[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

                              ----------                              
                                                                   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

                              ----------                              


                         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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