growth in that field.
Ms. Baldwin. Dr. Zerhouni, thank you very much for your
time.
Mr. Pallone. Thank you.
The gentleman from New Jersey, Mr. Ferguson.
Mr. Ferguson. Thank you, Mr. Chairman.
Dr. Zerhouni, thank you again for being here today. We very
much appreciate your testimony and your leadership at NIH over
the years. I think you know that I personally am an admirer of
yours and appreciate the dialogs that we have had over these
years.
Mr. Chairman, I just wanted to submit two things for the
record that I referenced in my opening statement. One is the
autologous--this is a Journal of the American Medical
Association study published April 11, 2007, documenting the
progress that has been made with adult stem cell research in
type 1 diabetes. I can give this to you. I would like to submit
it for the record, please.
Mr. Pallone. Without objection.
Mr. Ferguson. And the other was the quotation that I
mentioned from Dr. James Thomson that was in an article in the
Washington Post from November 30, 2007. I would like to submit
that.
Mr. Pallone. Without objection, so ordered.
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Mr. Ferguson. Thank you.
Dr. Zerhouni, I think you are familiar with Celgene
Cellular Therapeutics. They are a biotech company in my
district in New Jersey. They do--really one of the leaders in
stem cell research. They do really extraordinary work and they
have developed a clinical application to create blood stem
cells by using human placenta-derived stem cells along with
umbilical cord blood cells. The first application of this
particular technology was completed at Louisiana State
University Health Sciences Center, the Health Sciences Center
at Children's Hospital on March 28, 2008, just not very long
ago. It was big news. They treated a pediatric patient who was
suffering from acute lymphoblastic leukemia, which is a cancer
of the bone marrow and the blood.
Mr. Burgess. I think he knows.
Mr. Ferguson. Yes, I am sure he knows, but I want to have
it on the record. Thanks, Mike.
I think I began my statement by saying I am sure he knows,
but in any event, yet another example of remarkable progress
and potential treatments that are coming from again not an
embryonic stem cell source but a different source, in this case
placental stem cells and cord blood stem cells. It just further
highlights for me, we were talking about these essentially two
different things and I mentioned before some of the sort of
ethical questions obviously that are still out there, and you
have referenced many times in your discussion about the ethical
considerations as we look at these different types of research,
and clearly I think we would all agree that there are things
that we can do but ought not do in life, right? But that is
really not the question that we are talking about here today.
The question that we are talking about today is, should all
things that we can do be funded using taxpayer money? That is
really the question that we are getting at here today, and
voters in New Jersey just last fall decided embryonic stem cell
research was not something they wanted their taxpayer dollars
to go to fund. So that was one opportunity for voters to be
heard. But we have to have that conversation all the time
certainly in this committee and this subcommittee.
I wanted to pursue something you had talked about before,
and clearly for many people, even obviously Dr. Thomson and
others who have raised questions, certainly ethical
considerations about embryonic stem cell research, and I am
genuinely curious about this because I don't know where this
goes. If there are ethical concerns that some people, many
people have about embryonic stem cell research, the nature of
that research today, my question is, I guess I don't have a
good enough imagination or certainly not scientific expert
enough to know, where does it go when perhaps, as you said,
perhaps years down the road if some treatment or progress comes
from embryonic research--I talked to the researchers in my
district and in New Jersey about placental and stem cell and
cord blood research, and one of the things they love about that
kind of research is, when they come up with an application, as
they seem to have, there is a virtually limitless supply of--I
don't know what you would call it--raw material, you know. How
many children are born every day? How many placentas and cord
blood, you know, we have a virtually limitless source of these
cells. If we were to get to that point some years in the
future, as you have said, with embryonic research, where does
the raw material come from?
Dr. Zerhouni. You are asking extremely important and
difficult questions. I think we absolutely do not want to
obviate the need for a deep conversation. Likewise, scientists,
as I described, believed that progress will come from our
understanding at the deepest level of the molecular program
that is timed to create the cells or create the appropriate
neurons or that lead to understanding disease and eventually
cure it. Most scientists when you talk to them, in my
conversations with them, including Dr. Thomson, would say the
picture 20 years down the line is that as we discover these
programming factors, we probably won't need any particular one
source. We will program, if you will, the software of any cell.
Am I talking about science fiction? No, it has already happened
in front of us; with four factors we have reprogrammed skin
fibroblasts. So I think the discussion will evolve, and you are
right. Is there anything that we could do that should not be
done? I just gave you an example of the autologous bone marrow
transplants in young type 1 diabetes patients where we know
that 1 in 20 will die within 100 days of having received that
transplant. That is just as important a consideration as the
other consideration that you referred to, which is what is the
limit, what is the barrier here. We clearly as scientists--now,
I am talking from the scientific point of view--if you
understand that the problem for us is to truly advance the
cures that we need to implement which are dependent on our
understanding of DNA programming and reprogramming and how do
you modulate that, the embryonic stem cell is just unique in
the sense that it can self-propagate. If it wasn't for that, I
don't think scientists would be as excited about it. The fact
is, you cannot get cord blood cells to multiply the way you get
an embryonic stem cell to, but that doesn't mean it is not
possible because we also are showing that it is doable. So,
Congressman Ferguson, I know you have thought about this, and
we have had these conversations. I don't know where the happy
medium is, but I know that we cannot close our eyes to the fact
that the progress may come from any one of these sources. So
NIH wants to fund all of those areas of research, whether it be
cord blood or placenta, and we do.
Mr. Pallone. We are out of time, 3 minutes over.
Mr. Ferguson. If I could just close on that and respond, I
think that is a very thoughtful answer. I appreciate it. The
researchers that I have talked to in our district and in----
Mr. Pallone. Mr. Ferguson, not for anything but we are 2
minutes over so we have to move on.
Mr. Ferguson. Sorry.
Mr. Pallone. All right. Ms. Baldwin? Oh, she is not here.
Mr. Burgess.
Mr. Burgess. Thank you, Mr. Chairman. Let me just yield 30
seconds to my friend from New Jersey to finish his thought.
Mr. Ferguson. That is extremely courteous, Dr. Burgess.
Thank you.
I would just say that the researchers that I have talked to
in our district have raised that question with me, and these
are all folks who agree, and I think everyone would agree that
all types of research, particularly as we have seen embryonic
stem cell research and these other types of research, are
extremely interesting, extremely interesting and potentially
valuable. I think anybody who is being honest would have to
acknowledge that they are potentially valuable. The question
that we are struggling with here is not whether it should
exist, not whether the embryonic stem cell research should
exist. That is not the question that we are dealing with today
or have been dealing with. The question is, where are we going
to spend scarce taxpayer dollars? On the most promising,
immediately beneficial examples and research or are we going to
roll the dice on other forms? That is really the question that
we are after today, so I appreciate it. Thank you, Dr. Burgess.
Mr. Burgess. You are very welcome. That is a rhetorical
question. It doesn't require a response.
Let me ask you--Mr. Ferguson raised another very good
point. What about just the volume of material that is going to
be required to do the type of research or to provide the
therapeutic benefit? There is a virtually unlimited supply of
cells from amniotic fluid and cord blood and a relatively
finite supply of human embryonic stem cells, regardless of
whether or not any funding source is lifted.
Dr. Zerhouni. Well, as you know, because of the new
discoveries, we have sort of bypassed this issue of being able
to expand the cells that we have through the induced
pluripotent stem cells. There is no doubt that when you look at
placenta or cord blood, we have--we are unable to take a cord
blood sample and expand it to use in patients other than young
children.
Mr. Burgess. But if I could interrupt you, what about the
pluripotent cell from amniotic fluid?
Dr. Zerhouni. Well, there is one documented work from Dr.
Atala there and we are looking forward to see what the
expansion potential of that is there, but there is no doubt
that scientists will explore every door. The one thing that we
don't know is where the magic answer is. So everybody is really
going to explore all of those avenues. We want to support them
all.
Mr. Burgess. And we talked a little bit about funding, and
I wish I had a great deal of time to spend on that, but as far
as Dr. Anthony Atala's work is concerned, is any of that
supported by NIH funding?
Dr. Zerhouni. Oh, definitely, yes.
Mr. Burgess. So he has an ongoing grant from NIH?
Dr. Zerhouni. Oh, yes, I think he has had it for a long
time.
Mr. Burgess. And reports are coming back to you so you are
able to evaluate the work that is going on down in North
Carolina?
Dr. Zerhouni. Absolutely. We are keeping a close eye on our
investments.
Mr. Burgess. And I am happy that you do. Let me just ask
too on this, since Mr. Barton brought up the issue of the
reauthorization and the $29.5 billion that was the baseline
funding in the reauthorization bill and the increases were
slated to be 5 percent per year. Were you able to get to that
amount last year in the appropriations cycle through Congress?
Dr. Zerhouni. No, and it is something that we will have to
consider in the long term and look at the long-term impacts. I
think the increases have been below inflation, and we have
managed and tried to reorganize our priorities, but they have
not been at the authorization level.
Mr. Burgess. Correct, and the reason for that bipartisan
reauthorization was to give you the certainty of that funding
stream so that when you go out and hire young scientists to
start new labs, you will know that you will be able to continue
to fund that. I won't ask you to be a prophet here but what do
you intuit about this year's appropriations cycle as far as the
NIH is concerned?
Dr. Zerhouni. I appreciate your point, Dr. Burgess. I think
you are very aware. I know from our conversations that you
know, based on your own experience, that young scientists make
decisions not on the basis of today but on the basis of what
they see coming, and as we send a message that is discouraging,
there is a definite sense out there of young scientists
deciding not to go into science.
Mr. Burgess. I would just remind those who are in the party
in control that control now the appropriations process, we were
criticized when we were in control for leveling off the funding
for several years after a doubling and now it appear that even
in spite of the hard work that was done by both sides of the
dais in this committee in the last Congress that that doesn't
seem to be reaching the level that any of us had intended.
Let me just ask one last line of questions, and I mean to
get a response to this in writing. Currently, as far as the
treatment of diabetes, the ability to implant an islet cell
from a cadaveric source currently exists. Is that correct?
Dr. Zerhouni. That is correct.
Mr. Burgess. And NIH is using that and that is successful,
but those patients will have to take a drug to inhibit
rejection from that point on. And I don't think the human
embryonic stem cell has ever been able to produce insulin that
would impact blood sugar, but if it did, and if that cell were
then implanted like other islet cells have been implanted from
a cadaveric source, would that same requirement for taking
anti-rejection drugs be required for that individual?
Dr. Zerhouni. So it all depends on where the cell comes
from. If it comes from the patient himself or herself, no.
Mr. Burgess. Which is why the reprogramming activities----
Mr. Pallone. We are going to have to----
Mr. Burgess [continuing]. Are so exciting.
Mr. Pallone. One more question and that is it because you
are over too.
Mr. Burgess. For the anti-rejection medication.
Dr. Zerhouni. It would make sense but I would be very
careful, Dr. Burgess, because when you reprogram a cell with
outside factors and viruses and so on, it is not clear that you
won't have an immune response. This needs study, but in theory
you are correct.
Mr. Burgess. Very well. I will yield back, Mr. Chairman.
Mr. Pallone. Thank you.
The gentlewoman from North Carolina, Ms. Myrick.
Ms. Myrick. Thank you, Mr. Chairman, and I echo all the
accolades that others have said. We appreciate all the hard
work you do. Thank you for that.
I wanted to ask you, I was really astounded when I learned
that the scientists at Wake Forest and Rutgers had actually--
this was funded by the Pentagon, by the way--but they managed
to grow a human ear and it was generated from the stem cells of
a badly wounded Marine, and they grew it on the back of a
mouse, as I am sure you probably know, to be transplanted onto
the Marine, and my question is twofold. One, can you comment on
the promise of such research, and do you think that a bill like
H.R. 810 alone would allow for this same sort of breakthrough,
even though the embryo lines eligible for Federal funding under
the bill may not actually come from the patient, and would
scientists need to create or clone embryos in order to tailor-
make therapies like this in the near term, I am talking; not 30
years from now but in the near term?
Dr. Zerhouni. As I said, I think currently the difficulty
of using adult stem cells and using them clinically is less
because we have had a lot more experience. We have had 40 years
of experience. What you are referring to is tissue engineering,
which is the specialty that you are mentioning that you are
aware of, and in tissue engineering, we have learned how to
grow cells, for example, vessels or skin cells, on a 3-
dimensional basis. That is currently available. We have grown
skin, for example, for burn patients for many, many years
already. The real issue, though, is how do you change the
destiny of a cell to become an islet cell? So we know how to
make the same cell expand into the same cell. We don't
necessarily know how to take that cell, even though it is
pluripotent, into replacing a neuron. That is the prospect of
what we are doing, Congresswoman.
Ms. Myrick. I appreciate it. And Mr. Chairman, if you would
allow me, I have two articles by Dr. Atala at Wake Forest that
I would like to submit for the record, if I may.
Mr. Pallone. I looked at the one Mr. Ferguson gave me and
it was not easily understood, so I will ask that you give me
those copies and then we will take a look at it again, if that
is all right, and----
Ms. Myrick. No, it is fine.
Mr. Pallone. Let me take a look.
Ms. Myrick. Thank you.
Mr. Pallone. The gentleman from Oklahoma? No? OK. I think
that completes our questions, and thank you, Dr. Zerhouni.
Dr. Zerhouni. You are welcome.
Mr. Pallone. We really appreciate your testimony and all
that you did and all that you continue to do. Thank you.
Dr. Zerhouni. Thank you very much.
Mr. Pallone. I would ask our second panel to come forward.
I want to welcome our second panel, and let me introduce
everyone from left to right once we have the signs posted here.
Welcome. I will start with, on my left is Dr. John Gearhart,
who is the C. Michael Armstrong professor of medicine at the
Institute for Cell Engineering at the Johns Hopkins University.
And then we have Dr. Amit Patel, who is director of cardiac
cell therapy, the Heart, Lung and Esophageal Surgery Institute
Surgery at UPMC Presbyterian in Pittsburgh--I am sorry--UPMC
Presbyterian, McGowan Institute of Regenerative Medicine in
Pittsburgh, Pennsylvania. And then Mr. Douglas T. Rice from
Spokane Valley, Washington. Dr. George Daley, associate
professor of pediatrics for the Karp Family Research--I guess
that is your address, I am sorry--associate professor of
pediatrics at the Children's Hospital in Boston. And then we
have Mr. Weyman Johnson, Jr., who is chairman of the National
Multiple Sclerosis Society, and from my own State of New
Jersey, Dr. Joseph Bertino, who is interim director and chief
scientific officer for the Cancer Institute of New Jersey. Good
to see you again. And then we have Dr. John K. Fraser, who is
principal scientist with Cytori Therapeutics--I hope I got that
right--in San Diego, California.
And as I said before, we have 5-minute opening statements.
They become part of the record, and you may, in the discretion
of the committee, be asked to submit additional written
statements for inclusion in the record, depending on the
questions that we get to.
We will start with an opening statement by Dr. Gearhart.
STATEMENT OF JOHN D. GEARHART, PH.D., C. MICHAEL ARMSTRONG
PROFESSOR OF MEDICINE, INSTITUTE FOR CELL ENGINEERING, JOHNS
HOPKINS UNIVERSITY
Mr. Gearhart. Thank you, Mr. Chairman and members of the
Committee. Ten years ago, we had our first hearing in Congress
on embryonic stem cells. This was the result of the
publications from two laboratories of the discovery of these
cells. I had the privilege of being part of that, and Dr.
Harold Varmus was here at the time as the director of the NIH
and he at that time put forward what these cells could be used
for, the potential of these cells, and I thought it would be
interesting to the Committee to review his comments and then to
tell you where I see as an active researcher in this field
where our science is with embryonic stem cell research.
So the initial comment that he made was that these cells
could be a boon to basic science, to understanding human
biology and human development. And indeed, we see that one of
the primary uses to date of these cells is to understand some
of these very early events in embryogenesis for which there is
no other avenue of research to understand how we go from a
single cell egg up to an individual that has 200 trillion
cells. What are the processes involved? And so we and others
have used these cells and culture to discover new genes, new
genes that are involved in the formation of the central nervous
system, of the heart, and recently in our laboratory we
discovered 40 new genes in the very earliest stages of the
development of the circulatory system, which happens within the
first few weeks of our development. There is no other way that
we could have gotten this information, and these are critical
genes. We can demonstrate by shutting them down, manipulating
them as we do, that they are important in development. This is
just but one example of the use of these cells that are going
to be made in understanding how our program, the genetic
program that Dr. Zerhouni mentioned, is played out so that we
can get a handle on birth defects and ultimately on some of the
disease processes that occur in our bodies. And this has been
an extremely exciting development.
Secondly, he mentioned that these cells could be used in
the testing of drugs and factors directly on human cells
without having to subject patients to them, and we see this
happening now, of culturing a variety of different cell types,
having them in culture and subjecting them to different types
of toxins, drugs of different kinds, and see the response of
the cells without going through either animal models, which
sometimes aren't important, or directly a variety of different
human genotypes. This is occurring.
Also, we see remarkable work being done on figuring out how
we go through these lineages, how a single cell can become one
of the 260 different cell types. This isn't trivial. We have a
cell in culture that can form all of those cell types. How do
we get it to form just a liver cell or a dopaminergic neuron?
And we are figuring out these processes by trying to mimic what
is occurring in an embryo and then using that information to
direct the specialization of that cell. This is enormous from
the standpoint of saying, well, if we are going to develop some
kinds of therapies, we are going to have to get a homogenous
population of cells that we know what they are that we can put
into a patient. This is extremely important.
Another avenue he said would be the use of these cells in
transplantation research for diseased or damaged tissue, and we
now see in the published literature dozens and dozens of
examples of where cells derived from human embryonic stem cells
have been placed into animal models either for disease or
injury. Yes, there are variable outcomes to this but it shows a
great deal of promise.
So in all of these avenues, we are seeing this in research,
and I just want to tell you that is going to take a while. This
is something else that came out of this initial meeting, was
this projection of how long it would take for us to develop
these kinds of therapies for patients. It is going to take
years, and much of it is safety. We don't want to place cells
into a patient without knowing what their fate is going to be
and how we can regulate it, and we are getting a handle on that
in the modes of delivery, the types of cells we put in, whole
new--we have made radiologists even richer from the standpoint
that when we first went to the FDA, we were asked, if you are
putting in 300,000 cells, we want to know where every one of
those cells is going. We want live-time tracking of these
cells. So we are delighted at the progress of this.
Now, let me tell you how I----
Mr. Pallone. I hate to interrupt you all because what you
are saying is so important but we have a long panel, so you
have to wrap it up.
Mr. Gearhart. That is fine. Well, I have recently seen how
policy issues can trump science and I am very disappointed.
Reference was made recently to the Army's Institute of
Regenerative Medicine announcement of $250 million. I think you
should be aware that what was not permitted in those studies
was anything dealing with embryonic stem cells, and I just feel
that we are shooting ourselves in the foot by not also having
that avenue explored for some of this very important
regenerative medicine.
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Mr. Pallone. Thank you, Doctor.
Dr. Patel.
STATEMENT OF AMIT N. PATEL, M.D., M.S., DIRECTOR OF CARDIAC
CELL THERAPY, THE HEART, LUNG AND ESOPHAGEAL INSTITUTE, UPMC
PRESBYTERIAN, MCGOWAN INSTITUTE OF REGENERATIVE MEDICINE
Dr. Patel. I would like to thank the Chairman and the
members of the Committee for giving me this opportunity to
testify before you. I just have to make a quick note that the
testimony that I am giving today is of my own opinion and not
necessarily that of the institution that I am currently
employed by.
My career has really been developed and based on the
treatment for cardiovascular disease. I am a cardiac surgeon
and a translational scientist, meaning my goal is to take the
science that many of the panelists here have been doing for
longer than I have been alive that they first started and how
can we most safely and efficiently help the patients who have
the disease today, and based on that, cardiovascular disease,
as we know, is the greatest cause of death in America. There
are millions of patients every year who die from new heart
attacks, limb ischemia, not getting enough blood supply to
their legs, and the most end stage, which is about 5 million
patients with heart failure. Fifty percent of those patients
will die within 5 years of their diagnosis in the most severe
forms. So the question that I have and I try to help my
patients with is, I do bypass surgeries, I do valves, I do
heart transplants, but with our limited organs, the risks of
complications of anti-rejection medications, I have to find
other solutions that safely can help these patients just
because I can't help all of them, and every day I get calls
from patients from within the United States and around the
world, can you provide me a therapy, just as Dr. Zerhouni said.
But the key is, how we can do it safe and effective here in
the United States. And so there are two problems that we have
tried to solve and by no means have an answer to but have some
early treatments for is, for heart failure, our basic problem
is, we have a pump that just cannot supply enough blood by
delivering enough oxygen to the entire body, and in patients
with limb ischemia, these are patients that due to lack of
enough oxygen and blood supply to their legs, these patients
end up with amputations. So when you combine those together,
the total loss that was reported by the American Heart
Association in 2005 was $394 billion, $242 billion from the
healthcare expenditures and $152 billion from loss in
productivity from death and disability. So as we know how
dramatic of an impact this has on not just the capital
resources but human resources, that is the two things that we
have really focused on.
So our role of stem cell therapy really has been, well,
what do we want to do. It is great for these very complex
diseases and disorders such as Parkinson's and other
neurological or immunological problems, but our goal is very
simple. We need a heart that has more ability to pump by either
providing more cardiac myocytes, or heart muscle, and
increasing the blood supply, developing new blood vessels. And
in patients with limb ischemia, how can we develop more blood
cells that will prevent these patients from getting amputation.
So it sounds like a simple solution that we need to address, so
our goal has been, how can we help the patients today with the
cells that we have available that have been safe, and the
question of safety is always an issue.
Five years ago, when we first started some of the earlier
clinical work, that was a very significant concern and we
received one of the first FDA approvals to do human trials here
in the United States, and a similar group in Texas also
received this approval using bone marrow-derived cells, and it
is not to say that that was the perfect answer or solution
because before that, in France, patients had received biopsies
of their muscle from their thigh, they expanded them in culture
and injected them into their heart and caused significant
irregular heartbeats. So translating too early from the science
without knowing a lot of the answers is also not the right
answer, so there has to be a safe and ethical balance. But now,
when those same myoblasts in the United States were taken in a
safer fashion, delivered with a catheter in heart failure
patients, that is now expanded to a phase II 390-patient
clinical study that is funded by industry. So it is not that
the cells are bad, it is knowing the right indications for the
patients and the right way to culture them.
We have been able to take bone marrow in varied forms. The
earliest science and animal work showed great potential that
these bone marrow cells magically will become all these
different cell types. The reality is that this may happen in
the dish but it is very unlikely in our patients that this will
happen, but the key is, how can we most safely, effectively do
this, not only for our adult cells but all the other
multipotent cells that we are hoping to deliver, such as the
adipose cells, which you will hear about, amniotic cells,
placental, menstrual, and even the embryonic, so it is the
whole litany of cells. It doesn't matter where the cell comes
from, we still need to go through the same questions to how to
provide the most safe, reliable delivery of cells, also issues
of dosing. It is very similar to pharmacological therapies that
we need to know doses, toxicities, where are all these cells
going to go. I could put them in the heart. If I flush them
down the arteries in the heart, greater than 90 percent of the
cells end up in the lungs, liver, or spleen. The question is,
what are they doing there. So when it is their own cells, there
has been a level of safety now after about 8 years of
treatments throughout the world in registered trials. There is
probably about four times as many unregistered patients who
are----
Mr. Pallone. I am going to ask you to wrap up.
Dr. Patel. Sure. That in the 1,000 patients that have been
treated in registered trials, there has been definite safety
shown with bone marrow-derived cells. There has been a modest
improvement in cardiac function, and in the right selected
patients, there has been a very significant improvement that
has shown decreased death, decreased re-admission, and up to 5
years now the safety along with sustained improvement. There is
the possibility that these patients may need redosing, but the
biggest issue is, we have been benefited by the NIH. There is
the center of cell therapy, center of heart failure and cardiac
surgery where we could further answer a lot of these scientific
questions along with providing clinical therapies for patients
here in the United States today so they don't have to go
overseas and get unregulated and unscrupulous therapies where
they have to pay a lot of their own money. Thank you.
The prepared statement of Dr. Patel follows:]
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Mr. Pallone. Thank you, Dr. Patel.
Mr. Rice.
STATEMENT OF DOUGLAS T. RICE, SPOKANE VALLEY, WASHINGTON
Mr. Rice. My name is Doug Rice and I am 62 years old. I
have congestive heart disease and diabetes. I could be one of
over 750,000 people that die in the United States yearly, but I
am not dead, not because I shouldn't be, but there is a
resolution to this problem. I am not a miracle, a phenomenon,
but a living person that by the grace of God was saved from a
disease that kills approximately 2,000 people daily. However, I
had to travel to Bangkok, Thailand, and go in debt to do
something that should be readily available in the United
States. I used my own adult stem cells and a simple angioplasty
procedure to have my life given back to me. Your own adult stem
cells have so much more to give than we give them credit for. A
lot of other diseases are being treated successfully by just
using the adult stem cells.
My story is simple. In 1992, I had my first heart attack
and was also diagnosed with diabetes. That same year, my mother
died of congestive heart failure and diabetes, just like what I
have. Also just last year, my sister died of what I have. I
have had numerous heart attacks and diabetes episodes as well
as having to be jump-started at least three times. I have had a
TMR--that is a transmyocardial revascularization procedure--
that uses a laser to drill holes in the left ventricle to get
better blood flow. This did not help. In 1998, I was given only
2 years to live unless I received a heart transplant. Because
of my diabetes, I did not quality for it. We tried different
things that helped, and then in November of 2005 I could not
walk but a few feet. I had to sleep sitting up and was just
worn out. My ejection fraction, the amount of blood my heart
pumps out each beat, was around 11 percent. The average is over
50 percent. My cardiologist, Dr. Canaday, said at best I had 4
months without a mechanical heart pump to survive. It was
battery operated, and I decided that I did not want to be
battery powered.
That night my best friend, Sheba Rice, went on the Internet
looking for new heart treatments. She found Thera Vita, a
company in Bangkok, Thailand, that had been having success
using the adult stem cells. We contacted them, went to Bangkok
in January of 2006, and other than drawing blood, shipping to
Israel, then having the adult stem cells sent back and
implanted in me via angioplasty, it was simple. The hardest
part was the 20-hour flight. When I returned to Spokane, within
a month my ejection fraction was tested. It was 28 percent and
going up. I felt better than I had felt in years. I was
motivated to tell the world, and that is when I found out that
over 750,000 people a year die from heart disease.
These 750,000 heart patients that will die do not make the
mainstream press, no newspaper articles of any significance,
and certainly most politicians in Washington don't even like to
discuss it. Sadly, it is a fact, if a family dies in a car
wreck, children are gunned down in a school or a disgruntled
person shoots or maims his or her coworkers, it is big news.
But 750,000 people die at a rate of over 2,000 people a day and
no one takes time to talk for them. Not all are old. Some are
very young and with families and friends to care about. Most
people just don't realize that they die although almost
everyone knows someone that has died or will die from this
disease.
The Federal Government has spent millions of dollars on
embryonic stem cells but not one person has been treated and
the animals tested often get tumors.
By some estimates, over 400,000 people with various cancers
and other diseases have been successfully treated and most are
alive to talk about the adult stem cell treatment using their
own blood cells or ones from cord blood cells. The honest
experts say maybe in 10 or 20 years embryonic stem cells might
have potential to treat someone, but not now, and there is
something that works now. The adult stem cells work. What does
it take to make people realize that a bird in the hand is worth
two in the bush, especially when it comes to people's lives?
If you ask most people about stem cells, they only heard
about embryonic because that is all you hear about. Education,
education, education and the facts regarding adult stem cells
are the only way to succeed in moving this issue to the
forefront for funding and actual treatments now.
I get a lot of calls on a daily basis because I have been
treated with my adult stem cells, and the most frequent
question is, why did you have to go to Thailand? Because there
is no treatment available in the United States. I had to pay
for it myself. My insurance did not cover the costs of this
treatment, though I heard that in Germany, insurance covers
stem cell treatment for heart disease. I also know that much of
the stem cell debate in recent years has been drastically
increased funding for embryonic stem cells despite the fact
they have not treated patients for any disease. Patients are
being increasingly treated with adult stem cells but we need
drastically more Federal funding for adult stem cell
treatments. These cells aren't patentable, so private
investment is far behind. The government should spend more on
clinical trials so Americans like myself can have the same
chance at a treatment that I had. I am just one man, and all I
can do is talk to everybody I know, and it is a fact, you ask
anybody what a stem cell is, and the first word out of their
mouth is embryonic because that is all you ever hear. I listen
to every TV station, news station and you never hear the word
``adult stem cells.'' I am alive because of it.
Thank you.
[The prepared statement of Mr. Rice follows:]
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Mr. Pallone. Thank you, Mr. Rice.
Dr. Daley.
STATEMENT OF GEORGE Q. DALEY, M.D., PH.D., PRESIDENT,
INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH AND ASSOCIATE
PROFESSOR OF PEDIATRICS, CHILDREN'S HOSPITAL BOSTON
Dr. Daley. Thank you, Mr. Chairman and members for the
chance to testify. It is difficult to add much to what Dr.
Zerhouni talked about. He really gave a very spirited and
compelling argument in support of an integrated approach to
stem cell research.
I am here to give the perspective of a physician scientist.
I am from Children's Hospital and Harvard Medical School and I
am also the current president of the International Society for
Stem Cell Research. My laboratory studies blood development,
blood cancers, and various experimental transplantation
therapies, and in my clinical duties at the Children's Hospital
I take care of kids with a variety of blood diseases and so I
see firsthand the advantages and the limitations of the current
therapies such as adult stem cell therapies.
All stem cells, whether they are embryonic, fetal,
neonatal, adult, have great promise for medicine. However, I am
concerned because the recent breakthroughs in the reprogramming
of adult skin cells have renewed the calls for limitations on
embryonic stem cell research, and I wish to testify
unequivocally that enacting such limitations would be unwise.
My organization, the International Society for Stem Cell
Research, continues to assert, as do I think the vast majority
of scientists, that only through an expanded support for all
avenues of stem cell research can we ensure the most rapid pace
of discovery.
Much excitement in stem cell research has focused on this
remarkable property of embryonic stem cells, a property we call
pluripotency, that was described by Dr. Zerhouni. This is the
capacity for a cell to generate any tissue in the body. It is
an enormously valuable property. Recently several laboratories,
including my own, reported that a small set of genes which were
originally discovered because of their link to pluripotency in
embryonic stem cells, can be inserted into human skin cells to
convert them to a cell which is like a seed for all tissues in
the body, a cell that very closely resembles but may not be
identical to embryonic stem cells. I can show you the scar on
my forearm. We can do this with any patient, and in a matter of
weeks take skin cells and turn them into pluripotent stem
cells.
This is no doubt a major breakthrough in medical research
and it is going to have important implications for modeling
disease, and I certainly hope that one day it is going to usher
in new cellular therapies. But I have to caution and reiterate
the caution of Dr. Zerhouni that realizing this promise is
going to take time. A major concern for this new methodology is
the viruses that we use to carry the reprogramming genes. They
themselves are linked to cancer. And even if we can remove
viruses from this process, the genes and pathways that are
activated in the cells are also associated with cancer and we
don't know how these cells are going to respond. We don't know
what their long-term predispositions to abnormal growth or even
cancer might be.
Furthermore, I want to say that even though my lab has
generated these induced pluripotent stem cells, my lab will
continue to vigorously study embryonic stem cells. First, we
need to directly compare the properties of our embryonic stem
cells against the properties of our induced pluripotent, or
iPS, cells. And there are already some whispers in the
community and some preliminary data that iPS cells are not as
robust as embryonic stem cells for the formation of certain
tissues, but it is going to take years for scientists to
understand the similarities and differences.
I would also mention that even though we have iPS cells, my
laboratory will continue to vigorously pursue somatic cell
nuclear transfer. Reprogramming by nuclear transfer is faster
than gene-based reprogramming and may entail very different
mechanisms that will teach us a lot about how to make
pluripotent tissues better. The iPS breakthrough is being
heralded by opponents of stem cell research as a solution to
the long-smoldering debate over the necessity for embryonic
stem cells, and we have heard the arguments before. We heard
them in 2002 when multi-potential adult progenitor stem cells
were announced. We heard them later in 2004 and 2006 when fat
and amniotic fluid stem cells were announced and again we are
hearing them today. Congress has been wise not to yield to
these arguments. I remind you that it was basic stem cell
research that really led to the breakthroughs in iPS cell
research.
Yesterday I gave an address to the Congressional Biomedical
Research Caucus and I answered the question, ``Do we still need
embryonic stem cell research?'' with a resounding ``yes.'' And
I would say that embryonic stem cells remain the gold standard,
will remain so for the foreseeable future, and there is still
real value in passing H.R. 810, the original bill put forth by
Members Castle and DeGette.
I look forward to answering your questions in the Q&A
period. Thank you.
[The prepared statement of Dr. Daley follows:]
Statement of George Q. Daley
Thank you for the invitation to speak today on the subject
of stem cell science. My name is George Daley and I am an
Associate Professor of Biological Chemistry, Medicine, and
Pediatrics at Children's Hospital Boston and Harvard Medical
School, a core faculty member of the Harvard Stem Cell
Institute, an investigator of the Howard Hughes Medical
Institute, and the current President of the International
Society for Stem Cell Research (ISSCR), the major professional
organization of stem cell scientists worldwide. My laboratory
studies blood development, blood cancer, and experimental
transplant therapies for diseases like sickle cell anemia,
immune deficiency, and leukemia. In my clinical duties at
Children's Hospital, I care for patients with these devastating
blood diseases, and see first hand the need for better
treatments. Stem cell research offers hope.
Let me recount the stories of two patients I cared for
recently at Children's Hospital that illustrate the
shortcomings of current therapies. One was a young African-
American boy with sickle cell anemia, suddenly struck down by
what we call a pain crisis. When I saw him in the emergency
room, he was writhing on the gurney and whimpering in pain.
Despite powerful, high doses of intravenous morphine, I was
unable to give that child adequate relief from his pain and
suffering for several days. A second case was an infant who
suffered repeated infections and had spent half his young life
in the hospital hooked up to intravenous antibiotics. His
disease was immune-deficiency, and unfortunately he had no
sibling donors for a potentially curative adult stem cell
transplant. Stem cell research is laying the foundation for
improved treatments for these kids and countless other children
and adults with debilitating, life-threatening diseases.
All stem cells--whether from embryonic, fetal, neonatal, or
adult sources--hold great promise. The crowning scientific
achievement of the twentieth century was the sequencing of the
human genome, and the dominant mission of twenty-first century
science is to discover how that blueprint drives the formation
of tissues and organs, and how tissues are sustained, repaired,
and rejuvenated over time. Stem cell research goes to the core
of human biology and medicine.
Much excitement in stem cell research has focused on a
property of embryonic cells called pluripotency--the capacity
to generate all of the tissues in an organism. Recently,
several laboratories, including my own, reported that a small
set of genes linked to pluripotency in embryonic stem (ES)
cells can be inserted into human skin cells to induce
pluripotency--to endow skin cells with this same remarkable
capacity to become a seed for all tissues in the body. By using
gene-based reprogramming to make these so-called induced
pluripotent stem cells (called ``iPS cells''), scientists can
now produce customized, patient-specific stem cells in the
Petri dish. In a matter of weeks, we can take cells from a
patient's forearm and transform them into pluripotent stem
cells that we believe closely approximate embryonic stem cells.
This is a major breakthrough in medical research, empowering
scientists to create cellular models of human disease. It may
also mean that one day we will treat patients with rejuvenated
and repaired versions of their own tissues.
Realizing this promise will take time. A key concern is
that the viruses used to carry the reprogramming genes into
human skin cells can cause cancer. Moreover, the genes and
pathways the viruses stimulate are themselves associated with
cancer, raising the concern that even if viruses can be
eliminated from the process, the reprogrammed cells might
remain predisposed to cancer. For these reasons, iPS cells may
never be suitable for use in patients. I sincerely hope that
iPS cells are the long-sought-after customized patient-specific
stem cell, but much more research must be done.
Even with iPS cells in hand, my laboratory will continue to
study embryonic stem cells. First, we need to directly compare
the capacity of these two types of stem cells to generate
specific tissues. Some very preliminary data has suggested that
iPS cells may be less potent than embryonic stem cells in
making blood, while others are noting a deficiency in making
heart muscle cells. It will take years for scientists to
understand the similarities and differences between these two
valuable classes of pluripotent stem cells. Even with iPS cells
in hand, my laboratory will continue to investigate somatic
cell nuclear transfer as a means of generating pluripotent stem
cells. Reprogramming by nuclear transfer is faster and may
entail very different mechanisms than gene-based reprogramming.
Learning why may lead to better methods for making iPS cells.
The iPS breakthrough is being heralded by opponents of
embryonic stem cell research as a solution to the long-
smoldering debate over the necessity of embryonic stem cell
research. We have heard the arguments for many years, first
made when multi-potential adult progenitor cells (MAPCs) were
reported in 2002, and later when stem cells were isolated from
Fat and Amniotic fluid; we are told that alternatives are
available that preclude the need for embryonic stem cell
research. Congress has been wise to not yield to such
arguments. Indeed, it was embryonic stem cell research that led
directly to the breakthrough in iPS cells, and my own
laboratory was poised to generate iPS cells in large part
because of our experience and expertise in deriving and
culturing human embryonic stem cells. Today, it would again be
a mistake to place limits on the tools available to biomedical
scientists to pursue the next medical breakthroughs. The right
course for biomedical science and ultimately the right decision
for patients and our health care system, is to expand the scope
of federal funding for all forms of stem cell research,
including the many lines of embryonic stem cells created after
the President's artificial deadline of August 9th, 2001.
Yesterday, in my address to the Congressional Biomedical
Research Caucus, I was asked the question: ``Do we still need
research on embryonic stem cells?'' to which I replied a
resounding "Yes." Embryonic stem cells remain the gold standard
today and will remain so for the foreseeable future. If we are
to maximize the pace of scientific discovery and accelerate
development of new treatments for disease, we must continue to
vigorously pursue all forms of stem cell research, using ES
cells derived from embryos, pluripotent stem cells generated by
nuclear transfer and gene-based reprogramming, and adult stem
cells. Passage of the bill H.R. 810 originally proposed by
members Castle and Degette remains a worthy goal.
----------
Mr. Pallone. Thank you, Dr. Daley.
Mr. Johnson.
STATEMENT OF WEYMAN JOHNSON, JR., J.D., CHAIRMAN, NATIONAL
MULTIPLE SCLEROSIS SOCIETY
Mr. Johnson. Thank you, Chairman Pallone and Ranking Member
Deal. Thank you, all the members of the Committee. I am honored
to be invited to speak here today among many distinguished
panelists and to represent individuals who live with chronic
disease.
Expanded embryonic stem cell research will advance our
progress in many diseases, but today I will focus on one,
multiple sclerosis, and it is not because it is more important
than any disease, it is because it is the disease I know about.
It is the disease that comprises my story.
I first learned close-up about multiple sclerosis when I
was just a kid, 12 years old, and my father was diagnosed with
multiple sclerosis. He is no longer living, but late in his
life, MS affected him severely. His own sister, Allene was the
first person I met with MS. She was diagnosed in the mid-1950s.
I never knew Allene, unless she was in a bed or in a
wheelchair. When I was a child, I was told that incidence of MS
in our family was merely a coincidence. Today, through genetic
research, we know that it is simply not true.
In 1989, my own sister, Lanay, who is only a few years
older than I am, was diagnosed with multiple sclerosis. Today,
she uses a power wheelchair to move everywhere she goes. Her
hands don't work well anymore. She can no longer teach the way
she did in the public school systems in Georgia for many years.
She can no longer play the piano the way she did so
beautifully. When I think about the sanctity of life, I include
my sister's life in those thoughts.
A few years after she was diagnosed, I was diagnosed. In
our family, we hate this disease. We hate its impact on our
family and other families. We hate the threat it poses to
future generations. While I have not been severely disabled by
multiple sclerosis, I have seen its severe effects up close.
The scientific community is making progress into the
genetic factors involved in multiple sclerosis. There are still
more questions than answers, however. All kinds of research
must continue.
I remember being told that multiple sclerosis is a disease
that doesn't affect my friends in the African-American
community, that it is only for white people. With scientific
advance, we have found that is not true. We also used to hear
that this disease did not happen to children, but that is not
true either. We know now that there are thousands of children
in the United States and thousands of children throughout the
world who live with this disease. All kinds of research must
continue.
Before 1993, there were no treatments at all for multiple
sclerosis. Now we have six. But there is a wide spectrum of
disability among people living with multiple sclerosis. Most of
the available therapies work only for those on the lucky end of
the spectrum, like me. For people like my sister on the more
unlucky end, there are still few remedies. All kinds of
research must continue.
Every hour, somebody new is diagnosed with multiple
sclerosis. It is an unpredictable, often disabling disease of
the central nervous system. The progress, the severity, the
specific symptoms of MS in any one person still cannot be
predicted. The cause is unknown, and there is no cure. But
embryonic stem cell research holds unique promise to repair
nerve cells to slow the progression of MS and to find a cure. I
am just one person living with a chronic disease, but I am also
privileged to serve as the chair of the board of the National
Multiple Sclerosis Society. At the National MS Society, we
believe that all promising avenues of research that could lead
to new ways to prevent, repair, slow the progression or cure MS
must be pursued with adherence to the strictest legal and
procedural guidelines.
I salute Congresswoman Capps. She was chosen last year as
our organization's legislator of the year. We thank her for her
support for people with MS. I salute in absentia Dr. Burgess,
who is a member of the MS Caucus of the House of
Representatives. He and I might not agree categorically on
every issue but I appreciate his support and the support of
other Congresspersons for people with MS.
I am asking you today to expand Federal policy in embryonic
stem cell research and to ensure that research continues for
the more than 400,000 other Americans who live with MS and the
100 million Americans with other diseases and conditions.
Research on all kinds of stem cells is critical because we have
no way of knowing now which kind of stem cell will be of the
most value for MS, for Parkinson's, for Alzheimer's, for
cancer, for heart disease, for many other conditions. Just as
with genetics and race and age, there is much left to learn
about how to treat and cure MS, about how to treat and cure
other diseases. Expanding our embryonic stem cell research is
just one avenue.
As I close, I will note one side note. Our organization in
January of 2007, along with our sister organization, the MS
International Federation, sponsored an embryonic stem cell
symposium in San Francisco. The heartening part of that
symposium was that there was new research about repair that was
available. The disheartening part was that there were not very
many American scientists leading on the cutting edges. I think
that is a shame that we may have abdicated our leadership role
in the intellectual and scientific progress in the world. We
ask for your commitment not to give up on legislation like the
Stem Cell Research Enhancement Act. We don't have the luxury of
time. Like many others who live with a chronic disease, I know
that maybe not today, maybe not next week but I pray soon with
patience and continued research, there will be a world without
multiple sclerosis and a world of decreased disease.
Thank you very much for helping move us closer, and thank
you for your time.
[The prepared statement of Mr. Johnson follows:]
Statement of Weyman Johnson
Summary
Summary of my personal and family experiences with
a chronic, disabling disease.
Speak to a patient perspective on my own diagnosis
with multiple sclerosis.
Speak to the position of a national voluntary
health organization, as chairman of the board of the National
Multiple Sclerosis Society.
Speak to the need for continued research and the
hope it brings for people living with chronic diseases and
conditions nationwide.
Support the need for the Committee and Congress to
remain committed to legislation like the Stem Cell Research
Enhancement Act.
Embryonic stem cell research holds an incredibly
unique promise for people living with chronic diseases and
conditions, and the progress made to date on embryonic stem
cell lines should not be abandoned.
Testimony
Thank you Chairman Pallone and Ranking Member Deal. Thank
you members of the Committee. I am honored to be invited to
speak here today among many distinguished panelists and to
represent patients who live with chronic disease.
Many diseases could benefit from expanded embryonic stem
cell research. But today I will focus on one--multiple
sclerosis. Not because it is more important than others, but
because I know multiple sclerosis.
I remember multiple sclerosis and how it entered my life as
a child, in 1964, just barely 13 years old. My father received
a diagnosis of MS suddenly. He died in 2001. His sister, my
aunt Allene, also had MS. Research into this disease, into
genetics was just starting to evolve in the 1960s.
There were good doctors then, but they did not recognize a
genetic connection. They said MS in my family was a mere
coincidence. Because of research, we now know that is not true.
My own sister, who's only a few years older than I, lives
with MS. She uses a power wheelchair, her hands don't work well
anymore, she can no longer teach the way she did, or play the
piano the way she did. A few years after she was diagnosed, so
was I. We hate this disease, its impact on our family, and the
threat it poses to our future generations.
We are making progress into the genetic factors involved in
multiple sclerosis. However there are still more questions than
answers. The research must continue.
I remember being told that MS is a disease that doesn't
affect my friends in the African American community. This is
only for white people from Minnesota. With good science, we
have found that's not true. The research must continue.
We also used to hear that this disease does not happen to
children. But that is not true either. We now know there are
thousands of children in the United States, thousands of
children throughout the world, who live with this disease. The
research must continue.
Before 1993, there were no treatments at all for multiple
sclerosis. Now we have six. But there is a wide spectrum among
people living with MS. Most of the therapies will only work for
those of us on the lucky end of the spectrum like me. But for
people like my sister, on the more unlucky end, there's still
not much out there that provides effective treatment. So the
research must continue.
Every hour, someone new is diagnosed with MS. It's an
unpredictable, often disabling disease of the central nervous
system. The progress, severity, and specific symptoms of MS in
any one person still cannot be predicted. The cause is unknown,
and there is no cure. But embryonic stem cell research holds an
incredibly unique promise to repair nerve cells, to slow the
progression of MS, to help find a cure.
One area that holds great promise, but is often
misunderstood, is Somatic Cell Nuclear Transfer. We have seen
some exciting breakthroughs. But as with all science, this
research takes time. We are still exploring this avenue for
medical research. I have hope that SCNT will succeed because of
its promise to repair nerve cells, creating new tissues, and
more. I know that researchers are focused on the idea of
creating cells and tissues for transplantation and research.
They are trying to understand how different genes are turned on
and off. They are not focused on cloning. I know that as we
explore somatic cell nuclear transfer research more, we will
see greater potential for developing individualized cell and
tissue therapies. That holds great promise for people living
with MS like me, whose body's own defense system is attacking
the myelin surrounding and protecting our central nervous
system.
I am but one person living with a chronic disease. But I am
also fortunate to serve as chairman of the board of the
National Multiple Sclerosis Society. We believe that all
promising avenues of research that could lead to new ways to
prevent, repair, slow the progression, or cure MS must be
explored, with adherence to the strictest ethical and
procedural guidelines. The National Multiple Sclerosis Society
believes that all promising avenues of research that could lead
to the cure or prevention of multiple sclerosis or relieve its
symptoms must be explored. The Society supports the Stem Cell
Research Enhancement Act to expand the number of approved stem
cell lines that are available for federally funded research.
The Society supports the conduct of scientifically meritorious
medical research, including research using human cells, in
accordance with Federal, State, and local laws and with
adherence to the strictest ethical and procedural guidelines.
Research on all types of stem cells is critical because we have
no way of knowing which type of stem cell will be of the most
value in MS research. Stem cells--adult or embryonic--could
have the potential to be used to protect and rebuild tissues
that are damaged by MS, and to deliver molecules that foster
repair or protect vulnerable tissues from further injury.
So I ask you to expand the federal policy on embryonic stem
cell research and ensure that research continues --for the more
than 400,000 other Americans who live with MS and 100 million
Americans with other diseases and conditions. Research on all
types of stem cells is critical because we have no way of
knowing at this point which type of stem cell will be of the
most value--for multiple sclerosis, for Parkinson's, for
Alzheimer's, for cancer, for heart disease, for spinal cord and
brain injuries, for many other conditions.
Just like with genetics and race and age, there is so much
left to learn about how to treat and cure MS, about how to
treat and cure other diseases. Expanding our embryonic stem
cell research is just one avenue. But it is an avenue of
research that must continue. Federal barriers must be lifted.
You might see that I am not the only person living with MS
on Capitol Hill today. Hundreds of MS activists are visiting
with their legislators on the Hill right now, talking about the
need to advance medical research.
Embryonic stem cell research remains one of the most
promising avenues of research to cure diseases and end
suffering. I am not a scientist, but I am an observer of
science. And I know that science is a matter that requires some
patience. That's why we must expand the important work done to
date with embryonic stem cell lines. The research must
continue. So we can improve the lives of people with chronic
diseases and conditions. So we can improve the lives of
families for generations to come. For my grandchildren and for
yours.
We need your commitment to not give up on legislation like
the Stem Cell Research Enhancement Act. We don't have the
luxury of time. Like many others who live with a chronic
disease, I know, maybe not today, maybe not next week, but I
pray soon, with patience and continued research, that there
will be no more disease. Thank you for helping us move closer,
and thank you for your time.
National Multiple Sclerosis Society
Policy Position
Embryonic Stem Cell Lines Available for Federally-Funded Research
Position: The National Multiple Sclerosis Society believes
that all promising avenues of research that could lead to the
cure or prevention of multiple sclerosis or relieve its
symptoms must be explored. The Society supports the Stem Cell
Research Enhancement Act (H.R. 3 and S. 5) to expand the number
of approved stem cell lines that are available for federally
funded research.
The Society supports the conduct of scientifically
meritorious medical research, including research using human
cells, in accordance with federal, state, and local laws and
with adherence to the strictest ethical and procedural
guidelines. Research on all types of stem cells is critical
because we have no way of knowing which type of stem cell will
be of the most value in MS research. Stem cells--adult or
embryonic--could have the potential to be used to protect and
rebuild tissues that are damaged by MS, and to deliver
molecules that foster repair or protect vulnerable tissues from
further injury.
Request: We urge Congress to support the Stem Cell Research
Enhancement Act of 2007 (H.R. 3 and S. 5) at all levels of the
legislative process. This legislation would increase the number
of approved embryonic stem cell lines that can be used in
federally-funded research by allowing new lines to be generated
from embryos that have been donated for research purposes by
people using the services of in vitro fertilization clinics,
while establishing important ethical protections.
Supporting Rationale: There is broad agreement that the
policy limiting the number of stem cell lines available for
federally funded research is flawed.
An insufficient supply of stem cell lines
currently exists, as only 22 of the 70 approved lines are
available to researchers. In addition, all of the available
lines are contaminated by nutrients from mouse feeder cells.
Many in the scientific community believe that these stem cell
lines are unsuitable for research and hinder U.S. scientists'
ability to capitalize on the potential breakthroughs from
embryonic stem cell research.
At the same time, it has become increasingly clear
that stem cell research holds tremendous promise for MS and
many other diseases and disorders. Research suggests that stem
cells might have many uses: for delivery of growth factors and
drugs, for tissue culture systems for drug and gene discovery,
for understanding and modeling MS, and for repairing or
protecting brain tissue.
However, our scientific advisors have told us that
we still don't know which type of stem cells will be most
valuable for MS research, and thus we must support policies
that promote the conduct of research using all types of stem
cells.
----------
Mr. Pallone. Thank you, Mr. Johnson.
Dr. Bertino.
STATEMENT OF JOSEPH R. BERTINO, M.D., INTERIM DIRECTOR AND
CHIEF SCIENTIFIC OFFICER, THE CANCER INSTITUTE OF NEW JERSEY
Dr. Bertino. Mr. Chairman, members of the Committee, thank
you for inviting me to present my testimony today.
New Jersey has been a leader in supporting stem cell
research. In 2004, the Stem Cell Institute of New Jersey was
created by a memorandum of understanding between Rutgers, the
State University of New Jersey, and UMDNJ-Robert Wood Johnson
Medical School. The State then committed $8.5 million to
support work at the Stem Cell Institute, including $5.5 million
in capital funds to Robert Wood Johnson Medical School and
Rutgers University for laboratory renovations and GMP
facilities.
In December 2005, New Jersey became the first State to
finance stem cell research that included research on human
embryonic stem cells. The Commission on Science and Technology
awarded a total of $5 million to 17 research teams.
In 2006, the finance committee of the General Assembly
passed a $250 million bill to build stem cell research
facilities in New Brunswick, Camden, and Newark. One hundred
fifty million dollars of this was for a joint Rutgers-Robert
Wood Johnson Stem Cell Institute in New Brunswick. And just
last year, New Jersey awarded grants totaling $10 million to
stem cell researchers, including two grants to fund core
laboratories for embryonic stem cell research.
Despite polls that show that the majority of New Jerseyans
were in favor of supporting embryonic stem cell research, a
referendum was defeated in 2007 that would have provided $450
million over 10 years to support all stem cell research, not
only embryonic stem cell research. The major reasons for defeat
of the referendum were believed to be the off-year election,
with fewer than 30 percent of voters coming to the polls, and
the concern that this would add to the public's tax burden.
Governor Corzine continues to be a strong supporter of stem
cell research and the building of the joint Robert Wood
Johnson-Rutgers Stem Cell Institute in New Brunswick. Key
members of the New Jersey legislature also continue to strongly
support stem cell research.
For the past 2 years, over 50 investigators from academia
and pharmaceutical companies in New Jersey have been meeting
monthly to report their work in stem cell research, to discuss
progress in the field and to plan collaborative experiments.
Two types of stem cells are found in the bone marrow:
hematopoietic stem cells, that form blood cells; and
mesenchymal stem cells, capable of differentiating or forming,
for example, bone or cartilage or nerve cells. Hematopoietic
stem cells are now used at Robert Wood Johnson Hospital and
throughout the world to treat patients with cancer following
chemotherapy or immune diseases. Mesenchymal stem cells from
bone marrow or cord blood are being tested for their ability to
prevent graft vs. host disease after marrow transplantation,
and other uses under study by New Jersey investigators include
targeting tumors with mesenchymal stem cells carrying toxins,
and use in regenerative medicine, in particular spinal cord
injury and damaged hearts.
Researchers at both Rutgers and UMDNJ have special
expertise and interest in neural stem cells that have the
potential for treatment of brain disorders as well as to serve
as models to promote drug discovery.
We know that cord blood, placenta, and amniotic fluid are
also a rich source of stem cells. Clinical trials are in
progress, for example, by Wise Young from Rutgers, with
collaboration of investigators in China using a subset of cord
blood cells to treat spinal cord injury. The characterization
of stem cells from placenta is under study by Robert Wood
Johnson Medical School investigators in collaboration with
Celgene, a New Jersey-based biotech company.
Work on human embryonic stem cells, as you heard, has been
hampered by Federal guidelines that limit studies to 20 cell
lines that have been around for several years and have
limitations. Rutgers and Robert Wood Johnson Medical School
stem cell researchers with New Jersey State funding have been
able to expand research activities using newly established
embryonic stem cell lines, and importantly, the completion of a
GMP facility at the Cancer Institute/Stem Cell Institute which
allow stem cells to be produced in quantities necessary for
clinical studies.
The funding provided by the State of New Jersey has
provided key support for both the research outlined above and
additional research focused on a variety of important disease
conditions including multiple sclerosis, Parkinson's disease,
Alzheimer's disease, and diabetes, and a key part of our
efforts has been the establishment of stem cell banking of
umbilical cord blood and other stem cells. In New Jersey, stem
cell banks are leaders in this field.
I would be happy to answer any of the committee's
questions. Thank you very much.
[The prepared statement of Dr. Bertino follows:]
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Mr. Pallone. Thank you, Dr. Bertino.
Dr. Fraser.
STATEMENT OF JOHN K. FRASER, PH.D., PRINCIPAL SCIENTIST, CYTORI
THERAPEUTICS
Dr. Fraser. Good morning, Mr. Chairman, members of the
Committee. Thank you for this opportunity.
My name is John Fraser, and I am principal scientist at
Cytori Therapeutics, Inc., a publicly traded adult stem cell
company based in San Diego, California. Cytori is at the
forefront of bringing adult stem cells to patients as we are
currently selling a stem cell-based product in Europe,
conducting three separate clinical trials and have a technology
which has now been used in over 200 patient procedures. From my
graduate studies in New Zealand through to a post-doctoral and
faculty appointment to UCLA, my entire research career has been
in the field of adult stem cells.
The topic of today's meeting is consideration of stem cells
as the future of medicine, and indeed, stem cells will be an
important part of the clinical armamentarium going forward. But
as we have heard, this is nothing new. Hematopoietic stem cells
have been used in medicine for at least 50 years, and we
referred earlier to the pioneering work performed in the late
1950s by Dr. E. Donnall Thomas, who performed bone marrow
transplant studies that ultimately led to his award of the
Nobel Prize for Medicine in 1990. Like many, I consider 1961 as
the birth date of the stem cell field as that was the year that
James Till and Ernest McCulloch published research that led to
the description of the very first stem cell, the hematopoietic
stem cell, still widely considered to be the model for all
adult stem cell types.
Hematopoietic stem cells make bone marrow transplants
possible. This is because they have the ability to regenerate
the entire blood system of the recipient for the rest of that
person's life. Simply put, hematopoietic stem cells are the
regenerative engine of the blood system. In my opinion, this is
a key point of distinction between adult stem cells and
embryonic stem cells. Embryonic stem cells are capable of
immense proliferation and essentially universal plasticity.
This is because they are, first and foremost, developmental
cells. They are derived from a cell mass from which the entire
organism develops.
By contrast, adult stem cells are, first and foremost,
regenerative cells responsible for maintaining and healing
organs and tissues in the face of daily wear and tear, injury
and disease. They are, by their nature, repair cells. They act
in response to a need and they shut off once that need is
completed. One way to look at this is to view embryonic stem
cells as responsible for generating all the tissues of an
organism while adult stem cells are responsible for maintaining
and healing them.
The natural role of adult stem cells in repair and
regeneration makes them ideally suited to clinical use. This
has been proven in tens of thousands of bone marrow transplant
patients over the last 40 years. This paradigm, as you have
heard, is now increasingly being repeated as other adult cell
types associated with repair and regeneration are being applied
in different diseases.
In our own case, Cytori has initiated several clinical
studies using cells obtained from the patient's own fat,
adipose tissue, which is recognized as one of the richest and
most accessible sources of adult stem cells. The goal of these
studies is to bring forth new treatments for the millions of
patients suffering from heart disease as well as other issues
such as reconstructing the breast following partial mastectomy.
We also intend to start studies in intervertebral disc repair.
Other researchers have published case reports and small
clinical studies using fat tissue-derived stem cells and
treating certain kinds of wound complications with bone marrow,
GHVD, and in bone defects. Published preclinical studies have
indicated potential in treating renal damage associated with
chemotherapy, preserving dopaminergic neurons in a Parkinson's
disease model, treatment of liver damage, ischemic, and
hemorrhagic stroke, and in tissues as disparate as the cornea,
the lung, and the vocal fold.
Published clinical studies with other types of adult stem
cells have shown improvement in cardiac function, in inherited
brittle bone disease, liver disease, and peripheral vascular
disease, to name but a few.
However, as you have heard, there are still many unanswered
questions, and clearly, additional science is needed. In
certain settings, the mechanism through which adult stem cells
provide benefit is not well understood. It is also not yet
clear which adult stem cells provide greatest efficacy in which
diseases. These are important questions that companies such as
Cytori have neither the resources nor oftentimes the incentive
to address.
For example, certain potentially beneficial populations
fall outside of patent protections, providing limited incentive
for companies to invest their resources in proving a technology
that may then be applied without their participation. Without
Federal support, much of this promise could be left to wither
on the vine.
Cytori believes that ultimately science and the marketplace
will determine which technologies will succeed. We have looked
at the field of regenerative medicine, performed our own basic
science, preclinical, and now clinical research, and we are
optimistic regarding the ability of our approach to harness the
natural role of adult stem and regenerative cells to provide
clinically effective and cost-effective treatments for a range
of human diseases in the near future.
We urge your continuing support of adult stem cell
research. Thank you.
[The prepared statement of Dr. Fraser follows:]
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Mr. Pallone. Thank you, Dr. Fraser.
We will take questions now, and I will recognize myself for
5 minutes initially.
I have to start with you, Dr. Fraser, because of what you
said originally, and I noticed that you didn't make any
reference in your statement now to the fact that--and I will go
back to what you said in the previous one, that increasing
funding to embryonic stem cell research means a decrease in
funding to other stem cell research. I don't want to get into
it, but basically we had one statement earlier in the evening
and then it was revised, you know, based on what you said
today, and I don't see any more reference to this idea that
increasing funding to embryonic means a decrease in funding to
others. So why did you take that out?
Dr. Fraser. Sir, I received my formal invitation to attend
this meeting while I was at the airport in San Diego on my way
here. At that time there was a version of my testimony which
was under review and was sent to committee staff before it had
been completed. I contacted committee staff----
Mr. Pallone. Well, no, you are more than welcome to change
it. I am just asking why. Why is it no longer----
Dr. Fraser. I think the initial comments that I made
overstated the position. I think the point that was made there
and which is no longer is that you have to make difficult
decisions. You can't find everything, and for every dollar you
take away--sorry--every dollar you add somewhere else, you have
to take it away from somewhere else, and I----
Mr. Pallone. Well, not necessarily, but----
Dr. Fraser. Well, that would be nice, but we all know the
realities of the current fiscal and economic situation. I am
simply encouraging you not to take away funding from adult stem
cell research.
Mr. Pallone. Well, I don't think we are suggesting that,
but I mean, do you support embryonic stem cell research?
Dr. Fraser. The company has an official position which says
we have no official position regarding embryonic stem cell
research.
Mr. Pallone. What about you personally?
Dr. Fraser. I am not here as an individual, I am here
representing the company.
Mr. Pallone. So you just basically have no response to that
question?
Dr. Fraser. Well, sir, I have spent my entire career in
adult stem cells. That was not a conscious decision. That was
pretty much an accident when I was in graduate school. I am
very happy with where I am. I am certainly not saying----
Mr. Pallone. I am just trying to find out whether you
support----
Dr. Fraser. I am not----
Mr. Pallone [continuing]. Support embryonic stem cell, and
you don't want to answer that?
Dr. Fraser. Embryonic stem cells are valuable, and research
that has been performed under the NIH funding with the current
situation has produced valuable insights.
Mr. Pallone. All right. I will leave it at that. Thank you.
Let me ask Dr. Bertino a couple of questions, and thank you
again for being here today. I am obviously proud of my home
State in that we were the first to publicly finance embryonic
stem cell research, and of course, the new Stem Cell Institute,
which is going to be in my district in New Brunswick, but given
what our State and many other States are doing in terms of
taking the initiative on their own to advance embryonic stem
cell research, some have argued that there is no need for
additional Federal funding, you know, the States and private
sector can do it on their own. But can you speak to this? Do
you believe that New Jersey and other States with similar
initiatives have enough financial resources to achieve the full
potential that stem cells may hold, or do you think there is a
need for additional Federal funds?
Dr. Bertino. I think there is clearly a need for additional
Federal funds.
Mr. Pallone. I think the mic is not on, Doctor. There you
go.
Dr. Bertino. What we are seeing already is that more and
more investigators are becoming interested in stem cell
research because of the tremendous impact this paradigm shift
is having on medicine, and as we attract the youngest and most
talented researchers in this area, we have to provide them with
funds, and the State at this level cannot take care of all the
exciting research that is possible. I think if the stem cell
bill was approved and we did get the $450 million over 10
years, I think that would have been a major step in supporting
all the good research in the State, but that didn't happen.
Mr. Pallone. And what about the money that is being spent
in stem cell research in the United States versus, you know, in
other parts of the world? Is the United States on par with
other countries; are we falling behind? Is that going to
imperil our ability to recruit top researchers unless we spend
more money by comparison to other countries?
Dr. Bertino. I don't know the details and I can't really
answer that question. There are pockets of good research money
for stem cell research from different States,--I think
Connecticut, New Jersey, California--but there are many States
that have not stepped up to the plate.
Mr. Pallone. Well, let me ask Dr. Daley, if you don't mind,
in terms of United States versus other countries and whether we
are doing enough and may fall behind and not maybe get
researchers to come here.
Dr. Daley. I think one of the real issues is the supply and
demand. The real question is, how many--we have a huge number
of very, very gifted scientists here in the United States, many
of whom I think have been scared off from the embryonic stem
cell field because of the political concerns and the lack of
funding. In other parts of the world, and I think about
Singapore and China, they have specifically invested in this
area because of the vacuum left by the Federal policy in the
United States. I have heard that directly from representatives
of the Economic Development Board of Singapore. They want to
know what we are not able to do because they want to invest in
that, because that gives them a competitive advantage. So I
think, you know, it is always hard to say what might have been
but I can tell you that had we had a more expansive Federal
policy, the kinds of breakthroughs we are seeing today might
have happened years ago. We might have been even further along.
I think the United States--I am still very, very bullish on
what the United States can do and contribute in stem cells and
I hope that the Federal policy will get behind the scientists
because we enjoy the greatest community of scientists in the
world.
Mr. Pallone. Thank you. Thank you to all of you.
Mr. Deal.
Mr. Deal. Thank you.
I thank all of you for being here today. You certainly have
some varied points of view here. As I listened to all of you,
though, I think I detected at least three examples of
successful clinical applications of adult stem cells, I think
Dr. Patel, Mr. Rice, and Dr. Fraser specifically. Maybe I
missed it, but did any of you suggest that there are successful
clinical applications of embryonic stem cells?
Dr. Daley. This is really an interesting question that
keeps coming up. There is no way that a cell which was
discovered only 10 years ago would be able to compete with the
clinical results of hematopoietic stem cells, which were
introduced into therapy in the 1950s. It took 30 years before
the discoverer of bone marrow transplant, E. Donnall Thomas,
was actually recognized with the Nobel Prize for that. I think
it is really unfair to hold embryonic stem cells to the same
kind of standard. They are new. This is a new technology.
Mr. Deal. I wasn't questioning whether it was fair or not.
I was questioning about what the facts are.
Dr. Daley. Well, the facts are that this is a fresh, new
technology which is finding its way into the laboratories and
will ultimately find its way into having a clinical impact. I
think we have a responsibility to educate the public that
scientific cures don't happen overnight, that this is a very
long and tedious path and it involves basic investments. The
NIH has been tremendous for supporting basic research and we
enjoy the tremendous benefits in our healthcare system, we
enjoy the tremendous benefits in our biotechnology industry,
but we are at risk of not taking advantage of the tremendous
possibility of embryonic research because of a Federal policy
which has limited investments in that very exciting area.
Mr. Deal. Well, I think the answer was, I did not hear any,
and the second question then, Dr. Daley, since you have taken
it on in the context of----
Dr. Daley. I think you need to ask that question in another
10 years.
Mr. Deal. All right. Well, that is my next question----
Dr. Daley. And then we will see how things stand.
Mr. Deal. --if you will let me ask it. How soon do you
expect clinical applications from embryonic stem cell research
to be used?
Dr. Daley. So I want to say that it is very important that
we educate the public about the nature of medical discovery.
After I leave this hearing, I am flying to Chicago where the
International Society for Stem Cell Research is convening its
clinical translation task force. We have a group of 30
scientists, and bioethicists from all over the world who are
tackling the question of what is a prudent approach to
translate this new science of stem cells into real clinical
therapies. We already know there are companies that are
attempting to commercialize both adult and embryonic stem
cells. You heard reference to the Geron Corporation, which may
in fact introduce the first clinical trial of an embryonic stem
cell-derived cell to treat spinal cord injury. There is a big
difference and a delay between the first introduction of a
treatment into human patients and realizing real clinical
benefit. If you look back at the history of medical technology,
whether we are thinking about therapeutic antibodies or drugs,
there is often a 20-year time lag. I would anticipate that we
have to take another 10 years, so 20 years after the original
introduction of embryonic stem cells, before we start to see
therapies based on stem cells.
Now, in the much nearer term, we are already benefiting
from 25 years of understanding mouse embryonic stem cells. In
1981, mouse embryonic stem cells were first isolated, won the
Nobel Prize for Martin Evans this past year. There have been
countless numbers of mouse models of human disease that have
been generated, funded by the NIH which have revolutionized our
understanding of cardiovascular disease, neurodegenerative
disease, cancer and the like. So in indirect ways, that
investment in basic research is translating into cures.
Mr. Deal. Well, you are not suggesting, though, that we
should not continue research and investment in adult stem cell
research, are you?
Dr. Daley. I think my testimony clearly stipulates that we
need a vigorous and increased support for all forms of stem
cell research. We are having a very difficult time as
scientists right now through the NIH because the budget has
been kept flat. We had a doubling, and it created a tremendous
infusion of talent, great, high-caliber talent into American
science, and now we are seeing a receding because we can't
support all that momentum.
Mr. Deal. We are very proud of our side for being able to
double that budget on our side, so join with us to get some
pressure on these folks to make sure we keep that 5 percent as
a minimum increase every year.
I think my time is probably expired. Thank you, Mr.
Chairman.
Mr. Pallone. Thank you, Mr. Deal.
I recognize the gentlewoman from Colorado, Ms. DeGette.
Ms. DeGette. Thank you very much, Mr. Chairman. Before I
question, I have two unanimous-consent requests. The first one
is that I be allowed to submit testimony of Dr. Debra Mathews
on the ethics of stem cell research. We have cleared this with
the Minority. For the record, we tried to get Dr. Mathews to
come but because of the short notice, we were unable to. And my
second UC request is to submit Dr. Zerhouni's chart that he
referred to in his testimony for the record.
Mr. Pallone. Let me also mention that I have the copies of
the documents that the gentlewoman from North Carolina gave me.
I am no less knowledgeable on the subject after having glanced
at them than I was before but I would also unanimous consent
that they be submitted as part of the record.
Without objection, all four documents will be submitted. So
ordered.
[This information was unavailable at time of printing.]
Ms. DeGette. Thank you very much, Mr. Chairman.
I only have 5 minutes so I am going to ask the panel if
they would mind giving short answers to my questions if
possible because I have a lot of ground to cover.
I wanted to ask you first Dr. Gearhart, as a researcher,
has the research community found that the restrictions on
Federal funding for embryonic stem cell research that were
enacted in 2001 affected research in the area of embryonic stem
cell research?
Mr. Gearhart. Well, it has.
Ms. DeGette. And briefly, how has that----
Mr. Gearhart. In several ways. One Dr. Daley referred to is
students and post-docs and fellows coming to the lab and
looking at long-term support in this area, very problematic in
this country as we look back in 2001, we didn't know where it
was going, and this was before there was a big----
Ms. DeGette. And so it is limiting the number of people who
want to go into that type of research?
Mr. Gearhart. Well, yes. They have to be practical and look
to see what kind of a future there is.
Ms. DeGette. And do you think that the research itself
would benefit if a greater number of embryonic stem cell lines
were allowed under the Federal----
Mr. Gearhart. Oh, absolutely. I think we have arguments for
utility, performance and safety that trump all of that, and
there are many experiments that we don't want to do with some
of the existing lines. It is not worth the effort.
Ms. DeGette. And someone, I think Ms. Capps, asked Dr.
Zerhouni about the Federal funding for facilities and how
people were having to build parallel labs. Are you finding that
also happening in the research community where private
universities or other groups are feeling like they can't use
anything that has had Federal funding involved with it?
Mr. Gearhart. Well, we do. It varies from institution to
institution. At Hopkins, the decision was made, not by us, that
we could use the same facility but the bookkeeping from where
someone's funding is coming from as either salary or supplies,
we have to mark all of this as to which one is federally
approved, which is not federally approved. It becomes a
bookkeeping and practical nightmare under those conditions.
Ms. DeGette. And at other facilities, they have determined
that if there is any Federal funding in those labs----
Mr. Gearhart. That is correct. They will build a separate
lab.
Ms. DeGette. They are building separate labs.
Mr. Gearhart. Absolutely.
Dr. Bertino. In New Jersey, we have built separate labs
because it is too much of a hassle.
Ms. DeGette. And also in Colorado, by the way.
Dr. Daley, I wanted to ask you, you are the president of
the International Society for Stem Cell Research, and someone
asked you briefly about the international implications, but I
have learned through talking to researchers at the
international level that the U.S. restrictions are also hurting
the international research because of collaboration issues. If
a scientist in Singapore, for example, wants to collaborate
with a U.S. scientist, the restrictions are having an impact on
that. Is that correct?
Dr. Daley. Oh, absolutely.
Ms. DeGette. Could you explain briefly why that is so?
Dr. Daley. Yes, well, it is not only international, it is
interstate concerns. I mean, I have a colleague, a very
respected colleague, Sean Morrison in Michigan, who can't do
the kinds of research that I do in my own lab because it is
restricted in Michigan so that limits the kinds of
collaborations that we can have. Science is increasingly a
global activity. We are about to have our international
meeting, we will have 2,500 scientists from all over the world,
and we have this patchwork quilt of regulations. It is not good
for science.
Ms. DeGette. And would it also be fair to say that it would
be helpful to have a national ethics oversight system for the
research that is being done here, much like----
Dr. Daley. No doubt.
Ms. DeGette. --in the United Kingdom and in other
countries?
Dr. Daley. No doubt.
Ms. DeGette. Now, it sounds like it was actually your skin
cells that were used in this iPS experiment. Is that right?
Dr. Daley. Well, I tried, but my skin cells didn't yield an
iPS line.
Ms. DeGette. Well, the iPS research, I am assuming that
hasn't led to any kind of clinical cures for anything, even
though it has been touted by some as the alternative to
embryonic stem cell research, has it?
Dr. Daley. No, it hasn't.
Ms. DeGette. And I would also expect that since that
research is 10 years behind human embryonic stem cell research
and 20 or 30 years behind mouse embryonic stem cell research,
the clinical applications for iPS are going to be that much
farther out down the road from now, correct?
Dr. Daley. Well, we are hopeful that we can piggyback on
some of the embryonic stem cell research and accelerate that.
Ms. DeGette. If we expand embryonic stem cell research
lines that Federal funding can be used for, would you expect
that that would also help your iPS research then?
Dr. Daley. Yes.
Ms. DeGette. Why is that?
Dr. Daley. Well, I mean, we still don't know enough about
these iPS cells to even know and predict with confidence we
will ever be able to use them in patients. I am confident that
they will be valuable for modeling disease. We are already
doing that in our own laboratory, and I think it is a very
important point that so much of the debate has focused on
whether or not stem cells will directly cure disease, but I
want to reiterate the value of basic research and the fact that
these stem cells are really changing the paradigm of that
research.
Ms. DeGette. And this is exactly what Dr. Zerhouni was
talking about, isn't it?
Dr. Daley. Actually, what Dr. Zerhouni was arguing, and it
is the first time I have really heard it argued so
compellingly--in fact, I would love to have him come and give
that speech to my stem cell research laboratory--is that all of
the questions asked by scientists about stem cells are really
the same. It is about programming of cell fates, and so we
never have these kinds of disagreements at our scientific
meetings about embryonic versus adult. This is a debate that
happens in Congress.
Ms. DeGette. Thank you very much.
Mr. Pallone. Thank you.
Mr. Pitts.
Mr. Pitts. Thank you, Mr. Chairman.
Dr. Gearhart, do you support gestating human children to
later fetal stages to harvest issues to treat disease?
Mr. Gearhart. Absolutely not.
Mr. Pitts. Does anyone in the panel support that? OK.
Dr. Daley, do you think that the Federal government should
fund somatic cell nuclear transfer or cloning for research?
Dr. Daley. I do support it because I think it has enormous
medical implications. The study of somatic cell nuclear
transfer research, I do support that, yes.
Mr. Pitts. And so you think that should be legal?
Dr. Daley. It is legal.
Mr. Pitts. And you think it should remain legal. Do you
think that the Federal government should fund research in which
animal eggs and human cells are mixed to create embryos that
are part animal, part human?
Dr. Daley. I believe that this range of experiments that
you are defining are best left to the experts in the scientific
community to set the priorities. I do believe that there are
scientific arguments to support that area of research as has
been supported by the United Kingdom. So, yes, I do believe
that that is a potentially valuable area of research and it
should be under the purview of the scientific community.
Mr. Pitts. And that should be legal?
Dr. Daley. It is legal.
Mr. Pitts. It is legal and should remain legal?
Dr. Daley. Yes.
Mr. Pitts. Dr. Patel, how many patients have you treated
for heart disease with adult stem cells?
Dr. Patel. In our team, we have treated over 100 here in
the United States but we have had over 30 groups from around
the world come and train and try the different techniques. The
key is, we do it in a very regulated and ultimately our goal is
to have it as safe as possible so now that some of the trials
have evolved to phase III trials, both in Germany and in
Brazil, where they are all federally-funded trials since they
are mostly bone marrow-derived treatments. The problem is that
even though we treat patients as still experimental, there are
people who try to do these as approved or unregulated
therapies, and that is our biggest concern irrespective of the
cell, and we do worry that when you take the more multipotent
cells, that we are going to see severe adverse events which
could potentially shut down our entire field just due to the
fact that patients are going to these countries and having
these unregulated therapies. So we are actually very happy that
the NIH has created these centers for at least cardiovascular
disease where we could offer these type of treatments in
controlled trials here in the United States today.
Mr. Pitts. Do you agree that adult stem cells show promise
only for blood diseases or autoimmune diseases and that they
don't show as much promise as embryonic stem cells for things
like Parkinson's or spinal cord injury or macular degeneration
or diabetes?
Dr. Patel. Well, my expertise is cardiovascular disease.
Mr. Pitts. What about the heart?
Dr. Patel. So in the heart, adult stem cells show great
promise and there are many different types that we need to
continue to work that actually can differentiate in the lab to
new heart muscle and blood vessels. The key is safely
translating those therapies into patients. So in terms of other
diseases, there are clinical trials for type 2 diabetes, also
for Parkinson's and also for spinal cord disorders but
currently they are not ongoing in the United States. These are
all trials that are either in Europe or in South America that
are funded by their governments, and hopefully as some of these
posters and presentations are presented at the ISSCR and the
ISCT, that as the academic community goes through these trials,
we can hopefully bring these back to the United States and see
if we can replicate them, just as the iPS cells were originally
created in Japan and Dr. Daley's group along with others were
able to reproduce that so that will advance the field and also
keep it a very safe therapy.
Mr. Pitts. Dr. Daley, you support human cloning. You
stated, I think, yesterday that human cloning is necessary to
do iPS research. Since there are no human cloned embryonic stem
cell lines, yet there are 124 human iPS lines including at
least 15 human iPS cell lines that you have developed according
to your publication online in Nature at the end of 2007, how do
you justify that statement?
Dr. Daley. Mr. Pitts, I am very pleased that you are
reading my paper in Nature.
Mr. Pitts. My staff did.
Dr. Daley. Oh, OK. Well, if you read that paper or your
staff and some of my other publications, I think you would see
the justification, and that I have written that there is a
strong distinction between your use of cloning and the
legitimate medical applications of copying cells, copying cells
so that we can learn about this reprogramming process that Dr.
Zerhouni described. It is a fascinating and important
fundamental question in biology. We still don't know whether
the reprogramming we are inducing with these candidate genes is
the same process of the reprogramming that happens with nuclear
transfer. We think this is a frontier of medicine with enormous
potential, and I think that we should allow the scientists to
explore and use all of the tools available to them subject to
very rigorous and very scrupulous scientific and ethical
review, and that has been done for my own experiments through
at least four different institutional review committees.
Ms. DeGette [presiding]. The gentleman's time has expired.
The gentlelady from California.
Ms. Capps. Thank you, Madam Chairwoman.
I want to continue this line of thought. We need to have
several more things on this, Madam Chairwoman. This is a very
important issue. To follow along the previous questioner, Dr.
Daley, we are confused often here and I think the media is too,
which influences us a lot. Do you support reproductive cloning?
Dr. Daley. No, I don't.
Ms. Capps. And maybe you want to take a minute, this is a
big issue. When you talk about human cloning, people get really
scared and react with sort of blanket prohibitions. Could you
just expand a little bit on that so we understand clearly? And
then this harkens back to me, this need for ethical oversight,
even with respect to how other countries are dealing with it
and how they are filling in the vacuum, as you have said,
because we have created one.
Dr. Daley. There has been an enormous amount of public
debate and some scientific discussion about the value versus
the risks to society of using nuclear transfer. Nuclear
transfer is the method that has been used in animal biology to
perform reproductive cloning for many different mammalian
types--mice and dogs--and there are legitimate scientific
reasons to do this and there are issues of animal husbandry
which have supported this. There is also one methodology for
using nuclear transfer to establish stem cell lines. That has
been enormously productive in mice. My own laboratory, together
with Rudy Jaenisch, has published using nuclear transfer to
treat a genetic disease in a mouse. Recently these nuclear
transfer lines have been produced from primates. It has not
been done from humans. And I think that much of the enthusiasm
is now going to be diverted to producing these stem cell lines
using the iPS methodology. So my own laboratory is performing
an enormous amount of experiments on the iPS methodology, but
because of the scientific value, the intrinsic scientific value
of the nuclear reprogramming, we continue to pursue that. But
it is very important to draw the distinction between copying
cells and copying babies. No one in the scientific community--
and I chaired last year the International Society's guidelines
on human stem cell research, and there was a clear prohibition
against productive cloning. So no legitimate scientists think
that this is an area of great interest, but many scientists
feel that understanding nuclear transfer so that we can
reprogram individual cells is highly, highly valuable. And so
you will see, I think, broad consensus for studying the various
ways of reprogramming because no one knows yet which way is
ultimately going to be the most valuable.
Ms. Capps. And doesn't this also speak to a federally-
established set of guidelines that could direct the way this
kind of research is done so that we can be proud and confident
that our scientists will clearly be able to distinguish between
the various levels of research to safeguard the threats that
many people are concerned about?
Dr. Daley. The NIH has enormous respect from all of the
scientists in this country and it has always played a critical
role in scientific peer review and scientific oversight, and I
think it has been unfortunate that it has not been able to play
its routine leadership role in this critical area of this
exploding biology.
Ms. Capps. I want to try to get one other question in, if I
can. With the description of adult stem cells coming on to the
scene and they are being lauded as the end-all, then there are
many, even among our colleagues, who say that well, we don't
need embryonic stem cell research then, and I know you have
been around this, but clearly for the record. Also in terms of
the long-term effects of it, do we really know--I think Dr.
Patel has alluded to this. It is very new technology that we
really don't know the end results. Maybe you would use the
remaining time to distinguish there.
Dr. Daley. It is just--it is far too premature to imagine
how we are going to use embryonic, neonatal, adult in the many
different indications. I am confident that we are going to find
very, very valuable applications for adult stem cells and that
is why we need to continue to work in those areas, but why
close any doors?
Ms. Capps. Thank you very much.
Ms. DeGette. The gentleman from Texas, Mr. Hall.
Mr. Hall. I am sorry that I didn't get to hear all the
testimony, and I am more sorry than that that I don't really
know how to ask what I want to know. I have an illness in my
family for which there is no cure, and it was illness that was
treated for some time for Parkinson's 18 months to 2 years, and
on my way back up here one time, I asked my wife to give me her
file, and I like to read everything I can read about
Parkinson's, and by the time I got up here, I wanted to go
directly to a hospital or doctor's office because I had almost
every symptom, but I am 85 years old. I am the oldest guy in
the Congress. I am the dean of the United States Congress, and
people think that is bad but it is not nearly as bad as
somebody saying don't he look natural.
So I ask you this question, and it is a very important
question to me and I don't know how to ask it properly, but I
think Mr. Rice went overseas to have his treatment and it has
been suggested that we go to India, that that was where the
best available treatment was. I don't think we could stand
that. Another to Mexico. I am not inclined to do that; another
to Seattle, that there were some treatments there that was
available. And as most acknowledge, it is not paid by
insurance, and I have had price estimates all the way from
$25,000 to $40,000 to $60,000, and none of those are too great
if I thought it would help her for 15 minutes. The decision was
made that she didn't have Parkinson's because the week I read
all that, I went back and said we will go to Mayo and know what
we have, and we went to Mayo, stayed 4 days, didn't want to
know if she needed an appendectomy or ingrown toenail or
anything else. The question was, did she have Parkinson's, and
the answer after 4\1/2\ days was absolutely not. Three weeks
later, a letter back saying that, however, she could have peri-
Parkinson's.
Now, that would be distressing to some but it was hope to
me because I understand stem cells one day might eradicate
Parkinson's. I have heard that said and that may be an
overstatement, but what are the facts with the effect of stem
cells on Parkinson's? Who should I ask that?
Mr. Gearhart. We have done some work on this. The stem cell
therapies for Parkinson's actually began by using portions of
fetal brains that were obtained through abortion in northern
Europe. This was a standard measure of care. Patients receiving
these cells did improve over a period of time and then they
lost that improvement and came back to what they were before.
These cells really weren't stem cells. These were fully formed
dopaminergic neurons, the cells that are lost here, and they
just don't hook up appropriately when they are fully formed.
There was a clinical trial in the early 1990s here in Denver
that reported the same thing pretty much. The newer
technologies that are being worked on in the laboratory, and
this is all through now animal modeling of Parkinson's disease,
in which we can grow in great abundance and derive and grow
dopaminergic neurons from embryonic stem cells. It is one of
the most robust sources of these cells. These cells have been
introduced into various animal models from rats to mice to
monkeys in which we see very much the same thing. There was a
very interesting series of experiments, summary of experiments
published in Nature recently in which the evidence showed that
these cells can go in, they can integrate, they can function
for a long period of time.
Now, this brings up another issue. Some of these cells that
were grafted in are beginning to show the cellular basis of
Parkinson's disease. We know that there is a certain morphology
associated and subcellular components that indicate Parkinson's
disease, something we have not mentioned here. We have
mentioned only that we are growing cells to replace those that
are lost. We have said very little about the companion
compartment of this that is so critical. We have got to learn
more about the pathogenesis of disease and how to shut it down.
We mentioned autoimmune for many of the diseases that are at
the basis of this. If we don't learn what that is about,
putting new cells in isn't necessarily going to help you.
So what we are seeing, and a short answer here, is that
there is an improvement in patients, well, at least in animals
and in the patients that had the fetal tissue grafts, but it is
not of long standing.
Mr. Hall. Let me ask you this, and I note that some asked
whether or not we were aware that the leading experts on
embryonic stem cell research now says treatments from that
source may be one or two decades or more away. Is that what you
are saying?
Mr. Gearhart. Yes. At the moment, we are going through
proof of concept experiments. These are laboratory-based
animals.
Mr. Hall. I am getting close to my 5 minutes.
Mr. Gearhart. Right. So----
Ms. DeGette. You are over 5 minutes, so if Dr. Gearhart
could----
Mr. Hall. May I ask one more question?
Ms. DeGette. Sure.
Mr. Hall. If we do avail ourselves of this thrust for stem
cells, and it has been told to me so simple that you put two
stem cells in, one finds and destroys and the other takes it
place, well, I am willing to accept that but I know it is much
more than that. But is there any danger if the stem cells do
not help?
Mr. Gearhart. Oh, absolutely.
Mr. Hall. That they will do damage?
Mr. Gearhart. Yes, absolutely. There is----
Mr. Hall. Briefly tell me yes or no.
Mr. Gearhart. Yes. I would be happy to give you lots of
data on that.
Mr. Hall. And I will take that up with the folks that I am
talking to. Thank you for that.
Ms. DeGette. Thank you very much. I really want to thank
this panel for coming on very short notice. It was an excellent
panel, and every single witness added to our knowledge. As I
mentioned at the beginning, this is the first hearing that we
have had in the Energy and Commerce Committee ever on all of
these cell therapies, so it has been very useful and I know on
behalf of Mr. Pallone, I want to thank all of you for the
Committee. This concludes all questioning.
In conclusion, I want to remind the members that you may
submit additional questions for the record to be answered by
the relevant witnesses. The questions should be submitted to
the committee clerk within 10 days, and the clerk will notify
the offices of the procedures.
Without objection, this meeting is adjourned.
[Whereupon, at 3:15 p.m., the subcommittee was adjourned.]
[Material submitted for inclusion in the record follows:]
Statement of Hon. Anna G. Eshoo
Thank you, Chairman Pallone, for convening another hearing
on the important topic of stem cell research. Congress has
clearly demonstrated our commitment to expanding stem cell
research in our country. We've held hearings, we've debated,
and both chambers passed legislation. Unfortunately, the
Administration does not share our view.
The very first veto of President Bush's was stem cell
research and the expansion of Federal funding for it. We cannot
overlook the necessity and potential of this research and the
new treatments and discoveries that will invariably come from
this exciting area of science, saving lives, and eradicating
the pain and suffering of so many. I have cosponsored
legislation to provide federal funds for stem cell research and
continue to be a strong advocate on this issue.
We cannot continue to allow the United States to fall
behind our international counterparts because of the current
restrictions. Our scientists are hamstrung, able to only use
federal funds on human stem cell lines derived prior to the
President's ban in August 2001. As those cells lines age, they
undergo biological changes that reduce their scientific
potential. To be the world's leader, researchers in our country
should not be reduced to using old stem cell lines that are of
limited value. Our constituents who suffer from diabetes,
spinal cord injuries, Parkinson's, and many other diseases are
relying on us to give American researchers the tools and
resources they need to develop new treatments. Stem cell
research has far too much potential for us to restrict federal
funding which limits the hopes and dreams of the American
people.
The result of our Federal policy on stem cells today is
sending our best scientists to research facilities overseas.
Those who are still in the U.S. are watching from the sidelines
and it is only a matter of time when the breakthroughs will
occur.
Stem cells and the treatments and discoveries locked within
them represent the future of health and medicine. I'm pleased
that we are once again bringing attention to the issue of stem
cell research. I thank the witnesses for being here today and I
look forward to their testimony. My hope is that we can reverse
the current federal policy and lift up the million of Americans
who will benefit from an enlightened policy.
----------
Statement of Hon. Barbara Cubin
Thank you Mr. Chairman.
Today's hearing gives this committee a valuable opportunity
to examine recent breaththroughs in stem cell science. Stem
cells are literally building blocks of human life. They hold
the promise of curing or treating a host of serious diseases,
from Parkinson's and Alzheimer's to heart disease and diabetes.
There are several accounts of stem cell therapies that are
working right now to treat disease. Doug Rice of Washington
State, who will be sitting on our second panel today, will
share the improvements he has experienced with his heart
condition using stem cells isolated in his own bloodstream.
Blood stem cells have also been used by researchers from
Northwestern University and Brazil to successfully treat type 1
diabetes. Thirteen of the fifteen patients involved in the
trial became insulin-free according to the Journal of American
Medical Association.
As a strong supporter of Alzheimer's research, I am
particularly encouraged by research at the University of
California, Irvine, in which scientists are using stem cells to
restore the memory of mice. The research could lead to
breakthroughs not just for Alzheimer's, but also stroke and
traumatic brain injury.
Perhaps one of the more exciting stem cell advances is the
development of induced pluripotent stem cells. In this
astonishing process, genes are added to ordinary skin cells in
order to create stems cells with potentially therapeutic
applications. While the science and its application to humans
is still developing, the cells are believed to be pluripotent,
that is, capable of differentiating into any cell type.
All of these treatments and potential treatments have one
vital characteristic in common. Their stem cells were derived
in ways that did not involve the destruction of a human embryo.
The induced pluripotent cells in particular hold the promise to
be just as versatile as embryonic stem cells, both in treatment
and for research purposes.
I cannot support Federal funding for embryonic stem cell
research that harms or destroys any human life. As we work
tirelessly to improve the health of the ill, this is still no
justification for taking another human life. Moreover, no
embryonic stem cell has been used to treat disease or injury,
while adult stem cells are being used clinically at this very
moment.
This hearing is entitled, ``Stem Cell Science: The
Foundation for Future Cures.'' With induced pluripotent stem
cells, we have an ethical foundation for future treatments.
With other adult stem cells, the future is now. The Federal
Government owes it to millions of disease suffering Americans
to support the development of these therapies.
With that, I welcome our panelists. Thank you Mr. Chairman.
I reserve the balance of my time.
----------
Statement of Hon. Lois Capps
Thank you, Chairman Pallone, for holding this hearing.
Even though our current Administration has prohibited
federally funded embryonic stem cell research, America's
biomedical research community has continued on with this
important work.
Our Nation's leading scientists know the facts.
They know that both adult and embryonic stem cell research
hold the potential to cure some of humanity's most devastating
diseases:
Cancer, Diabetes, Parkinson's, Alzheimer's and I'm sure,
many more.
I'm so pleased to have some of those leading scientists
with us here today.
To share with us the truth about stem cell research.
About the nature of embryonic stem cell research and about
the promise of adult stem cell research.
Adult stem cell research is crucial.
We need it.
But we need embryonic stem cell research, too, because one
is not a replacement for the other.
They are two pieces of a large puzzle.
I am proud that my own state of California has been a
leader in this field and filled in some gaps where the federal
government has been absent.
But state and private funding are only pieces of the
puzzle.
Federal dollars, predominantly through the NIH, are the
primary source of funding for basic research--
The kind of research that identifies the fundamentals for
future research that will eventually lead to cures.
It is quite frankly embarrassing to have taken this big
step backward over the past few years as the rest of the world
has soared ahead.
But again, I'm so thankful that we have scientists, health
care professionals, patients and other advocates who have found
ways to keep research going so that we won't waste any more
time in our quest for those cures.
Finally, I'd like to thank my colleague, Diana DeGette, for
her tireless leadership on this issue.
I look forward to hearing from today's witnesses.
I yield back.
----------
Statement of Hon. Edolphus Towns
Let me thank you Chairman Pallone and Ranking Member Deal
for holding this timely hearing on ``Stem Cell Science: The
Foundation for Future Cures.''
Embryonic stem cells may hold the key to curing a host of
debilitating conditions that affect millions of people around
the globe. These diseases include Parkinson's disease,
diabetes, traumatic spinal cord injury, Purkinje cell
degeneration, heart disease, cancer, multiple sclerosis, vision
and hearing loss, and others.
Given advancements in research, it is appropriate that we
convene at this time to assess current developments in stem
cell research, discuss the use of adult stem cells versus
embryonic stem cells, and explore a new method known as
``somatic cell nuclear transfer''.
In 2007, I co-sponsored and voted in favor of
Representative DeGette's bill to authorize embryonic stem cell
research, and am proud of it passage in Congress. It was a dark
day for all people who suffer from diseases that may be cured
by this research when the President vetoed the bill, H.R. 3.
When the administration imposed additional restrictions on
embryonic stem cell research with its 2001 embryonic stem cell
policy and 2007 executive order, it crippled U.S. research
efforts in these areas. Thankfully, Japan and Europe continued
with their embryonic stem cell research and moved the world
forward in the quest for cures. It is time that the U.S. resume
its place as a preeminent contributor to this critical effort.
To this end, I welcome efforts to create a record of the
work of NIH, FDA, the private sector, and other countries in
the area of stem cell research. This database is critical to
our coordinated efforts to advance stem cell research as
efficiently and effectively as possible.
I wholeheartedly believe that such research can be
conducted in an ethical manner. As a God-fearing man of faith,
I humbly appreciate it is God who is responsible for both
diseases and cures. Cures can only come about upon God's
command. I believe he wants us to move forward on research and
that we should let him shepherd us on this quest, and bring
relief to those who suffer from terrible diseases
unnecessarily.
Thank you Mr. Chairman. I respectfully yield back the
remainder of my time.
----------
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Douglas T. Rice, Responses to Questions from Hon. Joseph R. Pitts
June 19, 2008
U.S. House of Representatives
316 Ford House Office Building
Washington, D.C. 20515
Congressman John D. Dingell
Ref: The Honorable Joseph R. Pitts questions regarding
testimony on May 8th, 2008
Question: Could you please tell us about your experience
and difficulties in obtaining adult stem cell treatment for
your heart condition? What were the costs involved? Was
insurance coverage available? Was FDA approval available at
that time?
Answer: As I testified at the hearing, I was given 3-4
months to live without a Heart Transplant, since I was
diabetic, I was not eligible and did not want to have the
Mechanical Heart transplant as I have seen the results and have
never seen anyone get better. After verifying that no solution
was available in the U.S., my ex-wife went on line looking for
new technology and found that in Thailand, a company named
Theravitae was doing Adult Stem Cell transplants that were
successful. After meeting with my cardiologist, it was decided
I had no other chance to live and the risk versus reward was
worthwhile.
The costs were $40,000 plus airfare and you had to take
someone with you, total cost was approximately $50,000. I had
to borrow the money and move quickly to get there in time.
My insurance including the V.A. would not cover any of it,
though there was nothing available in the U.S., luckily I had
friends and family that wanted me around or I would be dead by
now.
FDA did not allow the use of the Adult Stem Cell in this
type of treatment though they allowed the use of the ASC in
Cancer and other illnesses. They would let you draw the blood
to send to Israel but not the cath procedure to insert the stem
cells. There are now successful trials being done in the U.S.
using the Adult Stem Cells on the heart with tremendous success
stories. Also, there is a new clinic using the same procedure
as Theravitae in the Dominican Republic by an American doctor
and has been very successful. He has recently saved the hands
and feet of a young athlete that had lost all circulation there
and after ASC treatment has saved them.
Question: To your knowledge, how many other patients have
been treated for heart disease using adult stem cells by the
doctors who treated you?
Answer: I believe that in Thailand, over 200 patients have
been successfully treated for end stage heart disease and other
heart related diseases. Me being one of them, also numerous
other ones I have met and speak with. I have been in contact
with other countries and most are using the Adult Stem Cell
treatment to save many lives.
To cover some very valid issues about the Adult Stem Cell
treatments that are being used in the U.S.
Almost a million people die every year in America from
Heart Disease, there has been a valid treatment for years using
ASC and yet Billions have been spent on researching the
Embryonic Stem Cells ( with no success ) when those funds could
have used for treatment rather than just research. Over 700,000
Cancer patients and other illnesses have been treated since
1959 and every day new success's are being tried and used.
Why, when a single celebrity dies does the news media cover
it for weeks and months, yet when almost a million Americans
die of a treatable disease, you never hear of them? I have
tried to get on national media to tell the facts with no
success, yet Embryonic are discussed all the time and they
don't work at this time if ever. Why does Congress have
hearings about it and the only thing really discussed is how
well ESC is progressing, yet not one human treated! What will
it take to convince anyone that with more treatment using
existing Adult Stem Cells, Americans could live with treatment?
I was privileged to be there to introduce The Patients
First Bill last year and yet it still hasn't passed. What is
the real reason that a true success in medicine is set aside
for something that doesn't work and even scientist say may
never?
I travel as much as I can to educate Americans on the Adult
Stem Cell and the difference with "Fact and Fiction" regarding
Embryonic Stem Cells. And, believe me there is a lot of fiction
going on about ESC.
Though I try, I am not financially strong enough to really
make a difference, but I try as best as I can. But how can we
let millions of people die every year when there is a possible
treatment that works now? How do we face the families of the
ones that could have been treated knowing that we are not doing
all we can to help. How can you, as their representatives not
stand up for them and fight for their right to live a better
life. How do you sit in meetings and basically just talk about
how great a job the funds you have allotted for research with
ESC has not saved one life while the funds you did not fund for
ASC could have saved millions? As an American, and one that was
allowed to live, but had to go to another country using
American technology to do so, I question the FDA's line of
thinking and to be honest our government.
I hope and pray that this will help move the Adult Stem
Cell Story into the news and the facts will speak for
themselves.
Respectfully,
Douglas T. Rice
Adult Stem Cell Recipient
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Weyman Johnson, Jr., Responses to Questions from Hon. Joseph R. Pitts
June 23, 2008
Honorable Joseph Pitts
Committee on Energy and Commerce
2125 Rayburn House Office Building
Washington, D.C. 20515
Dear Congressman Pitts,
Thank you for the opportunity to appear before the
Subcommittee on Health on Thursday, May 8, 2008 at the hearing
entitled "Stem Cell Science: The Foundation for Future Cures."
The field of stem cell research brings hope to millions of
Americans who are affected by chronic diseases including more
than 400,000 who are living with multiple sclerosis (MS). The
National Multiple Sclerosis Society believes all promising
avenues of research must be explored and remains committed to
ensuring all types of stem cell research is pursued under
strict ethical guidelines and in accordance with the law.
Enclosed in this correspondence is my response to the
questions Chairman John Dingell sent to me on your behalf. I am
happy to provide further detail if necessary.
The National MS Society stands by to serve as a resource to
you and any Member of the Committee.
Sincerely,
Weyman Johnson, Jr., J.D.
Chairman of the Board
Enclosure
Cc: The Honorable John Dingell, Chairman
Committee on Energy and Commerce
The Honorable Joe Barton, Ranking Members
Committee on Energy and Commerce
The Honorable Frank Pallone, Chairman
Subcommittee on Health
The Honorable Nathan Deal, Ranking Member
Subcommittee on Health
1) Question: Is the National Multiple Sclerosis Society
spending any research funds on "somatic cell nuclear transfer"
or human embryonic stem cell research? If so, how much is being
spent and what percentage of your research budget is allocated
for these types of research?
Response: From the beginning, the National MS Society has
funded research seeking clues to the cause, treatment and cure
of MS, and to spark research efforts around the world. Although
MS is not hereditary or contagious, it is believed to occur in
genetically susceptible people who are exposed to an infectious
agent, such as a virus or bacterium. These factors combine to
cause the person's immune system to attack myelin insulation on
nerve fibers.
The National MS Society is a driving force of MS research,
and as such, our research efforts support studies in many
different areas of scientific studies from immunology to
genetics to understanding ways to repair the damage to myelin.
The Society is expending nearly $45 million this year alone to
propel MS research forward, including funding over 440 new and
ongoing MS investigations in the U.S. and abroad, across all
areas of research.
Today the most exciting area of research, and one that
holds true promise for those individuals with MS, is in the
area of repair and protection of the nervous system. The
Society is currently not funding any projects using SCNT. Of
the 440 projects which we are currently supporting, 80 (18%)
are focused on repair using both human and animal cells. Of the
440 awards, 7 (1.6%) projects are using human embryonic stem
cells at an annual cost of $1.24 million (2.8% of our overall
annual research budget).
2) Question: Has the National Multiple Sclerosis Society
funded any adult stem cell research? If so, how much is being
spent and what percentage of your research budget is allocated
for these types of research?
Response: Some tissues and organs have little capacity for
self-repair. One such organ is the brain; and nerve cells or
neurons are known to be very restricted in their capacity to
regenerate following damage or disease. The adult brain and
spinal cord appear to have only a limited ability to produce
new neurons. This is one reason why recovery is often limited
when the nervous system is injured.
One of the most exciting frontiers in medicine is the
potential use of stem cells for treating diseases for which
there are no cures. One strategy is by replacing cells using
embryonic cells, and another strategy is using adult cells -
either from a donor or by using the patient's own cells. It is
important that both of these avenues are pursued.
Of the 440 projects which the National MS Society is
currently supporting, 6 (1.4%) projects are using human adult
stem cells at an annual cost of $2.0 million (4.4% of our
overall annual research budget). With regards to the use of
adult stem cells, it is important to clarify the two different
approaches which are being studied: one is to repair the damage
in MS, and the other is to use bone marrow adult stem cells in
transplantation to reconstitute the immune system. To date, it
is the latter research which has shown some promise as a
treatment in some individuals with aggressive MS. MS
investigators are currently studying whether bone marrow
transplantation is an effective treatment in a group of closely
matched people with MS. Since the immune system is misdirected
in MS, the hope is that by transplanting these adult bone
marrow stem cells, one can reconstitute a naive immune system
that will not attack myelin and thereby, will correct itself.
The second use of stem cells is to repair the damage in MS.
We know that the damage is occurring in the central nervous
system, namely, the brain and the spinal cord and the optic
nerves. So we need to figure out a way that repair, actually,
occurs at the site of the injury. We can broadly divide the
research efforts into two categories. One is, can we promote
the cells that are already there, what we call the adult
endogenous progenitor cells, to function more effectively, or
is the challenge going to be do we have to provide the cells
from outside? We know that during an attack of MS, the myelin
is injured and; we also know that there is an element of
repair. But how do we stimulate the repair in the body and what
is the best source of cells to use? If we had ways of directing
the function of these adult stem cells, then these are the
cells that would, actually, be the ones used in the disease
repair.
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