[Senate Hearing 111-942]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 111-942
 
           THE PROMISE OF HUMAN EMBRYONIC STEM CELL RESEARCH

=======================================================================

                                HEARING

                                before a

                          SUBCOMMITTEE OF THE

            COMMITTEE ON APPROPRIATIONS UNITED STATES SENATE

                     ONE HUNDRED ELEVENTH CONGRESS

                             SECOND SESSION

                               __________

                            SPECIAL HEARING

                   SEPTEMBER 16, 2010--WASHINGTON, DC

                               __________

         Printed for the use of the Committee on Appropriations


       Available via the World Wide Web: http://www.gpo.gov/fdsys

                               __________



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                      COMMITTEE ON APPROPRIATIONS

                   DANIEL K. INOUYE, Hawaii, Chairman
ROBERT C. BYRD, West Virginia        THAD COCHRAN, Mississippi
PATRICK J. LEAHY, Vermont            CHRISTOPHER S. BOND, Missouri
TOM HARKIN, Iowa                     MITCH McCONNELL, Kentucky
BARBARA A. MIKULSKI, Maryland        RICHARD C. SHELBY, Alabama
HERB KOHL, Wisconsin                 JUDD GREGG, New Hampshire
PATTY MURRAY, Washington             ROBERT F. BENNETT, Utah
BYRON L. DORGAN, North Dakota        KAY BAILEY HUTCHISON, Texas
DIANNE FEINSTEIN, California         SAM BROWNBACK, Kansas
RICHARD J. DURBIN, Illinois          LAMAR ALEXANDER, Tennessee
TIM JOHNSON, South Dakota            SUSAN COLLINS, Maine
MARY L. LANDRIEU, Louisiana          GEORGE V. VOINOVICH, Ohio
JACK REED, Rhode Island              LISA MURKOWSKI, Alaska
FRANK R. LAUTENBERG, New Jersey
BEN NELSON, Nebraska
MARK PRYOR, Arkansas
JON TESTER, Montana
ARLEN SPECTER, Pennsylvania

                    Charles J. Houy, Staff Director
                  Bruce Evans, Minority Staff Director
                                 ------                                

 Subcommittee on Departments of Labor, Health and Human Services, and 
                    Education, and Related Agencies

                       TOM HARKIN, Iowa, Chairman
DANIEL K. INOUYE, Hawaii             THAD COCHRAN, Mississippi
HERB KOHL, Wisconsin                 JUDD GREGG, New Hampshire
PATTY MURRAY, Washington             KAY BAILEY HUTCHISON, Texas
MARY L. LANDRIEU, Louisiana          RICHARD C. SHELBY, Alabama
RICHARD J. DURBIN, Illinois          LAMAR ALEXANDER, Tennessee
JACK REED, Rhode Island
MARK PRYOR, Arkansas
ARLEN SPECTER, Pennsylvania
                           Professional Staff

                              Erik Fatemi
                              Mark Laisch
                            Adrienne Hallett
                             Lisa Bernhardt
                            Michael Gentile
                          Alison Perkins-Cohen
                       Bettilou Taylor (Minority)
                      Sara Love Swaney (Minority)
                      Jennifer Castagna (Minority)

                         Administrative Support

                              Teri Curtin


                            C O N T E N T S

                              ----------                              
                                                                   Page

Opening Statement of Senator Tom Harkin..........................     1
Statement of Francis S. Collins, M.D., Ph.D., Director, National 
  Institutes of Health, Department of Health and Human Services..     6
Human Embryonic Stem Cells.......................................     7
Adult Stem Cells.................................................     7
Induced Pluripotent Stem Cells...................................     8
Three Key Uses of Human ES Cells.................................     8
High-Throughput Drug Screening...................................     9
Prepared Statement of Francis S. Collins.........................    11
The Need for Additional Stem Cell Lines..........................    23
Limitations of Adult Stem Cells..................................    24
Importance of Federal Funding....................................    24
Stem Cell Injunction Dictated by Doctrine........................    26
Impact of Judicial Decisions.....................................    26
The Effect of the Preliminary Injunction on NIH Embryonic Stem 
  Cell Research..................................................    27
American Recovery and Reinvestment Act (ARRA) at NIH.............    28
Advances in Embryonic Stem Cell Research.........................    29
Embryonic Stem Cells as a Research Tool..........................    30
The Ethics of Human Embryonic Stem Cell Research.................    31
Prepared Statement of Senator Patty Murray.......................    32
Prepared Statement of Senator Dianne Feinstein...................    33
Statement of George Q. Daley, M.D., Ph.D., Director, Stem Cell 
  Transplantation; Associate Director, Stem Cell Program, 
  Children's Hospital Boston, Boston, Massachusetts..............    34
    Prepared Statement of........................................    37
Statement of Sean J. Morrison, Ph.D., Director, Center for Stem 
  Cell Biology, University of Michigan, Life Science Institute, 
  Ann Arbor, Michigan............................................    40
    Prepared Statement of........................................    43
Statement of Jean Peduzzi Nelson, Ph.D., Associate Professor, 
  Department of Anatomy and Cell Biology, Wayne State University 
  School of Medicine, Detroit, Michigan..........................    45
    Prepared Statement of........................................    48
Statement of Cody Unser, Founder, Cody Unser First Step 
  Foundation, Washington, DC.....................................    56
    Prepared Statement of........................................    58
Autologous Stem Cell Treatment...................................    60
Additional Committee Questions...................................    61
Questions Submitted by Senator Thad Cochran......................    62
Scientific Hurdles...............................................    62
Federal Funding..................................................    63
International Competitiveness....................................    63
Question Submitted by Senator Patty Murray.......................    63
Research Funding.................................................    63
Prepared Statement of the American Association for Cancer 
  Research.......................................................    65
The Court Injunction is a Setback for Scientific Discovery and 
  Cancer Research................................................    65
Human Embryonic Stem Cell Research Holds Much Promise for Cancer 
  Patients.......................................................    65
AACR Supports Sound, Ethical and Responsible Stem Cell Research 
  Poli- 
  cies...........................................................    66
Prepared Statement of the Americans for Cures Foundation.........    67
Prepared Statement of the California Institute for Regenerative 
  Medicine.......................................................    69
National Institutes of Health (NIH) Grants for Human Embryonic 
  Stem Cell Research Cover Critical Work on Which CIRM/California 
  Research is Dependent..........................................    69
Validating the Potential for Therapies Derived From Human 
  Embryonic Stem Cells...........................................    70
Type 1 Diabetes..................................................    70
Stroke...........................................................    70
Macular Degeneration (Age-related Vision Impairment or Blindness)    70
ALS--Lou Gehrig's Disease........................................    70
Alzheimer's Disease..............................................    71
Prepared Statement of the Student Society for Stem Cell Research.    71


           THE PROMISE OF HUMAN EMBRYONIC STEM CELL RESEARCH

                              ----------                              


                      THURSDAY, SEPTEMBER 16, 2010

                           U.S. Senate,    
    Subcommittee on Labor, Health and Human
     Services, and Education, and Related Agencies,
                               Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 10:05 a.m., in room SD-124, Dirksen 
Senate Office Building, Hon. Tom Harkin (chairman) presiding.
    Present: Senators Harkin, Murray, Specter, and Cochran.
    Also present: Senator Wicker.


                opening statement of senator tom harkin


    Senator Harkin. Good morning. The Senate Appropriations 
Subcommittee on Labor, Health and Human Services, and Education 
will now come to order.
    Well, this is the 21st hearing that this subcommittee has 
held on human embryonic stem cells starting back in December 
1998, 1 month after Dr. Jamie Thompson of the University of 
Wisconsin announced that he had isolated them for the first 
time. And I want to note for the record that it was Senator 
Specter who led this subcommittee at that time, who led the 
hearings beginning then and on through the remainder of the 
1990s and into the 2000s, and then when the gavel changed 
hands, I picked up from him and we have kept this effort going 
on. At that time, it was a very bipartisan basis. And so I just 
want to acknowledge the great leadership role that Senator 
Specter has played in this whole effort on embryonic stem cell 
research.
    It is a shame that we have to revisit this issue under the 
circumstances in which we find ourselves today. When President 
Obama lifted the Bush administration's restrictions on stem 
cell research a year and a half ago, most of us thought the 
fight was finally over. At last, we thought, there was a new 
approach to scientific research in this country, one that was 
dictated not by politics or ideology, but by ethical science. 
At last, we thought, our brightest young minds could enter this 
field without worrying that they would go to the lab one day 
and find the doors ordered shut by someone in Washington, DC. 
At last, we thought, we could begin to realize the promise of 
embryonic stem cell research. And we were on track to do that. 
The National Institutes of Health (NIH) instituted new 
guidelines to ensure that this research would be conducted 
ethically and responsibly. The number of stem cell lines 
eligible for federally funded research rose from 21 to its 
current total of 75. And the scientific community has 
responded, applying for and receiving NIH grants that are 
moving this research forward in robust and exciting ways.
    At the same time, of course, NIH continued to fund research 
on adult stem cells and on induced pluripotent cells (iPS) and 
numerous other approaches to regenerative medicine that could 
lead to treatments and cures.
    Embryonic stem cells have very special properties that no 
other cells can match, and that is why they offer so much hope 
to people who are suffering. That is why so many scientists are 
excited to have access to these stem cell lines and to see what 
they can learn from them.
    Then out of the blue came the preliminary injunction from 
District Judge Royce Lamberth. That action, once again, has 
placed a cloud of uncertainty over this entire scientific 
field. Thanks to a temporary stay by the D.C. Circuit Court, 
human embryonic stem cell research is, for the time being 
anyway, progressing just as it was before Judge Lamberth's 
ruling. But how long that will last is anybody's guess.
    Well, I can say this. We have come too far to give up now. 
If we do not win this battle in the courts, we will have to 
take it up in Congress. This research must continue. The 
politicians and activist judges who oppose it need to respect 
the views of the overwhelming majority of the American people 
who want this research to go forward. People across America--
and I am one of them--have too many loved ones and friends who 
have died from ALS, from Parkinson's, from spinal cord 
injuries, and other diseases that might one day respond to 
treatments made possible by embryonic stem cell research.
    I remember Christopher Reeve testifying before this 
subcommittee several years ago. I wish we still had him around 
today. I remember Rob Borsellino, a newspaper man from Iowa who 
had ALS, testified before our subcommittee. I wish we had him 
around still too.
    As long as there is a reasonable chance that this research 
could help ease human suffering and save lives, I believe we 
have a moral responsibility to pursue it.
    The purpose of today's hearing is to examine the promise of 
human embryonic stem cell research. We will look at the 
science. We will not relitigate the ongoing court case. None of 
the witnesses is prepared to discuss the legal arguments for or 
against the injunction. So I ask members of this subcommittee 
to refrain from asking them questions that are not in their 
area of expertise. And I say to our witnesses if you receive 
such questions regarding legality or court decisions, you 
should not feel required to answer them in any way. We want to 
stick to the science, where we are in the science, what is 
happening with all forms of stem cell research, what role 
embryonic stem cell research is playing in that whole area 
today.
    So before we begin, I would like to turn to Senator Cochran 
for any opening remarks he would like to offer.
    Senator Cochran. Mr. Chairman, thank you very much. We 
appreciate your calling this hearing, to give us an opportunity 
to further explore options for the Congress in dealing with the 
difficult choices we have to make in supporting research that 
is so important in finding cures for illnesses.
    I know when we first started looking into this area of stem 
cell research, my brother-in-law was dying of leukemia. Buzzy 
Clayton was one of the finest young men our State had produced, 
at that time, and he just had an outstanding future and was a 
wonderful person in every way. I am sure that is something that 
I will always keep in mind and remember his great loss. And 
there are many others like Buzzy Clayton who might benefit--we 
hope would benefit--from findings that are made through 
additional and more aggressive research on how to combat these 
terrible illnesses.
    I thank my colleague, Roger Wicker, who is here today to be 
our lead-off witness in this hearing. He has been a leader in 
this area for some time, and we commend him for his successes 
in his efforts.
    Senator Harkin. Thank you, Senator Cochran.
    I say to other people on the panel if you want to 
incorporate your statements into your opening questions, that 
would be fine too.
    But before we go to our panels, Senator Roger Wicker of 
Mississippi has asked to make a brief opening statement and we 
certainly welcome our colleague to this panel. Senator Wicker, 
if you have a statement, it will be made a part of the record 
in its entirety, and please proceed as you so desire. But 
welcome.
    Senator Wicker. Thank you very much, Mr. Chairman, and I 
appreciate the opportunity to be back with you. As you know, I 
served on the subcommittee in the House that is the counterpart 
of this subcommittee, and so it is wonderful to be here today. 
If I am doing something wrong on the microphone, maybe I will 
be the guinea pig and it will be ready for the rest of the 
panel.
    I appreciate the opportunity to appear on the subject of 
embryonic stem cell research. As you know, in 1995 I co-
authored an amendment to the Labor, Health and Human Services, 
and Education, and Related Agencies Appropriations Act 
prohibiting the use of taxpayer funds to create human embryos 
for research or support any research in which human embryos are 
harmed, destroyed, or subjected to risks not permitted for 
unborn children. This so-called Dickey-Wicker language has 
remained the law of the land for a decade and a half.
    In my opinion, the body of scientific evidence developed 
since 1995 has served only to strengthen the argument in favor 
of Dickey-Wicker, but the basic premise for the provision has 
not changed. It is this.
    Number one, the destruction or cloning of human embryos for 
research purposes raises profound moral and ethical challenges.
    Number two, the Federal Government should not be involved 
in subsidizing this controversial life-altering research with 
taxpayer dollars.
    Number three, there are limited Federal funds available for 
health-related research.
    Number four, if human embryonic stem cellresearch is to be 
done at all, it should be paid for with nontaxpayer funds.
    The chair mentioned Dr. James Thompson. He was the first to 
isolate human embryonic stem cells and one of the scientists 
who discovered the groundbreaking embryo-free way to produce 
genetically matched stem cells, known as iPS cells. iPS cells 
are adult cells that have been genetically reprogrammed to an 
embryonic stem cell-like state. This discovery has changed the 
debate on embryonic stem cells.
    When discussing the ethics surrounding embryonic stem cell 
research, Dr. Thompson himself said ``If human embryonic stem 
cell research does not make you at least a bit uncomfortable, 
you have not thought about it enough.'' Recent polling proves 
that embryonic stem cell research makes many Americans 
uncomfortable. According to a 2010 Rasmussen poll, 57 percent 
of Americans oppose taxpayer funding of embryonic stem cell 
research. In other words, the majority of Americans support the 
current ban on using taxpayer dollars to fund research in which 
embryos are destroyed.
    The question is, if we can use adult stem cells, reprogram 
them to act like embryonic stem cells, and avoid the ethical 
challenges, then why would we not take that approach?
    Some people would have us think that prohibiting Federal 
funding of embryonic stem cell research is stopping science 
entirely. I disagree. As we all know, private funds can be used 
for this research and are being used for this purpose. The 
distinction is whether or not the Federal Government should be 
subsidizing controversial, life-altering research with taxpayer 
dollars, especially when the majority of Americans oppose such 
a move.
    Federal funding is scarce. Indeed, because of funding 
limits, we simply are unable to afford all the research we 
would like to do. I submit that we should use limited taxpayer 
dollars on already proven research demonstrated in areas like 
adult stem cells. Adult stem cells are the ones that are 
treating people right now. In fact, treatments have been so 
effective that many doctors have turned to adult stem cell 
transplants as a standard life-saving therapy for hundreds of 
thousands of people, people suffering from dozens of diseases 
and conditions, including cancer, juvenile diabetes, 
Parkinson's, multiple sclerosis (MS), leukemia, lymphoma, 
spinal cord injuries, and corneal regeneration, among others 
are turning to adult stem cell treatments for help.
    An estimated 50,000 adult stem cell transplants are 
occurring annually worldwide using stem cells from bone marrow, 
umbilical cord blood, and other tissues. Research with adult 
stem cells has produced therapies for more than 70 afflictions 
and demonstrated promising results.
    Advancements in this field are happening every day. Just 3 
months ago, researchers reported they had restored vision to 
people whose eyes were damaged from chemicals. Doctors took 
stem cells from the patient's healthy eye and multiplied them 
in a lab to transplant to the damaged eye where they grew into 
healthy corneal tissue.
    Preclinical trials to treat spinal cord injury patients 
have also proven to be promising in recent years. At age 16, 
Laura Dominguez was paralyzed from the neck down in a car 
accident. Doctors treated Laura for spinal cord injury using 
her own nasal adult stem cells. As a result of the surgery and 
extensive physical therapy, Laura has regained feeling and 
movement in her lower body and she continues to make progress.
    iPS research is another promising field. iPS cells are 
producing unprecedented opportunities in medicine, toxicology, 
and drug discoveries. All over the world, hospitals and 
laboratories are developing IPSCs from individuals with various 
diseases. For example, a clinic in Ontario, Canada, has already 
created more than 130 IPSC lines for 11 diseases. This clinic 
is also working on making lines to address diseases such as 
autism and schizophrenia. If there are additional funds, Mr. 
Chairman, Congress should invest in this type of groundbreaking 
research.
    Supporters of embryonic stem cell research would like to 
ignore such accomplishments. They would suggest that providing 
Federal tax dollars on embryonic stem cell research is the only 
means of getting results. However, the accomplishments among 
adult and pluripotent stem cells versus embryonic stem cells 
prove otherwise.
    I am proud to say that for a decade and a half, the Dickey-
Wicker amendment has protected life. This debate involves 
profound ethical and moral questions. This is a matter of 
conscience for me, but more importantly, it is a matter of 
conscience for millions of Americans who are deeply troubled by 
the idea that their taxpayer dollars may be used to destroy 
another human life when there are other proven techniques 
available.
    Mr. Chairman and members of this subcommittee, I want to 
thank you very much for your time, and I appreciate the 
opportunity to testify on this important issue.
    Senator Harkin. Senator Wicker, thank you very much for 
your statement, and I know of your long-term interest in this 
area. And we thank you for your appearance before the 
subcommittee.
    I know you have a lot of important things and you are a 
busy person like everyone else, and so we thank you for being 
here. Thank you for your testimony. And you are excused, if you 
would like, unless you have something else you would like to 
add as an emphasis point or something like that.
    Senator Wicker. Thank you very much.
    Senator Harkin. All right. Thank you very much, Senator 
Wicker.
    Now we will call our first panel and that will be Dr. 
Francis Collins. Dr. Collins is no stranger to all of us here 
and certainly not to this subcommittee. Dr. Collins was sworn 
in last year as the 16th Director of the NIH, a physician 
geneticist noted for his discoveries of disease genes and of 
course for his outstanding leadership of the Human Genome 
Project. And prior to becoming the NIH Director, he served as 
the Director of the National Human Genome Research Institute at 
NIH.
    Dr. Collins received his B.S. from the University of 
Virginia, an M.D. from the University of North Carolina at 
Chapel Hill, and a Ph.D. from Yale.
    As I said, last August he was confirmed unanimously by the 
United States Senate to be our 16th Director of the NIH.
    So, Dr. Collins, welcome back. Your statement will be made 
a part of the record in its entirety. I have got the clock set 
at 10 minutes. Please take at least that amount of time. If you 
need a few more, we will give you that too to give us your 
thoughts and your views on where we are with embryonic stem 
cell research and the whole area of stem cell research and what 
the status is right now. Dr. Collins, welcome back.

STATEMENT OF FRANCIS S. COLLINS, M.D., Ph.D., DIRECTOR, 
            NATIONAL INSTITUTES OF HEALTH, DEPARTMENT 
            OF HEALTH AND HUMAN SERVICES
    Dr. Collins. Thank you. Good morning, Chairman Harkin, 
distinguished members of this subcommittee. I will make an 
abbreviated version of what is in the written statement, but 
thank you for the opportunity to describe some of the exciting 
science that surrounds human embryonic stem cell research. And 
I have some visual aids that perhaps will assist in terms of 
conveying some of these points, and you should have hard copies 
of those visuals in front of you.
    It is truly an honor to appear before you today to discuss 
this topic, and I would like to thank this subcommittee for its 
steadfast support of the NIH's mission, discovering fundamental 
knowledge about living systems and then applying that knowledge 
to fight illness, reduce disability, and extend healthy life.
    I also want to thank you for your leadership in advancing 
human embryonic stem cell research. From your very first 
hearing in December 1998, as the chairman has already referred 
to, this subcommittee has provided a forum for discussing the 
great promise of this research and has enabled NIH to invest 
more than $500 million in this promising research.
    But today there is a cloud hanging over this field. The 
preliminary injunction issued on August 23, now stayed pending 
further order from the court of appeals, has created deep 
uncertainty in the field of human embryonic stem cell research. 
Some of our Nation's most promising researchers such as these 
stem cell scientists you see here working in the laboratories 
of Drs. Morrison, Daley, and Melton are now asking should I 
even bother to submit my new ideas to NIH. And young scientists 
who were excited about careers in stem cell research are now 
worried about going into this field given the legal 
uncertainty.
    But let us keep the focus of this discussion where it 
belongs. The real reason for distress about the current legal 
uncertainty is that patients may have to put hope on hold. 
While we continue through this legal process, we must keep 
patients and their families foremost in our thoughts. Patients, 
after all, are at the heart of the NIH mission and are the ones 
who stand to benefit the most or to lose the most by the stem 
cell policies we are discussing today.
    I am not a lawyer. I speak to you today as a doctor and a 
scientist, and I appreciated the Chairman's exhortations that 
for the witnesses at this table, myself and Dr. Daley and Dr. 
Morrison, Dr. Peduzzi Nelson, and our wonderful advocate who is 
herself affected by a spinal cord illness, that we should stick 
to the science, and that is my goal.
    But I want to take a few minutes to outline for you the 
promise of human embryonic stem cell research, research that 
could be, frankly, hobbled permanently unless stable Federal 
funding can be assured over the long term. So let us go through 
this.
    There are three different types of human stem cells. All of 
them are interesting. All of them are important, and it is 
important to describe the properties of each.


                       HUMAN EMBRYONIC STEM CELLS


    Let us begin with human embryonic stem cells since that is 
the main topic of this morning's hearing. I will begin with a 
brief overview of the remarkable properties of these cells and 
then describe how they can be used to understand the molecular 
basis of development and disease to regenerate and repair 
tissue and to screen for new therapeutics.
    Human embryonic stem cells possess several unique 
characteristics. First, these cells are called pluripotent, a 
word which means they have the potential to become nearly every 
one of the different types of cells in the human body, as you 
see here.
    Second and importantly, these cells are self-renewing. That 
means they are able to multiply in essentially limitless 
numbers in the lab over many years and to be shared with 
researchers around the world.
    Now, before I go on to describe the potential applications 
of human embryonic stem cell research, let me emphasize, 
though, that as scientists, we are also intensely interested in 
other types of stem cells. Each has different properties, 
different potential applications. So let me speak for a moment 
about adult stem cells.


                            ADULT STEM CELLS


    These are found in various organs and tissues throughout 
the body. These cells have been studied for more than 50 years 
and have saved many lives through procedures such as bone 
marrow transplantation. But because they do not divide 
indefinitely and produce only a limited repertory of cell 
types, they are called multipotent rather than pluripotent. And 
that limitation makes them less than ideal for some types of 
research.



    But let me be clear and demonstrate it by the graph you see 
here. NIH is strongly committed to research using adult stem 
cells (in blue) because there may be other clinical 
applications for which they prove useful that we do not know 
about yet. So as you can see from this graph, we have been 
spending considerably more on adult stem cell research (in 
blue) than on human embryonic stem cell research (in red) for 
the last several years.


                     INDUCED PLURIPOTENT STEM CELLS


    Now a new and third category of stem cells are these so-
called iPS cells, which is what I will call them now. These 
were created as a direct result of the knowledge gained from 
studying human embryonic stem cells and understanding their 
biology. This type of stem cell was only first produced in 2007 
when scientists used a virus to insert molecular instructions 
into the DNA of skin cells, instructions that, amazingly 
enough, turned back the cells' developmental clock. These new 
iPS cells possess many properties of human embryonic stem 
cells. They continue to divide indefinitely and they have the 
potential to give rise to nearly all the cells of the human 
body. iPS cells have the added potential clinical benefit of 
avoiding transplant rejection since they can be derived 
directly from the patient. But let us be clear. They are not 
well understood yet. There is growing evidence, including from 
one of the members of the next panel, for subtle differences 
between iPS cells and human embryonic stem cells. Whether this 
will matter for clinical applications is not yet clear, but 
virtually all investigators working in the field agree that 
ongoing comparisons between iPS cells and human embryonic stem 
cells are critically important because human embryonic stem 
cells remain the gold standard for pluripotency. So to prohibit 
work on human embryonic stem cells will, thus, do severe 
collateral damage to the new and exciting research on iPS 
cells.


                    THREE KEY USES OF HUMAN ES CELLS


    Now I want to turn to the first of three key uses of human 
embryonic stem cells, going back to talk specifically about 
them for the rest of my comments.
    Their value in understanding the molecular pathways and 
development in disease is the first of these three. So, for 
example, you might ask, what genes are expressed in human 
embryonic stem cells and how is that programming altered as 
these cells move down pathways to become blood cells, muscle 
cells, or brain cells, and how does that go awry in the 
presence of a disease mutation and cause an illness or a birth 
defect? One of the very best windows we have now into human 
development is through these human embryonic stem cells. For 
example, scientists are using these cells to study diseases 
such as Fragile X syndrome, which is a developmental 
disability, a rather common one affecting primarily boys; Rett 
syndrome, a debilitating brain disorder affecting primarily 
girls; and Huntington's disease, a late-onset neurodegenerative 
disease.
    A second area, an exciting one and the one that has 
probably generated the greatest public excitement, is 
regenerative medicine, the idea that human embryonic stem cells 
could actually be used as a cell therapy to replace damaged 
tissues for somebody with Parkinson's disease or diabetes. We 
might someday be able to regenerate damaged heart muscle tissue 
in heart attack patients.
    One of the most exciting and most advanced possible 
therapeutic applications of human embryonic stem cells is for 
patients who have been paralyzed by catastrophic spinal cord 
injury. And here in this x-ray is an example of what that has 
done in this patient to disrupt the spinal cord. Researchers at 
the University of California-Irvine and the biotech company, 
Geron, as well as at several other universities and companies 
around the country, are pursuing the possibility that human 
embryonic stem cells can be directed to generate spinal cord 
cells for transplantation. And this summer, this being rather a 
landmark year, Geron began phase I clinical trials of its 
techniques for converting human embryonic stem cells into a 
type of neuronal cell called an oligodendrocyte, which is a bit 
of a mouthful, that is intended for injection into the 
patient's spinal cord at the site where injury has occurred.
    And I am going to show you a computer animation that will 
show you what this looks like. So we are now going to zero in 
on some neurons and their axons, which are transmitting 
electrical signals, which in the spinal cord have to cross 
great distances. And there, that is an oligodendrocyte that 
provides the insulation that allows those signals to pass. If 
the spinal cord is injured, the signals cannot go through. So 
adding these human embryonic stem cells that have been turned 
into oligodendrocytes should--and this has been documented in 
animals--allow a repair of what is otherwise a blocked signal, 
so that it can reach the limbs of the affected individual.
    The potential of this approach in restoring limb function 
has been repeatedly demonstrated in animal tests, some of which 
are pretty dramatic. But no one is sure whether this will work 
in humans with severe spinal cord injury, and even if it does, 
it will take years of additional rigorous research and testing 
before a standardized therapy can be developed. Yet, I think 
anyone looking at this opportunity would say the potential here 
is truly amazing.


                     HIGH-THROUGHPUT DRUG SCREENING


    A third area of opportunity for human embryonic stem cells 
and one that has not received as much attention--but I thought 
you should know about it, because it is actually quite exciting 
for scientists involved in this, is the potential to catalyze 
dramatic advances in therapeutics by using these cells as a 
tool to search for new drugs. Let us consider a specific 
example.
    We desperately need new drugs for a disease called 
amyotrophic lateral sclerosis (ALS). You mentioned you have had 
a witness on this very topic speaking about that, who is no 
longer with us, and this is a disease which, unfortunately once 
it appears, progresses rapidly. This is Lou Gehrig's disease. 
It is characterized by the progressive loss of motor neurons in 
the spinal cord which normally provide the connection between 
the brain and the muscles of the body.
    Now, ideally we would like to find a drug that stabilizes 
those human motor neurons against this kind of cell death, but 
how? Well, suppose you could test a library of hundreds of 
thousands of candidate drug compounds, knowing that somewhere 
in there, there might be one that would be valuable encouraging 
motor neurons to survive. That would be a very attractive 
approach to ALS. Well, can we actually do that?
    I am showing you now a video of three hard-working and 
uncomplaining yellow robots who are doing high-throughput drug 
screening, and this is in a facility right up in Gaithersburg, 
Maryland. This is done in a miniaturized format, supported by 
NIH, that allows researchers to test the effect of more than 
100,000 drug compounds in 48 hours and can, therefore, save 
months or years of time.
    This is not a pipe dream. It is a reality. Lee Rubin's lab 
at Harvard is carrying out exactly this kind of experiment for 
ALS right now. The possibility that human embryonic stem cell 
research might one day enable us to identify a therapy for the 
disease that claimed the lives of so many, including Senator 
Jacob Javits, gives you some hope that this new application may 
provide answers that we desperately need.


                               CONCLUSION


    So in conclusion, Mr. Chairman, I would like to emphasize 
that human embryonic stem cell research provides enormous but 
mostly untapped promise for medicine, but this field has been 
thrust into a precarious state. If this research is slowed or 
halted, the greatest loss will be suffered by the millions of 
Americans with conditions that might be helped by human 
embryonic stem cells. Such people include those suffering from 
heart disease, from diabetes, from liver disease, from vision 
problems, along with those afflicted by spinal cord injuries 
and neurodegenerative conditions like ALS and Parkinson's 
disease.
    The many messages I have received from patients since the 
issuance of the preliminary injunction on August 23 reflect 
these deep concerns. Let me just read you part of one such 
message written to me by the mother of two boys who have 
juvenile diabetes, and she suffers from early-onset Parkinson's 
disease. Here is what she says:

    ``I have held my breath with hope that my sons would 
benefit from the early stem cell research. I watched as 
American scientists and science fell further behind on the 
global scene during the past decade. In 2009, I had such hope 
that once again our medical schools and universities would 
begin to attract the best and brightest young minds to work in 
this exciting and promising area of research.''

    She finishes with this:

    ``This week's news was devastating to me. I had no idea how 
strongly I would be affected by it. Your message of support for 
the research once again gives me hope, hope that there will be 
change, hope that we will see effective treatments in our 
lifetimes for these devastating diseases.''


                           prepared statement


    Hope, Senator. When someone is seriously ill or has a loved 
one who is facing a life-threatening disease, it is often hope 
that sustains them, provides the strength and determination to 
prevail. Moving forward responsibly with all types of stem cell 
research gives us and them good reason for hope, hope that is 
informed by science, rigorous science. Patients and their 
families are counting on the research community to find those 
cures and treatments. Please help us do our part to turn that 
hope into reality.
    Thank you, Mr. Chairman, for your strong support of stem 
cell research, and I would be happy to answer any questions.
    [The statement follows:]

                Prepared Statement of Francis S. Collins

    Good morning, Chairman Harkin and distinguished members of the 
subcommittee. It is an honor to appear before you today to discuss 
human embryonic stem cell research. First, I'd like to thank this 
subcommittee for its steadfast support of the National Institutes of 
Health's (NIH) mission: discovering fundamental knowledge about living 
systems and then applying that knowledge to fight illness, reduce 
disability, and extend healthy life. NIH is grateful for the confidence 
that Congress--and this subcommittee in particular--has shown in our 
ability to achieve this mission, as evidenced by our current $31 
billion budget, and the $10.4 billion provided to NIH through the 
American Recovery and Reinvestment Act. Your support makes our mission 
possible, and we are very grateful.
    Nowhere has this support been more evident than in this 
subcommittee's leadership in advancing human embryonic stem cell 
research. From your first hearing in December 1998, this subcommittee 
has provided a forum for discussing the great promise this research 
holds. With your steadfast support, NIH has invested more than $500 
million in human embryonic stem cell research; one of the most 
promising research avenues of recent times.
    The preliminary injunction issued on August 23 by U.S. District 
Court Judge Royce Lamberth in the Sherley v. Sebelius case, now stayed 
pending further order from the Court of Appeals, has created 
uncertainty in the field of human embryonic stem cell research. Many 
researchers across the country have considered modifying their research 
plans to turn away from an area of research that, while promising, is 
now fraught with uncertainty. Some of our Nation's best researchers, 
who have written grant applications proposing innovative new ideas, are 
now asking, ``Should I even bother to submit my proposal to NIH?'' 
Likewise, young scientists excited about careers in stem cell research 
are concerned about going into this field, given the legal uncertainty.
    But the real reason for distress about the current legal 
uncertainty is that patients may have to put hope on hold. While we 
continue through the legal process, I hope that we can keep the 
patients and their families in our thoughts. They are at the heart of 
the NIH mission, and they are the ones who stand to benefit the most, 
or lose the most, by the stem cell policies we are discussing today.
    I am not a lawyer, and I speak to you today as a doctor and a 
scientist. In that capacity, I want to outline for you the promise of 
human embryonic stem cell research--research that could be hobbled 
permanently unless stable Federal funding can be assured over the long 
term.
    I want to begin with a brief overview of the remarkable properties 
of human embryonic stem cells and then describe how research using 
these cells will:
  --provide key insights into the molecular pathways in development and 
        disease;
  --allow for the development of tissue replacement or regenerative 
        medicine; and
  --enable more targeted and efficient screening of new drug 
        candidates.

Human Embryonic Stem Cells
    Human embryonic stem cells possess several unique characteristics. 
First, these cells are pluripotent, which means that they have the 
potential to become nearly every one of the different types of cells in 
the human body. Second, these cells are self-renewing, which means that 
they are able to multiply in essentially limitless numbers in the lab 
over many years and to be shared with many researchers around the 
world.
    To be sure, scientists are also interested in other types of stem 
cells. Adult stem cells are found in various organs and tissues 
throughout the body. These cells, also sometimes referred to as 
multipotent or somatic stem cells, can develop into a limited number of 
specific cell types, depending upon the organ or tissue from which they 
are derived. However, adult stem cells are less than ideal for many 
types of research and therapy because they do not divide indefinitely 
in culture, and they produce only a limited number of cells and cell 
types.
    In considering the relative benefits of adult and embryonic stem 
cell research, keep in mind that research on the most abundantly 
available source of adult stem cells, hematopoetic stem cells in bone 
marrow, began more than a half-century ago. In fact, Drs. E. Donnall 
Thomas and Joseph Murray were awarded the Nobel Prize in Medicine in 
1990, ``for their discoveries concerning organ and cell transplantation 
in the treatment of human disease.'' Indeed, this research has produced 
clinically validated and widely used treatments that reconstitute the 
immune system after leukemia, lymphoma, and various blood or autoimmune 
disorders have been treated with chemotherapy.
    NIH is strongly committed to research using adult stem cells 
because there may be other clinical applications for which they prove 
useful. NIH has invested many hundreds of millions of dollars over the 
years in adult stem cell research. Indeed, annually we are spending 
almost three times as much on adult stem cell research as on human 
embryonic stem cell research.
    A new and third category of stem cells are induced pluripotent stem 
(iPS) cells, which were created as a direct result of the knowledge 
gained from studying human embryonic stem cells. This type of stem cell 
was first produced in 2007, when scientists discovered that it is 
possible to instruct adult skin cells to return to a very early 
developmental stage. They accomplished this by using viruses to insert 
molecular instructions into the DNA of skin cells--instructions that 
acted to turn back the cells' developmental clock. These new cells 
possess many properties of human embryonic stem cells: they continue to 
divide indefinitely and are pluripotent, with the potential to give 
rise to all the cells of the human body.
    While induced pluripotent stem cells are of great interest to 
scientists, and have the added potential clinical benefit of avoiding 
transplant rejection since they can be derived directly from the 
patient, they are not well understood yet. A growing body of research, 
including a publication just 2 months ago from Dr. George Daley, who is 
here today, and his collaborators suggests that there are subtle, but 
potentially important differences between iPS cells and human embryonic 
stem cells. On close examination with powerful molecular 
fingerprinting, it seems that iPS cells retain some memory of the 
tissue from which they were derived. Whether this will matter for 
clinical applications is not clear, but virtually all investigators 
working in the field agree that additional comparisons between iPS 
cells and human embryonic stem cells are critically important. Human 
embryonic stem cells remain the gold standard for pluripotency: to 
prohibit work on human embryonic stem cells will thus do severe 
collateral damage to the new and exciting research on iPS cells.

Molecular Pathways in Biological Development and Human Disease
    While many researchers are focused on coaxing human embryonic stem 
cells to develop into a particular cell type, such as insulin-secreting 
cells or liver cells, understanding the basic biology of stem cells 
themselves will be extremely valuable to understanding human 
development. We have learned much about the genes required for 
pluripotency, but there is much more to understand. For example, what 
genes are expressed in human embryonic stem cells? How is that program 
altered as these cells move down pathways to become blood cells, muscle 
cells, or brain cells? How are these steps regulated? What happens if 
one of the genes doesn't function properly? Our best window into human 
development is using human embryonic stem cells.
    In addition to understanding normal human development more 
completely, human embryonic stem cells are providing key tools to help 
us study the origins of many devastating diseases that afflict babies 
and young children. Such research may even help to uncover targets for 
drug development. We now have a number of human embryonic stem cell 
lines that are known to carry mutations that cause specific diseases. 
For example, scientists are studying cell lines with a mutation in the 
FMR1 gene that causes Fragile X, a developmental disability. The FMR1 
gene normally makes a protein that the brain needs to develop properly. 
However, the Fragile X mutation in the FMR1 gene causes the body to 
make only a little or none of the protein. Research using human 
embryonic stem cells with this mutation showed that although the FMR1 
gene is expressed normally in Fragile X, it is turned off after the 
cells begin to differentiate. How this happens is something we can 
study using human embryonic stem cell lines. Dr. Daley also studies a 
number of human embryonic stem cell lines with various genetic 
mutations, and I am sure he can tell you more about his research.
    One ongoing NIH grant focuses on Rett syndrome, a debilitating, 
developmental brain disorder generally afflicting young females and 
caused by mutations in a gene called MECP2. This research uses human 
embryonic stem cells to generate human brain cells with a deficiency in 
the MECP2 gene, and then studies the effects of this deficiency on the 
development and functions of these brain cells. Such research could 
improve our understanding of Rett syndrome, and facilitate the 
development of therapies for it.
    Another research team has recently generated human embryonic stem 
cell lines containing mutations in the HTT gene that causes 
Huntington's disease, a late-onset neurodegenerative disease. 
Huntington's disease has been studied for a long time, but the normal 
function and pathogenesis of the protein coded for by the HTT gene is 
not fully understood.

Tissue Replacement or Regenerative Medicine
    One of the more exciting and high-profile potential applications of 
human embryonic stem cell research is the possibility that such cells 
can be programmed to replace or regenerate tissues damaged by disease 
or injury. For example, we might one day be able to regenerate damaged 
heart muscle tissue in heart attack patients, develop insulin-producing 
pancreatic beta cells to replace those lost or damaged in people with 
Type 1 diabetes, or restore spinal cord neural connections in patients 
paralyzed by catastrophic spinal cord injury.
    Part of the devastation that heart attack victims suffer is that, 
because of restricted blood flow and oxygen deprivation, their heart 
muscle cells die, leaving the heart much weaker and less able to pump 
blood throughout the body. Today we are studying the tantalizing 
possibility that human embryonic stem cells, or perhaps adult stem 
cells or iPS cells, might be programmed to replace damaged or destroyed 
heart muscle cells, known as myocardial cells. The prevalence of heart 
disease, along with the scarcity of hearts and heart tissues available 
for transplantation and the associated clinical and autoimmune problems 
of transplantation, make this line of research imperative.
    Type 1 diabetes is a disease in which a specific type of cell, 
insulin-producing pancreatic beta cells, is damaged or destroyed by the 
patient's own immune system. A major challenge is to understand the 
autoimmune response that kills these cells in children who then develop 
Type 1 diabetes, but human embryonic stem cells offer the hope that we 
might one day produce replacement cells that avoid the autoimmune 
challenges associated with today's rudimentary transplantation 
therapies. To do that, we need to know more about how stem cells are 
genetically programmed, how they differentiate, and how they renew 
themselves; but as our understanding and ability to work with these 
cells expands, we are laying the foundation for an entirely new--and 
much more effective--way to address the devastation of Type 1 diabetes.
    One of the most exciting--and most advanced--possible therapeutic 
applications of human embryonic stem cells is for patients who have 
been paralyzed by catastrophic spinal cord damage. Researchers at the 
University of California--Irvine and at the biotech company Geron 
Corp., as well as at other universities and companies around the 
country, are pursuing the possibility that human embryonic stem cells 
can be directed to generate spinal cord cells for transplantation.
    This summer, Geron began phase I safety trials of its technique for 
converting stem cells into a type of neuronal cell, known as 
oligodendrocytes, intended for injection into the patient's spinal cord 
at the site where it has been severed by injury. The hope, which has 
been repeatedly demonstrated in animal tests, is that the newly 
injected oligodendrocytes might repair the damaged insulation around 
the severed nerve cells of the spinal cord, and thereby enable those 
cells to once again send signals to the patient's limbs and organs. We 
are not sure that this approach will work, and even if it does, it will 
take years of additional research and testing before we can develop a 
standardized therapy using these techniques. Still, the potential that 
this research holds is truly amazing.
    For all of these efforts, there are many scientific challenges that 
must be addressed. We need to figure out how to get human embryonic 
stem cells to differentiate down specific pathways in a well-controlled 
process. We also need to make sure that the resulting cells behave in 
predictable ways. Because human embryonic stem cells are immortal and 
can proliferate endlessly--much like cancer cells--we need to be sure 
that they or their differentiated ``daughter'' cells do not produce 
tumors or otherwise harm patients. The field of regenerative medicine 
is young. It is unreasonable for us to think we will have cures within 
a set time period. It is also wrong to overpromise on the speed and 
scope of such research to patients and their families. But we must 
persevere and move this research forward in a strong and consistent 
manner. That is why the delay and uncertainty associated with the 
current legal situation is so disheartening for both researchers and 
patients. As I said at the time the injunction was issued, this 
unexpected development is like pouring sand into the engine of 
discovery.

Targeted, Efficient Screening of New Drug Candidates
    Recently, human embryonic stem cells have received increasing 
attention as a tool for drug screening. High throughput drug screening 
is done in a miniaturized format that allows researchers to test the 
effect of more than 100,000 chemicals on a gene, protein, cell, or 
organism of interest. It is a highly automated process that can test in 
one day what would otherwise take a researcher months or years. Because 
human embryonic stem cells can differentiate into specific human cell 
types in large quantities, they provide the foundation for high 
throughput screening of candidate drug compounds for a given disease. 
This means that we now have the capacity to identify efficiently drugs 
that work in a targeted cell type.
    This is not a promise, it is reality. Human embryonic stem cells 
are currently being used to identify drug candidates that can slow or 
stop the progression of amyotrophic lateral sclerosis (ALS). Also 
called Lou Gehrig's disease, ALS is an ultimately fatal disease 
characterized by the progressive loss of motor neurons, which provide 
the connection between the brain and the muscles of our body. The 
possibility that human embryonic stem cell research might one day 
enable us to identify a therapy for the disease that afflicts 
astrophysicist Stephen Hawking and claimed the life of Senator Jacob 
Javits, gives you some sense of the hope this new application might 
provide.
    There are very few drugs available for ALS, and none that prolong 
the patient's life for more than a few months. Dr. Lee Rubin, a 
researcher at Harvard's Stem Cell Institute, and his colleagues have 
developed an elegant set of studies to screen for drugs that prevent 
motor neuron death. The scientists differentiated mouse embryonic stem 
cells into large numbers of motor neurons and exposed them to thousands 
of compounds to find the ones that improve the survival of these vital 
cells. Dr. Rubin and his team identified a handful of promising 
compounds that they then tested in motor neurons derived from human 
embryonic stem cells. The most promising of these can now be moved 
further along the pipeline from drug discovery to clinical trials.
    Drugs fail for many reasons: lack of efficacy in humans is 
responsible for 30 percent of drug failures, and unpredicted toxicity 
is responsible for more than 20 percent of failures. The traditional 
methods of using animal or abnormal human cell lines for safety and 
efficacy testing provide a poor model of how a human will respond to a 
particular drug. Human embryonic stem cells can generate the 
appropriate cell type and even disease cell lines for efficacy testing 
early on, as in the case of the ALS study. They are also being used to 
understand the toxicity of promising compounds in the early stages of 
drug development. For example, liver toxicity is a common cause of drug 
failure. Human embryonic stem cells can be differentiated into human 
liver cells, or hepatocytes, which are then exposed to novel drugs to 
identify any obvious liver toxicity and provide early insight on how a 
drug will be metabolized by the liver. In this manner, human embryonic 
stem cells provide drug developers and researchers a model of how 
actual human livers will respond to a drug. Our hope is this will 
reduce the number of drugs that fail in human clinical trials because 
of low efficacy or unacceptable toxicity.

The NIH Stem Cell Guidelines
    President George W. Bush first funded research on human embryonic 
stem cells--but that decision only allowed the use of cell lines that 
had been derived before August 9, 2001. Ultimately, that only amounted 
to 21 cell lines, and as science moved forward it was clear that this 
somewhat arbitrary time stamp was significantly inhibiting the field. 
On March 9, 2009, President Barack Obama issued Executive Order 13505, 
Removing Barriers to Responsible Scientific Research Involving Human 
Stem Cells. The Executive Order states that the Secretary of Health and 
Human Services, through the Director of NIH, may support and conduct 
responsible, scientifically worthy human stem cell research, including 
human embryonic stem cell research. This Executive Order prompted a 
rapid expansion of scientific effort and progress.
    The President asked NIH to review existing human stem cell research 
guidelines and issue new guidelines, consistent with the President's 
Executive order, within 120 days. NIH immediately began a comprehensive 
review that resulted in draft guidelines that were published in the 
Federal Register for public comment on April 23, 2009. After careful 
analysis of more than 49,000 comments from scientific, patient 
advocacy, medical, and religious organizations, as well as private 
citizens and members of Congress, NIH published final guidelines, 
effective July 7, 2009. The guidelines provide a framework for funding 
scientifically worthy research using responsibly derived human 
embryonic stem cells. The guidelines restrict Federal funding to cell 
lines derived from embryos that: were created for reproductive purposes 
and were no longer needed for that purpose; were donated for research 
by individuals who sought reproductive treatment; and for which the 
donors gave voluntary written consent. Since the President issued his 
Executive order and NIH implemented its guidelines, 75 human embryonic 
stem cell lines have been approved for use in NIH-funded research. All 
were reviewed rigorously and found to meet the high ethical standards 
laid out in the NIH guidelines. The review process is so rigorous that 
48 stem cell lines have not been approved for use in federally funded 
research. Prior to the court's order, in fiscal year 2010, NIH funded 
199 grants for research on human embryonic stem cells totaling $137 
million. These grants support a broad range of research including 
studies to improve stem cell technologies, studies to compare different 
types of stem cells, and studies to develop cell types for use in 
treating debilitating diseases and disorders such as diabetes, liver 
failure, and neurodegenerative diseases.
    If the Government is not successful in defending the guidelines in 
this litigation, and NIH will have to withdraw future NIH support for 
all grants involving human embryonic stem cell research, drastic 
scientific consequences will occur. Since funding for these projects 
would be discontinued mid-stream, all the funds that have been put in 
accounts or already drawn down--$270 million over the 2- to 5-year life 
of these grants, including what has been provided fiscal year 2010--
would have been wasted. The research momentum that this subcommittee 
worked so hard to achieve would be lost.
    Young scientists may opt out of this field due to the chaos of 
stopping, then starting and now stopping again. More senior 
investigators may look to other countries, such as Singapore, China, 
and the United Kingdom to pursue their work. The greatest loss, 
however, will be for the millions of Americans with conditions 
currently under study with human embryonic stem cells. Such people 
include those suffering from heart disease, diabetes, liver disease, 
and vision problems, along with those afflicted by spinal cord injuries 
and neurodegenerative conditions like ALS and Alzheimer's disease. The 
many messages I have received from patients since the issuance of the 
preliminary injunction reflect these deep concerns. Here is part of 
just one such message:

    ``I am a mother of two adult sons with Type I diabetes (since age 
7), and a person with young onset Parkinson's disease. I have watched 
as my oldest son moved from taking the old beef/pork insulin to taking 
genetically engineered insulin, and have held my breath with hope that 
my sons would benefit from the early stem cell research.
    ``I watched as American scientists and science fell farther behind 
on the global scene during the past decade. In 2009, I had such hope 
that once again our medical schools and universities would begin to 
attract the best and brightest young minds to work in this exciting and 
promising area of research.
    ``This week's news was devastating to me. I had no idea how 
strongly I would be affected by it. Your message of support for the 
research once again gives me hope. Hope that there will be change. Hope 
that we will see effective treatments in our lifetimes for these 
devastating diseases.''

    Thank you, Mr. Chairman, for your strong support of stem cell 
research. I would be happy to answer any questions.































                THE NEED FOR ADDITIONAL STEM CELL LINES

    Senator Harkin. So, Dr. Collins, thank you. As usual, a 
very lucid and understandable presentation of a very complex 
issue and complex science.
    We will begin a round of 5-minute questioning. We have two 
votes, I think, starting at 10:45 a.m., if I am not mistaken. 
So we will do as much as we can, and then we will return.
    Because of the Executive order signed, the number of stem 
cell lines eligible for Federal funding rose from 21 to 75. Dr. 
Collins, why is this important to scientists? What can 
researchers do with these additional lines they could not do 
before? Would it help to have even more lines?
    Dr. Collins. I think President Bush deserves credit for 
being the first to provide Federal funds for human embryonic 
stem cell research, and that did lead to these 21 lines that 
have been utilized over the course of several years. But they 
were derived a long time ago and many of them under 
circumstances that today we would say are less than ideal. 
There are many advances in the science over that time table, 
and many other stem cell lines being derived during that time 
table since 2001 were not available to scientists who had great 
interest in studying them.
    In particular, to be able to have available human embryonic 
stem cell lines that have specific genetic mutations in them 
would be a great advance in terms of the ability to study 
certain diseases such as Fragile X, for instance, or 
Huntington's disease, and such were not in the collection of 21 
lines.
    Furthermore, those 21 lines were very nondiverse in terms 
of their origins, nearly all of them coming from individuals of 
Northern European background, and if you were seriously 
thinking about the possibility of utilizing these 
therapeutically, as Geron is now doing for spinal cord injury, 
that could greatly limit the ability to use them for people of 
different backgrounds. So there is enormous enthusiasm and 
intense interest on the part of the scientific community to 
have this panel broadened, and the Obama Executive Order of 
March 2009 made that possible under carefully described 
conditions to maintain the most ethical standards in terms of 
how such lines would have to have been derived in order to 
qualify for Federal funding. And NIH now has on its registry 75 
lines that have met those standards and more to come.

                    LIMITATIONS OF ADULT STEM CELLS

    Senator Harkin. Very good.
    In Senator Wicker's opening statement, he said the 
following:

    ``People suffering from dozens of diseases and conditions, 
including cancer, juvenile diabetes, Parkinson's, MS, leukemia, 
lymphoma, spinal cord injuries, and corneal regeneration, among 
many others, are turning to adult and induced pluripotent stem 
cell treatments for help.''

    My understanding has been that while adult stem cells are 
used routinely to treat blood diseases, this is not the case 
for any other type of disease. Could you please enlighten us on 
that aspect?
    Dr. Collins. Sure. Adult stem cell research has been 
studied now for more than 50 years----
    Senator Harkin. You said that.
    Dr. Collins [continuing]. And certainly has been primarily 
utilized clinically for bone marrow transplantation where it 
has been of great value. We are, as you saw from the graph, 
spending almost $400 million a year on non-embryonic stem cell 
research, looking for additional applications where adult stem 
cells could also be of benefit. And the Senator's opening 
statement mentioned some areas of potential interest, but they 
are far from being what you would call standardized care yet. 
They are experimental.
    I think one of the unfortunate aspects of the discussion 
about human embryonic stem cells is that it has somehow implied 
that scientists are opposed to research on adult stem cells. 
Not at all. Speaking for myself and the others who will be here 
today, we celebrate all of the ways that every kind of stem 
cell can be utilized for effective research, but should we not 
be pursuing the most exciting options in parallel and not 
assuming that we know one of them is going to be better than 
the other? Because right now, we have absolutely no reasons to 
say that. And I think most would assume that, depending on the 
application, adult stem cells may be better in one instance; 
embryonic stem cells may be better in another. We will never 
know if we are not allowed to do the research.
    Senator Harkin. So it is not just two different camps. It 
is a blending of all of this.
    Dr. Collins. Absolutely. Dr. Daley would probably tell you 
he has made major advances in both those fields, and he would 
be right.

                     IMPORTANCE OF FEDERAL FUNDING

    Senator Harkin. Lastly, we sometimes hear opponents of this 
research say Federal funding is not needed. There are other 
potential sources of money in the private sector. How would you 
respond to that?
    Dr. Collins. Well, of course, that was an argument that 
basically prevailed before there was any allowance for Federal 
funding for human embryonic stem cells and led to some States 
taking action. But most of the really critical observations 
that need to be made in terms of understanding the potential of 
human embryonic stem cells are unlikely to happen without the 
kind of Federal support in our best universities and medical 
centers around the country. That is where the talent often lies 
for doing those really fundamental explorations of the nature 
of these cells. To assume that private sector investment, 
although it is critical in terms of those ultimate 
translational steps, is going to be sufficient is to not 
understand the many steps that we need to pursue now in order 
to fully flesh out the potential of this approach to treating a 
long list of conditions.
    Senator Harkin. Thank you very much, Dr. Collins. My time 
is out. I do have a couple of follow-up questions, but I will 
wait my turn. I will turn to Senator Cochran for his.
    Senator Cochran. Dr. Collins, we were talking specifically 
today about the options for Federal support for research and 
specifically using embryonic stem cell therapy. What in your 
judgment would happen if we did not approve Federal funding or, 
if for some reason, the funding sources in the Federal 
Government to support this kind of research dried up for 
whatever reason--action of Congress or, heaven forbid, running 
out of money?
    Dr. Collins. If Federal funds were terminated for the 
support of human embryonic stem cell research, that would be an 
absolutely devastating outcome. You would see large numbers of 
scientists who have already developed a lot of momentum in this 
field becoming extremely disillusioned. You would see many of 
them potentially moving into other research areas or moving 
overseas. Most importantly, you would see that hope for the 
treatment of many diseases that we currently lack effective 
ways to intervene being dashed.
    I do not want to overstate here the potential for human 
embryonic stem cell research to solve all those problems 
because we just do not know, and we have to be careful that our 
hope does not turn into hype, and I think people here will be 
careful about that. But you know, if you were living in 1950 
and somebody said, you know, those iron lungs are working 
pretty well, maybe we do not need to do anything more about 
polio, what a terrible mistake that would have been. So we have 
some science now that is working in some areas, but we have 
this new potential to do something really that is game-
changing. To have that cut off at the knees would be a 
devastating blow.
    And let me say one other thing that I tried to put into my 
statement which I think has not been appreciated. It is not 
sufficient to say, well, we now have iPS cells, so we do not 
need human embryonic stem cells anymore. We have to compare 
those side by side, every step of the way right now, because we 
do not understand the subtle differences between them and what 
that might mean, and if we give up doing that comparison to the 
gold standard for pluripotency, we may damage the potential of 
iPS cells just as they are beginning to gather momentum.
    Senator Cochran. Well, I appreciate very much your being 
here today. I think your testimony has helped us understand in 
a real way, a practical way, what the consequences are for a 
breakdown in Federal support for this research. Thank you very 
much.
    Dr. Collins. Thank you, Senator.
    Senator Harkin. Senator Specter.
    Senator Specter. Thank you, Mr. Chairman. I thank you for 
your generous comments and for scheduling this early hearing to 
take up the important subject of embryonic stem cell research. 
I look forward to debating with Senator Wicker the issues which 
he has raised more appropriately on the Senate floor than in 
this hearing, I think, and would ask unanimous consent that a 
commentary by Bob Schieffer on 60 Minutes be included in the 
record where there is a comparison between those who oppose 
stem cell research with those who challenged the use of 
Galileo's telescope because they believed their doctrines and 
tradition had already told them what was necessary to be seen.
    [The information follows:]

                [From www.cbsnews.com, August 29, 2010]

               Stem Cell Injunction Dictated by Doctrine
                           (By Bob Schieffer)

    Last week two people I know were diagnosed with colon cancer, one 
of the deadliest of all cancers.
    Because my wife and I are cancer survivors, and because my mother 
died of cancer because she was afraid to go to the doctor, I've come to 
know a little about the disease.
    My friends have a serious illness, but there is a path to recovery 
that was not there not so long ago. And as I talked to them last week, 
I was again struck by the remarkable progress science is making to give 
them that path.
    Being told we have cancer no longer means we've been given the 
death penalty.
    Like all scientific breakthroughs, advances in cancer research 
began and depend on basic research--science's ability to go not where 
doctrine or tradition dictates, but where research takes it.
    Ironically, my friends were diagnosed about the time a Federal 
judge issued the injunction placing limits on stem cell research, an 
area that holds the greatest possibilities for medical breakthroughs 
since penicillin.
    I have the greatest respect for those who disagree, but to me 
putting restraints on stem cell research is not far from those who 
refused to look through Galileo's telescope because they believed their 
doctrines and tradition had already told them what they would see.
    Their beliefs, too, were deeply held--but where would the store of 
knowledge be had their view prevailed?
    As we again try to untangle the arguments over stem cells, let us 
also consider this: No civilization, no society, has survived if its 
people came to believe they knew enough and needed to know nothing 
more.

                      IMPACT OF JUDICIAL DECISIONS

    Senator Specter. The decision by the district court in the 
District of Columbia has had a very serious impact on the 
research. May the record show a nod by Dr. Collins. And I will 
ask him specifically about that. But in our informal 
discussion, he said that while they have been able to proceed 
with the expenditure of Federal funds with the circuit stay, 
that the researchers are very, very concerned about their 
ability to move forward. And we have a stay which has been 
issued until next Monday, September 20, and we do not know what 
will happen after that.
    That is why I moved very promptly, as soon as we were in 
session--on Monday of this week we went into session at 2:30 
p.m., and before 3 o'clock I had to go to the floor to 
introduce legislation to overturn the court decision because 
Congress has the authority to make this determination. It is 
not constitutional issue. It is a matter of statutory 
interpretation. And the evidence is overwhelming about the 
importance of embryonic stem cell research to deal with the 
maladies of the world. And if there are 400,000 frozen, which 
will be lost and that we are not dealing with human life--if 
they would be turned into human life, no one would suggest 
using them for medical research.
    The vicissitudes of the legal battle are very, very 
uncertain as to what will happen in the circuit court, whether 
the stay will be maintained or whether the Supreme Court might 
issue a stay. There have been surprising stays issued by the 
Supreme Court in the past several months. There was an Arizona 
campaign finance law which provided for public funding where 
the Court of Appeals for the Ninth Circuit upheld the law, 
overruling the district court, and the Supreme Court of the 
United States, without even a petition for certiorari filed, 
intervened in the case to grant a stay--really unheard of--on 
ideological grounds.
    We had a trial in process before Chief Judge Walker in San 
Francisco on the issue of gay marriage, and the Supreme Court 
intervened to stop televising on closed circuit television.
    So the legislature, the Congress, had better get busy and 
it better act on this subject so we do not await court action. 
We do not put the scientists under the pressure of knowing what 
may or may not happen.
    I have a couple of questions for you, Dr. Collins. The 
first question relates to the impact of the judicial decision, 
and I have gotten the information that more than $500 million 
has been expended on embryonic stem cell research. Well, 
actually three questions.
    Question number one is what has the impact been on the 
scientists now using NIH funding for embryonic stem cell 
research in terms of the uncertainty of the future.
    Number two, what results have been taken in a positive 
sense, which I know are very good for the more than $500 
million already expended?
    And what has been the consequence of the $10 billion in the 
stimulus package where you informally told me that it has 
created a tremendous excitement and a new wave of enthusiasm by 
researchers who had been discouraged by the failure of Congress 
to keep the pace, which we had moved from $12 billion to $30 
billion, but failure to keep the pace in funding since 2003?

  THE EFFECT OF THE PRELIMINARY INJUNCTION ON NIH EMBRYONIC STEM CELL 
                                RESEARCH

    Dr. Collins. Well, Senator, thank you for the question, and 
let me first say how appreciative I am personally and everyone 
at NIH is for the strong leadership you have shown over these 
years and your advocacy for the value of medical response and 
especially, because we are talking about it today, for stem 
cell research. That has been much appreciated and your 
articulation of the importance has always been right on target, 
as it just was here in your opening statement. And we are all 
grateful, indeed, for the way that you have shown that 
leadership. And you, together with our chairman, have played 
such a significant role in NIH being at this exciting place 
that we are right now in terms of medical research 
opportunities that, frankly, I did not dream we would be at 10 
years ago.
    But we also are here with this cloud over the enterprise in 
this very specific area of human embryonic stem cell research.
    When the judge issued that preliminary injunction, we were 
stunned, and basically after interpretations by the Department 
of Justice, took steps that we felt we had to with intramural 
researchers who were working with Federal funds at that very 
time doing embryonic stem cell research. We had to ask them to 
stop. With extramural grantees, if they had already received a 
grant and were spending down the dollars that they had already 
been allocated, they could continue, but they would need to 
come back for a renewal on an annual basis, and we basically 
said within a year, there will be no more funds because those 
annual renewals cannot be adhered to. And frankly, we had a 
bunch of new grants and renewals right in front of us, about 
244 grants, adding up to about $200 million, that were 
immediately put on hold, not to mention a whole other set of 
grants that were ready for peer review that we had to stick on 
the shelf because we felt the judge's order prevented us from 
acting on them.
    Fortunately and to our great relief, although temporary 
relief it apparently is, the stay on that particular injunction 
last week allowed us to catch up and to go back to doing what 
we had been doing, and we are working vigorously to be sure 
that we are doing the right thing here in terms of supporting 
the research that we had always intended to.
    But there is this cloud of uncertainty that hangs over the 
situation because there is not a clear path forward. And I 
think as you will hear from others at this hearing, that is 
creating great anxiety particularly amongst young scientists 
who are wondering, ``Do I have a career path here or is this 
something I better just not get involved in because it is too 
uncertain?''
    So the impact so far has been quite significant and is 
uncertain going forward. We are, as I tried to show in the 
graph in the opening statement, spending in the neighborhood of 
$188 million--sorry--$138 million on nonembryonic--I am sorry--
on embryonic and other types of nonadult--let me try that 
again. We are spending $138 million on human embryonic stem 
cell research and all of that was put into jeopardy. And that 
is an estimate for fiscal year 2010.

          AMERICAN RECOVERY AND REINVESTMENT ACT (ARRA) AT NIH

    In terms of your question about the ARRA dollars that have 
flowed to NIH, that has been an enormous infusion of energy and 
capability and excitement in a community that had, frankly, 
been struggling after 5 years of flat budgets and many 
innovative ideas lacking support. The infusion of that money 
has made it possible to put into place a whole host of 
innovative projects.
    One of the things I have done in my first year as NIH 
Director is to read a lot of those grants that came in because 
of ARRA to see what was there. And it is some of the most 
exciting science that you can imagine, and we have used it 
specifically to encourage people to put forward out-of-the-box 
ideas that might otherwise have not seemed worth trying in a 
very tough budget climate. This has supported breakthroughs in 
cancer, in heart disease, in diabetes, in autism, things that 
really have changed the whole landscape because of this 
opportunity to empower the community in ways that they had not 
been previously been able to do. And I want to thank you for 
your remarkable leadership in making that possible.
    Of course, we have another anxiety there, that the 2 years 
of ARRA are coming to a close, and the momentum that was 
started is now somewhat in question.
    Senator Specter. You testified about the advances 
generally, but specifically on stem cells with the $500 million 
expended, tell about the big results there.
    Senator Murray. Mr. Chairman, before Dr. Collins answers 
that, a vote has been called and I want to go to the floor. So 
I just want to say really quickly----
    Senator Harkin. Yes.
    Senator Murray [continuing]. That, Dr. Collins, your 
testimony is extremely compelling and understandable, and I 
really appreciate it. I think it clarified a lot for me.
    I want to thank Senator Harkin for his leadership on this, 
but I especially want to thank Senator Specter too whose voice 
we will miss on this panel. And we will continue to carry your 
spirit forward on this critical issue. I just wanted to say 
thank you very much.
    We do have a vote and I want to make sure we get to the 
rest of the panel.

                ADVANCES IN EMBRYONIC STEM CELL RESEARCH

    Senator Specter. Thank you, Senator Murray.
    Well, just focus for a moment, if you would, on the $500 
million already expended on embryonic stem cell research and 
what tremendous advances have been made there.
    Dr. Collins. So that is a long list. It has given us the 
opportunity at the basic level to begin to understand what it 
is that takes this cell with all of this potential and triggers 
it to become a neuron or a muscle cell or a pancreatic beta 
cell that makes insulin. Those signals, those elaborate 
pathways of development, are now being sorted out by 
researchers working on that with very powerful technologies, 
some of them coming from the genome project.
    And in terms of specific applications, you have heard of 
the application to spinal cord injury which is now in its Phase 
I clinical trial. That is the first one which has actually made 
it through that. That was a lot of Food and Drug Administration 
(FDA) review, believe me. But there are also applications which 
are looking very promising for eye diseases and for Type 1 
diabetes where human embryonic stem cells have been 
differentiated into the cells needed in that circumstance and 
have then been used in an animal model to show clear benefit 
and rigorous science, setting the stage then for human clinical 
trials in the not-too-distant future.
    On top of all that, human embryonic stem cells are being 
used, as I mentioned, to do drug screening because if you are 
looking for a drug that might help somebody with a muscle 
disease, you would really like to test and see does that work 
in human muscle cells? Well, we now have the ability to make 
human muscle cells because you can take human embryonic stem 
cells and tweak them to do that and then test hundreds of 
thousands of compounds to see what is there that would 
stabilize a muscle cell, make it healthy or make it better able 
to survive. A huge opportunity in drug screening which is 
happening both in the private sector and in academia. All of 
those things add up to that roughly $500 million and some, but 
we think we are just scratching the surface of the potential 
here.
    Senator Specter. Thank you, Dr. Collins. That is powerful.
    Senator Harkin. Thank you, Senator Specter.
    Dr. Collins and others, we have a vote. There are two 
votes. So we have about 6 or 7 minutes left in this vote. So I 
will recess the panel. We will go vote on one, and then we will 
vote on the next one. So it will be probably 15 minutes before 
we get back here. So I would like to say if anybody needs to 
use the facilities or something, we will be back in 15 minutes.
    What I would like to ask, Dr. Collins--I hate to impose on 
you, but there are a lot of things we need to cover. I would 
like to ask if you could stay during our second panel. I would 
appreciate that very much if you could. I am not going to put 
the guards at the door, but I would really like to have you 
stay.
    While we are gone, I am going to ask the staff to go ahead 
and put the other nameplates of the second panel up there.
    But I do have some follow-up questions for you, Dr. 
Collins, when we come back.
    Dr. Collins. No guards needed. I am happy to stay.
    Senator Harkin. All right, thanks, Dr. Collins.
    We will be right back.

                EMBRYONIC STEM CELLS AS A RESEARCH TOOL

    Senator Harkin. The subcommittee will resume its sitting. I 
thank everyone for their indulgence.
    Dr. Collins, just two other things I wanted to cover with 
you. One, in your testimony--and you mentioned it also in the 
slides--was the power of embryonic stem cells as a research 
tool. It sounds like even if these cells never actually end up 
being used as therapies in which they are transplanted into 
human beings, they could still teach us valuable information 
that could lead to treatments and cures. I just want to ask is 
that correct and just a slight elaboration on that.
    Dr. Collins. That is correct, and in two ways.
    One is that human embryonic stem cells, because they 
represent that most pluripotent, most undifferentiated cell 
type, but can be encouraged to go down various pathways to 
become muscle cells or brain cells or blood cells, they give us 
a window into how that development happens in humans in a way 
that we did not have before. And again, if you are able to 
understand what those signals are, you can also infer what goes 
wrong if one of those signals misfires, and many birth defects 
and many genetic diseases are in that category.
    The second way, which I also mentioned, is the ability to 
use these cells particularly if you turn them into neurons or 
muscle cells, or whatever it is that you need to study most to 
screen for new drug therapies. The way we got drugs in the past 
involved a variety of approaches, trying to identify a small 
molecule, an organic compound that would have the right 
properties to do something you want it to, but you have often 
had to try that in an animal model. These are human cells and 
they are human cells that you can convince to behave pretty 
much the way they would in a person except they are there in 
your dish, so you can do this without the risks of toxicity. A 
very powerful new way to find the next generation of drugs.

            THE ETHICS OF HUMAN EMBRYONIC STEM CELL RESEARCH

    Senator Harkin. Very good.
    Dr. Collins, on a kind of a more personal note, opponents 
of human embryonic stem cell research sometimes argue that it 
is immoral. They have raised it into a moral issue. Quite 
frankly, I have a number of friends, but I have one very close 
family friend who had a lot of trouble conceiving a child. She 
and her husband tried many different things. They finally went 
to a fertility clinic, and through in vitro fertilization, she 
was able to conceive and have a wonderful child. Actually twins 
who are very healthy. And so that is a real blessing that 
science was able to help them.
    Now, I do not know this for a fact, but in many of these 
cases, a lot of the embryos are left over from a process of in 
vitro fertilization. And at some point, the donors are asked 
what they want to do with them. And obviously, they are not 
going to keep them in liquid nitrogen forever, and so they are 
discarded. Some time ago, my friend said to me, well, but I 
understand they could be used for embryonic stem cell research 
that might help someone who is suffering. And I said, well, 
yes, that is true. She said, well, I would much rather do that.
    So it seems to me there is some morality there that we have 
not thought about, and as you know, under the guidelines that 
were issued only stem cells derived from leftover in vitro 
fertilization could be used with full consent of the donors and 
with no monetary consideration and could not be transplanted 
into a uterus. It had to only be used for stem cell derivation. 
So those are the ethical guidelines. Well, I just thought I 
would lay that out. A lot of people do not understand that.
    But you are well known not only as one of the world's 
foremost scientists, but as a man of faith. I actually did read 
your book. I thought it was very good, The Language of God. I 
think it is just one of the wonderful crossover books between 
science and faith. It is just a wonderful book.
    Can you talk about why you personally as a pre-eminent 
scientist are comfortable with this research? How do you 
reconcile your advocacy for embryonic stem cell research with 
your own faith and your own moral judgment?
    Dr. Collins. Thank you, Senator. I think you have already 
articulated the issues extremely well.
    I do believe that the human embryo deserves moral respect. 
It is a potential human being. This coming together of sperm 
and egg is the way we all got started, and that is not 
something to be taken lightly. But when you look at the 
circumstances that you have just outlined in terms of the 
consequences of in vitro fertilization efforts, benevolent as 
they are, giving couples a chance to have children who 
otherwise could not, one of those consequences is the existence 
of hundreds of thousands of frozen embryos that are being 
discarded potentially all the time.
    And then faced with the ethical choice in that situation, I 
have come to the conclusion as a person of faith that the 
alternative of discarding this embryo that is clearly not going 
to get used versus, for a small number of these, trying to turn 
them into a stem cell line that might ultimately teach us 
something about human development in medicine and ultimately 
help us come up with a treatment for Parkinson's disease or 
diabetes or spinal cord injury or some eye disease or liver 
disease, which of those is the more ethical choice?
    I think it is too easy to simply say, well, the embryo is 
an entity that is a potential human and therefore any 
consideration of using the word ``research'' in the same 
sentence is something we should be opposed to. We are not 
really being given that as an alternative. These embryos exist. 
They are going to continue to exist as long as in vitro 
fertilization goes forward, and it is. And certainly it has 
given many couples a chance for a new life in their families.
    So putting the reality test here, I believe that most 
people who look carefully at the issues, whether from a faith 
perspective or a purely humanistic perspective, come up with a 
conclusion that what is potential here justifies what we are 
talking about in terms of Federal funding of human embryonic 
stem cell research.
    Senator Harkin. Well, thank you very much for that profound 
statement. Thank you very much, Dr. Collins. We have statements 
submitted from Senators Murray and Feinstein to be included in 
the record.
    [The statements follow:]

               Prepared Statement of Senator Patty Murray

    Thank you, Senator Harkin, for holding this hearing. Stem cell 
research is not just about science--it's about hope. The hope of 
millions Americans who are suffering from diseases like Alzheimer's, 
Parkinson's, and diabetes. The hope of their friends, families, and 
loved ones who can't bear to see them in pain another day. And the hope 
of a scientific and medical community that is fighting against the 
clock to save lives and reduce suffering.
    Stem cell research offers this hope because it is one of the most 
promising fields in medical research today. And we simply cannot afford 
to allow potential cures to be slowed down or halted by the political 
process.
    That's why I was so glad when President Obama issued an Executive 
order in March of last year to lift the restrictions on funding for 
human embryonic stem cell research. This action took the handcuffs off 
of our scientists and made sure we were exploring every option for 
finding cures to debilitating diseases. Because as so many of us know, 
limiting Federal support of this research will continue to push 
embryonic stem cell research overseas. And our country will continue to 
fall behind in a critical, growing, and cutting-edge field.
    Because of the arbitrary limits on stem cell research that were 
imposed in the past, we are already getting off to a slow start--and we 
can't afford to fall any farther behind. Because in addition to helping 
patients--cutting-edge research also creates jobs and boosts the 
economy.
    My home State of Washington is home to world-class research 
institutions like the University of Washington and the Fred Hutchinson 
Cancer Research Center, just to name a few. They want to help patients. 
They want to do this research to help cure debilitating diseases, but 
we need to make sure they have the resources they need to succeed. And 
that great institutions in Washington State and across the country 
continue leading the way in science, research, and medical cures.
    Nothing sums this issue up better than a letter I received from a 
mom named Suzanne, from Seattle, whose 16-year-old son has diabetes.
    She wrote to me and said:

    ``For our family, embryonic stem cell research offers the hope that 
by the time our son finishes graduate school, scientists will be 
developing new therapies or even a cure for his diabetes. Every year 
that researchers are denied full access to the best cellular material, 
and the funds with which to study it, is a year wasted, and a year 
denied to my son to live outside the burden of his disease . . . Please 
keep hope alive for Charlie, and millions of kids like him.''

    Once again, this is about hope.
    I am going to keep fighting to make sure this hope stay alive for 
Suzanne and millions of others.
    And I am going to keep working to make sure nothing stands in the 
way of our medical researchers and doctors developing cures and 
reducing suffering.
    Thank you, again, Senator Harkin, for holding this hearing, and I 
look forward to continuing to work with you on this issue.
                                 ______
                                 
             Prepared Statement of Senator Dianne Feinstein

    I want to thank Chairman Harkin for calling this hearing today.
    Last month, the U.S. District Court for the District of Columbia 
issued an alarming decision that temporarily halted Federal funding of 
human embryonic stem cell research. That opinion has now been stayed 
pending appeal, but it should serve as a wake-up call to us all.
    We must do everything we can to protect this funding, which is 
essential to lifesaving research innovations.
    Human embryonic stem cells have the potential to become any type of 
cell in the human body, meaning that the potential for treatment of 
disease is unlimited. This kind of research is vital to finding cures 
for Alzheimer's, Parkinson's disease, diabetes, spinal cord injuries, 
and numerous other illnesses.
    National Institutes of Health (NIH) funding of these research 
projects holds out the possibility of a cure for millions of Americans. 
In the last year alone:
  --1.5 million Americans were diagnosed with cancer;
  --60,000 Americans were diagnosed with Parkinson's;
  --12,000 Americans suffered spinal cord injuries; and
  --1.6 million adults were diagnosed with diabetes.
    Those are just the new diagnoses--think of all the other Americans 
who continue to suffer from cancer, heart disease, Alzheimer's, 
Parkinson's, spinal cord injuries, and other catastrophic diseases who 
could potentially be helped by embryonic stem cell research.
    There is no question but that this research must be conducted 
within strict ethical guidelines, and these guidelines must, in my 
view, take into account the millions of people whose lives human 
embryonic stem cell research may dramatically improve or even save.
    President Obama's 2009 Executive order on research involving human 
stem cells paved the way for responsible scientific research and 
removed critical barriers to potential breakthroughs from this 
research.
    Under the Executive order, the strictest guidelines were put in 
place. Any embryo used must be left over following fertility treatment; 
it must be clear that the embryos would otherwise be discarded; 
individuals providing the embryos must provide written consent; and the 
donors may not be compensated for their donation.
    The District Court's decision, and its unprecedented and highly 
restrictive interpretation of the Dickey-Wicker amendment, cast all of 
this aside and placed in peril hundreds of millions of dollars in 
research grants.
    If the decision is not overturned:
  --50 requests for new NIH research funding will not be considered;
  --Roughly 12 requests that were likely to be approved will be frozen;
  --22 grants totaling about $54 million that are due for renewal in 
        September will be cut off; and
  --Another 199 grants already awarded for about $131 million will be 
        at risk next year.
    The Justice Department continues to fight the decision, and I, 
along with Senators Harkin, Specter, Boxer, and others, are committed 
to working to pass legislation in the Senate that ensures that Federal 
funding can continue.
    The United States has long been a leader in biomedical research and 
innovation. We have many of the best and brightest medical researchers 
in the world who are working to end suffering from all kinds of 
diseases. We should give them every tool we can to advance their work 
for the millions of American patients who are hoping and praying that 
they will find a cure.
    I want to thank Chairman Harkin again for holding this hearing, and 
I look forward to working together to protect and promote this vital 
research.

    Senator Harkin. Now I will introduce our panel here, and 
then we will have our testimonies. Dr. George Daley, professor 
of Hematology and Oncology at Children's Hospital in Boston and 
the Dana Farber Cancer Institute, also professor of Biological 
Chemistry and Molecular Pharmacology at Harvard Medical School. 
Dr. Daley is past president of the International Society for 
Stem Cell Research and chaired the International Task Force 
that wrote ethical guidelines for human embryonic stem cell 
research. He received his Ph.D. in biology from MIT and his 
M.D. degree from Harvard Medical School.
    Dr. Sean Morrison, Director of the University of Michigan 
Center for Stem Cell Biology, where he is also a professor of 
Medicine and a professor in the Life Sciences Institute. Dr. 
Morrison is also a director of the International Society for 
Stem Cell Research. He received his Ph.D. in immunology from 
Stanford University.
    Jean Peduzzi Nelson is an associate professor at the 
Department of Anatomy and Cell Biology at Wayne State 
University School of Medicine. Dr. Peduzzi Nelson received her 
B.S. from the University of Michigan and her Ph.D. from Wayne 
State University.
    Ms. Cody Unser is the founder of the Cody Unser First Step 
Foundation, an organization dedicated to raising research funds 
and public awareness for people afflicted with spinal cord-
related paralysis. Ms. Unser graduated in May from the 
University of the Redlands with a degree in biopolitics. She is 
now a graduate student at the George Washington University 
School of Public Health studying health policy.
    I thank you all for being here today, and I thank you for 
your indulgence because of our votes. I will make sure that all 
of your statements are made a part of he record in their 
entirety. And starting with you, Dr. Daley, working down, if 
you could sum up in 5 minutes or so--I will not hold you to an 
exact time, but we will start with 5 minutes and try to get it 
there so we can open it up for some discussion and questions. 
But again, Dr. Daley, no stranger to this subcommittee, welcome 
back.

STATEMENT OF GEORGE Q. DALEY, M.D., Ph.D., DIRECTOR, 
            STEM CELL TRANSPLANTATION; ASSOCIATE 
            DIRECTOR, STEM CELL PROGRAM, CHILDREN'S 
            HOSPITAL BOSTON, BOSTON, MASSACHUSETTS
    Dr. Daley. Thank you very much. Chairman Harkin, thank you 
for the invitation to testify.
    I am here to assert that human embryonic stem cells offer 
unique advantages for understanding human diseases and are 
essential to a vigorous national portfolio of stem cell 
research.
    However, recent upheavals in the Federal funding are 
disrupting our research. They are dissuading scientists from 
entering the field and they are threatening American pre-
eminence in the research.
    As director of the Stem Cell Transplant Program at 
Children's, I wish to first speak to the success we have in 
using adult stem cells. And we are using adult stem cells to 
cure kids with a variety of life-threatening diseases. We 
perform some 80 stem cell transplants per year for childhood 
leukemia, genetic diseases, and indeed, we have cured many 
kids. I was on rounds last week. I met an adorable little girl. 
She was about to receive her transplant for a very rare genetic 
immune disorder, and I found out she was the second in her 
family that we could very confidently say we would cure. So it 
is very, very heartening to save the life of a child.
    But I am also here to advocate as a scientist, and as a 
scientist I am sobered by the statistic that fewer than half of 
all patients treated with stem cells are cured, and despite 50 
years of research in adult hematopoietic stem cell transplants 
and practice--this is our most successful form of transplant--
blood cancers still relapse and patients still die. So as a 
scientist, I am working to improve these treatments through 
research on adult stem cells, embryonic stem cells, and iPS 
cells.
    I think it is a flawed argument to say that scientists 
should restrict their focus to adult stem cells, and I think it 
is a mistake to cast the different types of stem cells as 
competing priorities. Adult stem cells are not better than or 
more promising than embryonic stem cells. Embryonic stem cells 
are different, and to many scientists, they offer more hope in 
certain diseases like diabetes. Would it make sense as a 
Federal policy to fund cancer and cardiovascular research but 
not diabetes research? All of these are essential research 
avenues, and the most successful strategy to advancing cures is 
to let scientists decide which cells to study.
    Now, I have been a student of the hematopoietic stem cell, 
the adult stem cell, for 25 years, but starting about 15 years 
ago, I began envisioning a new approach to the research to 
generating blood stem cells from embryonic stem cells. And the 
idea was that we could generate customized blood stem cells in 
a way that would solve the immune rejection problems, solve the 
donor shortages, and allow us to perform gene repair, together 
with bone marrow transplantation.
    Now, we have succeeded in mice, and we have a lot of 
promise in humans. In 2007, I was one of three laboratories to 
produce iPS cells, and in 2008, my lab produced the first large 
repository of human disease-specific iPS cells.
    So why, given that I have pioneered the development of both 
adult stem cells and iPS cells, do I continue to advocate for 
human embryonic stem cells?
    Well, there are several reasons, and the first is that iPS 
cells and many other future areas of research are founded on 
the knowledge we have gained from human embryonic stem cells.
    Second, my own research and that of others is pointing to 
important differences between iPS and embryonic stem cells.
    And third, some diseases are simply more effectively 
modeled with human embryonic stem cells than iPS cells. We 
recently showed that you could model human Fanconi anemia, a 
disease that predisposes kids to leukemia, as well as Fragile X 
syndrome, which is the most common genetic cause of autism and 
mental retardation, and these were better modeled with human 
embryonic stem cells.
    So when we have so much to learn from embryonic stem cells, 
how can we conclude that we do not need to fund the research? 
We are told that restrictions on Federal funding will not 
inhibit stem cell research and that private philanthropy will 
fill the gap, but realistically research careers are founded on 
the architecture of Federal support. Investment by the NIH has 
made U.S. research pre-eminent. It has given us domination in 
the Nobel Prizes, and it has been an engine for our very 
vigorous biotechnology industry.
    Now, opponents of embryonic stem cell research will argue 
that adult stem cells are more promising, that embryonic stem 
cells have yet to cure anyone. Well, this is like arguing why 
try to develop new classes of antibiotics when we have got 
penicillins and cephalosporins. Let us continue to work to 
improve those.
    It is very curious. The only time I confront the argument 
that adult stem cells are superior and that embryonic stem 
cells should be replaced is at hearings like this. At 
scientific meetings, we discuss and debate adult and embryonic 
and iPS cells as all complementary aspects of cell and 
developmental biology.
    In my opinion, the arguments that adult stem cells obviate 
the need for embryonic stem cells are not scientifically 
driven. They are ideologically driven arguments to suppress 
embryonic stem cell research. And no matter how much progress 
is made with other forms of stem cells, embryonic stem cells 
will remain a vital research tool. embryonic stem cells are not 
contestants on Survivor that should be voted off the island. 
Expelling embryonic stem cells from the researchers' toolkit 
will gravely weaken the search for cures.
    Now, President Obama's policy has expanded access to more 
embryonic stem cell lines, and the court challenge has really 
come on us as a major blow. We have had immediate disruptions, 
but the long-term uncertainty is even more insidious. And I 
have several trainees who have toiled to make their projects on 
human embryonic stem cells work, and the uncertainty has really 
compelled some of them to abandon those plans. So these 
decisions which are driven by politics and not science are 
deeply disturbing.

                           PREPARED STATEMENT

    So let me finish by saying that although the injunction has 
been stayed, with the latest upheavals, we are again reminded 
that human embryonic stem cell research is on fragile and 
fickle footing and that new legislation is needed to sustain 
the momentum of embryonic stem cell research and to allow 
scientists and not politicians and not judges to determine 
which research priorities to pursue.
    Thank you.
    Senator Harkin. Dr. Daley, once again thank you for a very 
profound statement and for all the work that you have been 
doing in this area.
    [The statement follows:]

                 Prepared Statement of George Q. Daley

    Chairman Harkin and distinguished members of the subcommittee, 
thank you for the invitation to testify today on the subject of human 
embryonic stem (embryonic stem) cells. I am here to assert that human 
embryonic stem cells offer unique advantages for understanding a number 
of human diseases and are essential to a vigorous portfolio of stem 
cell research here in the United States. I also wish to recount how 
recent upheavals in Federal funding have disrupted our research and how 
ambiguous Federal policy saps the motivation of junior scientists and 
threatens American pre-eminence in this vital field of biomedical 
research.
    My name is George Daley and I am the Samuel E. Lux IV Professor of 
Hematology/Oncology and Director of the Stem Cell Transplantation 
Program at the Children's Hospital Boston and the Dana Farber Cancer 
Institute. I am also Professor of Biological Chemistry and Molecular 
Pharmacology at Harvard Medical School, Principal Faculty and founding 
member of the Executive Committee of the Harvard Stem Cell Institute, 
and an investigator of the Howard Hughes Medical Institute. I am past 
president of the International Society for Stem Cell Research (ISSCR; 
2007-2008), the major international organization of stem cell 
scientists with more than 3,000 members worldwide. I chaired the 
international task force that wrote ethical guidelines for human 
embryonic stem cell research (ISSCR Guidelines for Human Embryonic Stem 
Cell Research 2006) \1\ and as ISSCR President empanelled and 
participated in the international task force that wrote guidelines for 
the responsible clinical translation of stem cell therapies (ISSCR 
Guidelines for the Clinical Translation of Stem Cells 2008.).\2\ I am 
here representing the American Society for Cell Biology, whose members 
number some 10,000 scientists.
---------------------------------------------------------------------------
    \1\ Daley, G.Q., et al., Ethics. The ISSCR guidelines for human 
embryonic stem cell research. Science, 2007. 315(5812): p. 603-4.
    \2\ Hyun, I., et al., New ISSCR guidelines underscore major 
principles for responsible translational stem cell research. Cell Stem 
Cell, 2008. 3(6): p. 607-9.
---------------------------------------------------------------------------
    As Director of the Stem Cell Transplantation Program at Children's 
Hospital I speak as a doctor who uses adult stem cells to treat 
patients with life-threatening blood diseases--including leukemia, 
sickle cell anemia, immune-deficiency, bone marrow failure, and 
others--but I also speak as a scientist working to improve those 
treatments through research on adult stem cells, embryonic stem 
(embryonic stem) cells, and induced pluripotent stem (iPS) cells. Stem 
cell research is important to real live patients, and I believe to my 
core that stem cell research offers tremendous promise for curing a 
range of diseases. It is a mistake to cast the different types of stem 
cells as competing priorities. Adult stem cells are not better than or 
more promising than embryonic stem cells. And iPS cells do not obviate 
the need for embryonic stem cells. Would it make sense to fund cancer 
and cardiovascular research but not diabetes research? All are 
essential research avenues. The most successful strategy to advance 
stem cell research is to let scientists decide which cells to study.
    Let me first speak to the success and the limitations of adult stem 
cell therapies. Hematopoietic stem cells harvested from bone marrow, 
mobilized peripheral blood, and umbilical cord blood are the most 
successful adult stem cell treatments, and potentially curative for 
cancers of the blood and some genetic diseases. At Children's we 
perform some 80 stem cell transplants per year for childhood leukemia 
and conditions like immune deficiency. Casting our success in a 
positive light, we have cured many kids over the years. On transplant 
rounds last week, I was heartened to meet a little girl about to 
receive her transplant for a rare inherited immune condition, the 
second in her family that we will likely cure. Saving the life of a 
child is deeply gratifying. However, confronting our shortcomings, we 
must acknowledge that fewer than half of all patients treated with 
hematopoietic stem cell transplants are cured. Despite 50 years of 
research and practice in hematopoietic stem cell transplantation, blood 
cancers still relapse, and patients still die or become severely 
disabled because the transplant regimens are so toxic. Many patients 
who might benefit never make it to the transplant stage because they 
are too sick or lack a suitable donor.
    Such limitations of even our most successful adult stem cell 
therapies for blood diseases drive me, as a medical research scientist, 
to seek improvements through stem cell research. I have been a student 
of the hematopoietic stem cell for 25 years, and I remain an ardent 
advocate for research on adult stem cells. But starting more than 
fifteen years ago, I began to explore a new approach to bone marrow 
transplant based on making blood stem cells from embryonic stem cells. 
I envisioned one day generating customized stem cells perfectly matched 
to my patients, thus bypassing the challenge of immune matching, 
eliminating the problems of donor shortages, and making transplants 
safer because they would be performed with a patient's own cells. 
Moreover, for patients with genetic diseases, this new approach offered 
potentially safer ways to repair gene defects and to return healthy 
cells to the patient. Indeed, we have succeeded in treating mice with 
genetic immune deficiency with this strategy, and we are making headway 
towards the goal of developing similar treatments with human cells.
    Opponents of embryonic stem cell research will argue that adult 
stem cells are more promising, that embryonic stem cells have yet to 
cure anyone, and that with iPS cells in hand, embryonic stem cells are 
no longer needed. By similar reasoning, why try to develop new classes 
of antibiotics? Let's just keep trying to improve penicillin. The only 
time I confront the argument that adult stem cells are superior to 
embryonic stem cells and should replace embryonic stem cells is at 
hearings like this. At scientific meetings, discoveries with adult and 
embryonic stem cells are discussed and debated as integrated and 
complementary aspects of cell and developmental biology, not as 
contestants on American Idol. In my opinion, such arguments are not 
sound scientific advice, but rather ideologically driven attempts to 
prohibit scientists from using embryonic stem cells to search for new 
cures. No matter how much progress is made with other forms of stem 
cells, embryonic stem cells will remain a vital research tool, and any 
expulsion of embryonic stem cells from the researcher's toolkit would 
gravely weaken stem cell research overall.
    Embryonic stem cells are valuable because they are pluripotent, 
that is, able to make any tissue in the human body, and can grow 
indefinitely in a petri dish. In contrast, adult stem cells show a 
restricted potential for generating cells of a given tissue, and are 
difficult to propagate in a petri dish and thus available in limited 
quantities. Not all tissues regenerate from adult stem cells, which is 
a major reason why we need embryonic stem cells. Indeed, in juvenile 
diabetes, there is little or no regeneration of the insulin-producing 
beta cells that have been destroyed by immune attack. We are 
technically capable of transplanting a whole pancreas or isolated 
pancreatic islets to replace beta cells, but there is a shortage of 
these organs for transplanting even the most severe diabetics. 
Consequently, embryonic stem cells are being developed by the 
biotechnology company Novocell as an alternate and more readily 
available source of beta cells for treatment of diabetes.
    Only 3 years ago, a new form of pluripotent stem cell was 
introduced into stem cell research, the induced pluripotent stem cell, 
popularly called the iPS cell. At the end of 2007, my lab was one of 
three worldwide to report the successful derivation of human iPS 
cells,\3\ and in 2008, my lab was the first to produce a repository of 
customized iPS cells from patients with a range of diseases like 
Parkinsons, diabetes, and immune deficiency.\4\ iPS cells share the 
defining features of embryonic stem cells-pluripotency and limitless 
growth, and one goal of stem cell research is to refine techniques for 
making iPS cells that are indistinguishable from embryonic stem cells. 
Thus, given that iPS cells exist, why is there a need for human 
embryonic stem cells, and what is the value of continued development of 
new human embryonic stem cell lines?
---------------------------------------------------------------------------
    \3\ Park, I.H., et al., Reprogramming of human somatic cells to 
pluripotency with defined factors. Nature, 2008. 451(7175): p. 141-6; 
online Dec. 23, 2007.
    \4\ Park, I.H., et al., Disease-specific induced pluripotent stem 
cells. Cell, 2008. 134(5): p. 877-86.
---------------------------------------------------------------------------
    First, it is important to note that the iPS breakthrough was 
founded upon the study of embryonic stem cells, and isolation of human 
iPS cells depended upon specific culture conditions for human, not 
mouse, embryonic stem cells. Today, human embryonic stem cells remain 
the gold standard against which our cultures of human iPS cells are 
compared. Human embryonic stem cells hold many more secrets, and no one 
can be sure where the next breakthrough will emerge.
    Second, it is not clear that even ideal iPS cell lines are 
identical in all respects to embryonic stem cells. My lab and that of 
Konrad Hochedlinger recently demonstrated that iPS cells tend to retain 
chemical modifications of their DNA reminiscent of their tissue of 
origin, so that when the iPS cells are differentiated in the petri 
dish, they reflect a preference to form the tissues from which they 
were derived.\5\-\6\ This so-called ``epigenetic memory'' 
dictates that iPS cells made from blood cells make better blood than 
iPS cells made from skin cells. We are working towards ways to erase 
these memories, but these data teach us that in practice, iPS cells 
harbor important differences from embryonic stem cells that influence 
their behavior and potential utility in research and therapy.
---------------------------------------------------------------------------
    \5\ Kim, K., et al., Epigenetic memory in induced pluripotent stem 
cells. Nature, 2010 online July 19.
    \6\ Polo, J.M., et al., Cell type of origin influences the 
molecular and functional properties of mouse induced pluripotent stem 
cells. Nat Biotechnol, 2010. 28(8): p. 848-55.
---------------------------------------------------------------------------
    Third, although iPS cells provide a flexible alternative to 
embryonic stem cells in modeling human diseases, not all diseases are 
readily modeled with iPS cells. One of the first diseases we attempted 
to model with human iPS cells was a fascinating but rare condition 
called Fanconi anemia that leaves kids with bone marrow failure and a 
predisposition to leukemia and various cancers. Despite repeated 
attempts, we have been unable to generate iPS cells from patients with 
Fanconi anemia, and last year the laboratory of Juan-Carlos Izpisua-
Belmonte published that Fanconi anemia cells were resistant to iPS 
generation.\7\ Mice that lack the same genes as human Fanconi patients 
do not develop the same marrow failure and leukemia of human patients. 
Thus, we turned instead to modeling Fanconi anemia by depleting the 
relevant genes from human embryonic stem cells, and then examining the 
effects on human blood formation in the petri dish. Using genetically 
modified human embryonic stem cells, we discovered that Fanconi anemia 
alters the earliest stages of human embryonic blood development, 
teaching us that the condition develops in utero, such that children 
are born with stem cell deficiency, a new insight for a condition 
thought to develop only later in childhood.\8\
---------------------------------------------------------------------------
    \7\ Raya, A., et al., Disease-corrected haematopoietic progenitors 
from Fanconi anaemia induced pluripotent stem cells. Nature, 2009. 
460(7251): p. 53-9.
    \8\ Tulpule, A., et al., Knockdown of Fanconi anemia genes in human 
embryonic stem cells reveals early developmental defects in the 
hematopoietic lineage. Blood, 2010. 115(17): p. 3453-62.
---------------------------------------------------------------------------
    Another example where human embryonic stem cells offer an advantage 
over iPS cells is in the study of Fragile X syndrome, the most common 
genetic cause of mental retardation. Fragile X is caused by a defect in 
the FMR1 gene, which is expressed early in human development, but in 
affected individuals becomes aberrantly silent in adult tissues, 
including nerve cells. My Israeli colleague, Nissim Benvenisty, had 
generated human embryonic stem cells from discarded embryos that 
carried the gene defect. When these embryonic stem cells were 
differentiated in the petri dish, the gene shut off, just as it does 
during human development. In collaboration with the Benvenisty lab, we 
asked what would happen to the FMR1 gene in iPS cells made from skin 
cells of Fragile X individuals. To our surprise, the gene remained 
silent in iPS cells, showing that Fragile X-iPS cells differed from 
Fragile X embryonic stem cells, with only the embryonic stem cells 
reflecting the dynamic FMR1 gene silencing observed in human 
development.\9\ For studying gene silencing in Fragile X, human 
embryonic stem cells provide a unique advantage.
---------------------------------------------------------------------------
    \9\ Urbach, A., et al., Differential modeling of fragile X syndrome 
by human embryonic stem cells and induced pluripotent stem cells. Cell 
Stem Cell, 2010. 6(5): p. 407-11.
---------------------------------------------------------------------------
    Finally, human embryonic stem cells remain valuable tools for 
research. There is still much to be learned about human embryonic stem 
cells, and about how stem cells derive from human embryos. Only 
recently have we learned that human embryonic stem cells are markedly 
different from mouse embryonic stem cells, and represent a distinct 
type of pluripotent stem cell. Only recently have we learned that 
deriving human embryonic stem cells in reduced oxygen conditions 
preserves two active X chromosomes, which is the natural embryonic 
state, leaving us to question whether any of the existing human 
embryonic stem cells have been derived in an optimal way.\10\ When we 
still have so much to learn, how can we conclude that embryonic stem 
cells are no longer needed?
---------------------------------------------------------------------------
    \10\ Lengner, C.J., et al., Derivation of pre-X inactivation human 
embryonic stem cells under physiological oxygen concentrations. Cell, 
2010. 141(5): p. 872-83.
---------------------------------------------------------------------------
    We are told that restrictions on Federal funding do not inhibit 
stem cell research because private philanthropy fills the gap. 
Realistically, however, research careers are built upon the 
architecture of Federal grant support. Investment by the NIH has made 
the United States the pre-eminent incubator for biomedical research, 
has produced American dominance in Nobel prizes in medicine, and has 
contributed directly to our robust biotechnology industry. Medical 
research is one of the chief sectors projecting job growth over the 
next decade, and one of the few areas of technological innovation where 
U.S. leadership remains largely uncontested. A loss of Federal funding 
threatens American competitiveness in stem cell research.
    Unfortunately, during the last decade prohibitions and restrictions 
on Federal funding for human embryonic stem cell research has greatly 
restricted progress and dissuaded numerous scientists from entering the 
field. President Bush allowed funding for a very restricted set of 
cells--in practice only a small handful--but prohibited funding for the 
more than 1,000 human embryonic stem cell lines generated since his 
policy was enacted on August 9, 2001. Many of these embryonic stem cell 
lines have important advantages for medical research, like carrying the 
precise gene defects responsible for human disease. President Obama's 
policy has expanded access to many more lines and has succeeded in 
bringing many dozens of additional laboratories into the field, as 
evidenced by the new grants submitted or approved for research in the 
last year.
    Against this backdrop of rising enthusiasm after nearly a decade of 
frustration for patients, their families, their physicians, and the 
research community, the announcement of the injunction against Federal 
funding came as a major blow. I was justifiably confused by what the 
injunction meant for our research program, which depends heavily on 
Federal grant dollars, and personally, I was deeply discouraged and 
worried for the future of human embryonic stem cell research.
    Several cases illustrate the immediate harm to our research program 
and the potential harm to the careers of young scientists by the 
current confusion. A doctoral student in my lab has just completed 
nearly a year of work mastering a protocol for generating red blood 
cells from human embryonic stem and iPS cells, a critical step in her 
research on sickle cell anemia. Because of variability among the iPS 
lines, human embryonic stem cells are essential for her studies, and 
she has just started to have success with the H1 line of human 
embryonic stem cells. However, because she is being paid by Federal 
dollars and the future prospects are so uncertain, she has abandoned 
the use of human embryonic stem cells, and is instead restricting her 
efforts to iPS cells that may give sub-optimal red blood cell 
production. Such a compromise--driven by politics and not science--is 
deeply troubling. Several other scientists in my lab have altered their 
projects out of concern for a loss in Federal funding. Two scientists 
being funded on Federal training grants abandoned plans to test human 
embryonic stem cells for their response to a unique cocktail of growth 
factors that had stimulated blood stem cell formation from mouse 
embryonic stem cells. Moreover, I face losing my largest NIH grant, 
which is aimed at defining the precise similarities and differences 
between embryonic stem and iPS cells. I have been scrambling to come up 
with private funding so that I don't have to lay anyone off. I wrote to 
my seven co-investigators on this project and warned them not to expect 
funding for the second year, which would stop cold major new research 
collaborations that have already proven remarkably productive. 
Scientific research is challenging enough without adding the 
uncertainty and fickle nature of Federal support for one's research to 
the task.
    With the recent upheavals, scientists have again been reminded that 
human embryonic stem cell research is on fragile and fickle footing. 
The cloud that hangs over the field saps enthusiasm for planning a 
long-term program of NIH grant-funded human embryonic stem cell 
research, which is the bedrock of most research careers. Younger 
researchers are discouraged from entering the field, while established 
researchers like myself are spending a disproportionate amount of time 
on regulatory compliance, legal interpretation, program management, and 
external fundraising. With the economy in turmoil, private funding for 
stem cell research has become scarce. Ambiguity about Federal policy 
itself has a negative impact that extends beyond the practical 
restrictions of legislation. Having devoted the last 25 years of my 
career to aspects of adult and embryonic stem cell biology, I am 
convinced that human embryonic stem cells are critical to a multi-
faceted portfolio of NIH stem cell research, and in the long run will 
save lives. New legislation is needed to sustain the momentum of human 
embryonic stem cell research in the United States, and to allow 
scientists--not politicians and judges--to determine which research 
priorities to pursue.

    Senator Harkin. Now we turn to Dr. Morrison. Dr. Morrison, 
welcome. Proceed.

STATEMENT OF SEAN J. MORRISON, Ph.D., DIRECTOR, CENTER 
            FOR STEM CELL BIOLOGY, UNIVERSITY OF 
            MICHIGAN, LIFE SCIENCE INSTITUTE, ANN 
            ARBOR, MICHIGAN
    Dr. Morrison. Thank you, Senator Harkin, for the 
opportunity to testify today.
    I have spent my entire career doing stem cell research, 
almost exclusively adult stem cell research. The research in my 
lab has won a number of awards, including a Presidential Early 
Career Award from President Bush in 2003.
    Nonetheless, like nearly all leading stem cell researchers, 
I believe the Federal Government must support all forms of stem 
cell research, including human embryonic stem cells. We simply 
do not yet know what kinds of stem cells will yield the 
breakthroughs of the future and must pursue all forms of stem 
cell research to develop new therapies sooner rather than 
later.
    Stem cell scientists do not cluster into adult versus 
embryonic camps. This framing of the debate comes from 
political lobbyists. I interact regularly with hundreds of the 
leading stem cell scientists throughout the world, and 
virtually all of them believe that research must continue with 
all types of stem cells for the reasons George just 
articulated.
    Stem cell research is a remarkably fast-moving field that 
has taken a series of unexpected twists and turns over the past 
several years. There is no point in the last 10 years where we 
could have predicted even 2 years down the road where the field 
would be. Yet, at every point there have been people who 
believe they could predict the future and who could tell us 
which avenues of research should be abandoned. But until the 
research is done, we do not know what the answers will be.
    Think about the arguments that opponents have made as 
alternatives to embryonic stem cell research.
    First, they suggested that umbilical cord blood cells could 
replace embryonic stem cells. Yet, my lab has studied cord 
blood, and I can tell you that there was never any 
scientifically plausible basis for the argument that cord blood 
cells could do what embryonic stem cells can do. And you no 
longer hear much about cord blood as an alternative.
    Instead, they subsequently suggested that amniotic cells 
could replace embryonic stem cells, but those cells are 
biologically different from embryonic stem cells and, again, 
were never a plausible alternative. And again, you never hear 
about those cells anymore.
    Then opponents circulated lists of more than 70 diseases 
they claim could be cured with adult stem cells. What they do 
not tell you is that only diseases of the blood-forming system 
are routinely treated with adult stem cells and that many of 
the other ``treatments,'' in quotation marks, they cite are 
highly speculative, are often not based on sound science, and 
are prohibited from being sold to patients in this country by 
our FDA.
    The reality is that many types of stem cells are likely to 
yield scientific advances and potentially new therapies. And it 
would be foolish to place all of our bets on certain stem cells 
at such an early stage in the development of this field.
    For this reason, the International Society for Stem Cell 
Research has repeatedly recommended that all forms of stem cell 
research must be pursued and that patients should be cautious 
about claims regarding unproven adult stem cell therapies that 
are offered overseas. Where would we be right now if you had 
taken the advice of opponents of embryonic stem cell research 
and directed the NIH to focus their funding on umbilical cord 
blood cells or on amniotic cells? Promising research would have 
been abandoned in favor of the alternative du jour, sacrificing 
scientific progress and the opportunity to develop new 
therapies.
    The award my lab received from President Bush was for our 
work studying stem cells that give rise to the peripheral 
nervous system. One of the things we discovered is that a birth 
defect called Hirschsprung disease is caused by defects in the 
function of these neural stem cells during fetal development. 
In kids with Hirschsprung disease, the neural stem cells fail 
to migrate into part of the intestine, rendering that segment 
of the intestine nonfunctional. Our work suggested that we 
might be able to bypass that defect by transplanting neural 
stem cells into the nonfunctional portion of the gut. The 
problem is that neural stem cells with the right properties 
only exist during fetal development. So we decided to generate 
those cells by deriving them from human embryonic stem cells.
    Now, I want to emphasize this point because for the therapy 
we want to use a tissue-specific stem cell, a cell that in the 
newspaper is generally referred to as an adult stem cell. And 
yet, we have to obtain it from embryonic stem cells. So this 
illustrates why it is scientifically meaningless to frame this 
debate as a choice between adult and embryonic stem cells 
because we sometimes need embryonic stem cells to derive the 
adult cells that we want to use in the therapy.
    So this research in my lab is funded by the NIH, but it has 
suffered from repeated delays. First, the grant was delayed 
while NIH put in place its new embryonic stem cell policy. Then 
we received the grant but we were unable to spend the money 
until NIH had the opportunity to review and approve new stem 
cell lines for funding. And finally, we were able to start the 
research, but just 8 months later, the Federal injunction was 
issued.
    In the first few days after the injunction, I told my lab 
that if our funding were cut off, we would abandon our work on 
Hirschsprung disease. I have with me today Jack Mosher from my 
laboratory. Jack, you might want to stand up for a second. Jack 
is the guy in my lab who does this work. The project I have 
been telling you about is Jack's work, and his salary comes 
exclusively from this grant. Jack has dedicated the last 9 
years of his life to studying peripheral nervous system 
development, culminating in this project, attempting to 
translate the basic science that we have done to the benefit of 
patients. Yet, in those early days after the injunction, Jack 
did not know whether his work would survive the injunction, 
whether he would still have a salary, or what would happen to 
his career.

                           PREPARED STATEMENT

    So I would just sum up by saying that American science is 
the envy of the world because it is a meritocracy in which 
there is fierce competition to fund the best ideas. If we 
accept the principle that those who are not judged to have the 
best ideas can obtain judicial relief that blocks funding of 
the best ideas to allow the lesser ideas to compete, this will 
erode the very heart of American competitiveness. We owe more 
to the patients suffering from incurable diseases. We owe it to 
them to support all forms of stem cell research so that no 
matter where the science leads and where the cures come from, 
we can follow the most promising avenues of discovery.
    So I would urge you to clarify the Dickey-Wicker amendment 
so there can be no question regarding Congress' intent to fund 
the most meritorious science.
    Senator Harkin. Thank you very much, Dr. Morrison.
    [The statement follows:]

                 Prepared Statement of Sean J. Morrison

    My name is Sean Morrison and I'd like to begin by thanking Senator 
Harkin and the members of the subcommittee for inviting me to testify. 
By way of introduction, I am the Director of the University of Michigan 
Center for Stem Cell Biology, where I am also the Henry Sewall 
Professor of Medicine, a Professor in the Life Sciences Institute, and 
a Professor of Cell and Developmental Biology. I am also an 
Investigator of the Howard Hughes Medical Institute, a Director of the 
International Society for Stem Cell Research, and a member of the 
American Society for Cell Biology Public Policy Committee.
    I have spent my entire career doing stem cell research, almost 
exclusively adult stem cell research. The adult stem cell research in 
my laboratory has won many awards, including a Presidential Early 
Career Award from President Bush in 2003. Nonetheless, I'm here today 
to tell you that like nearly all leading stem cell researchers, I 
believe that the Federal Government must support all forms of stem cell 
research, including human embryonic stem cell research. We simply do 
not yet know what kinds of stem cells will yield the scientific 
breakthroughs of the future or what kinds of stem cells will yield new 
treatments for disease. Therefore, we must pursue all forms of stem 
cell research in order to have the greatest chance of developing new 
therapies sooner rather than later.
    Stem cell scientists do not cluster into ``adult'' versus 
``embryonic'' camps--this framing of the debate comes from political 
lobbyists. I interact regularly with hundreds of leading stem cell 
scientists from all over the world and virtually all of them believe 
that research should continue with all types of stem cells.
    Stem cell research is a remarkably fast-moving field that has taken 
a series of unexpected twists and turns over the past several years. 
There is no point over the past 10 years during which we could have 
predicted where the field would be, even two years down the road. Yet, 
at every point there have been people who believed that they could 
predict the future and could tell us which avenues of research should 
be abandoned. But until the research is done, we don't know what the 
answers will be.
    Think about the alternatives that have been offered by opponents of 
embryonic stem cell research.
  --First, they suggested that umbilical cord blood could replace 
        embryonic stem cells. Yet as somebody whose laboratory has 
        studied umbilical cord blood I can tell you that there was 
        never any scientifically plausible basis for suggesting that 
        cord blood cells could replace embryonic stem cells. The 
        opponents of embryonic stem cell research never talk about cord 
        blood anymore.
  --Instead, they subsequently suggested that amniotic cells identified 
        by Dr. Anthony Atala could replace embryonic stem cells. But 
        those cells are biologically different from embryonic stem 
        cells and were never a plausible alternative. Even Dr. Atala 
        has gone on record stating they are not an alternative to 
        embryonic stem cells. Again, you never hear about those cells 
        anymore.
  --Then, opponents of embryonic stem cell research circulated lists of 
        more than 70 diseases they claimed could be cured with adult 
        stem cells. What they don't tell you is that only diseases of 
        the blood-forming system are routinely treated with adult stem 
        cells, and that many of the other ``treatments'' they cite are 
        highly speculative and often not based upon sound science.
  --Most recently, opponents of embryonic stem cell research have 
        suggested that reprogrammed adult cells, so-called iPS cells, 
        should be studied instead of embryonic stem cells. While these 
        reprogrammed cells are very promising, George Daley and others 
        have recently shown that their properties are somewhat 
        different from embryonic stem cells.
    The reality is that all of these types of stem cells are likely to 
yield scientific advances, and potentially new therapies, but it would 
be foolish to place all of our bets on a single type of stem cell at 
such an early stage in the development of this field. For this reason 
the International Society for Stem Cell Research, a society 
representing thousands of stem cell scientists all over the world, has 
repeatedly recommended that all forms of stem cell research must be 
pursued, including adult and embryonic stem cells, and that patients 
should be cautious about claims regarding unproven adult stem cell 
therapies.
    Where would we be right now if you had taken the advice of 
opponents of embryonic stem cell research and directed the National 
Institutes of Health (NIH) to focus their funding on umbilical cord 
blood cells or amniotic cells? Promising research would have been 
abandoned in favor of the alternative du jour, sacrificing scientific 
progress and the opportunity to develop new therapies. We remain unable 
to predict the future. So blocking Federal funding for embryonic stem 
cell research at this juncture will certainly block scientific progress 
and will likely delay the search for new therapies.
    The Presidential Early Career Award that my lab received was for 
our work studying the stem cells that give rise to the peripheral 
nervous system. One of the things we discovered is that a birth defect 
called Hirschsprung disease is caused by defects in the function of 
these peripheral nervous system stem cells during fetal development. 
Hirschsprung disease affects 1 in 5,000 newborns and is caused by a 
defect in the development of the portion of the peripheral nervous 
system that regulates intestinal function. In kids that have 
Hirschsprung disease, the neural stem cells fail to migrate into the 
large intestine, rendering that segment of intestine nonfunctional 
because of the lack of nervous system in that segment. Surgery to 
remove the nonfunctional segment of intestine can save these kids' 
lives, but for many of these kids, their guts never quite work right, 
leading to life-long problems.
    We figured that if Hirschsprung disease is caused by a failure of 
stem cells to migrate into the large intestine, that we might be able 
to by-pass this migratory defect by transplanting stem cells into the 
nonfunctional portion of gut, and that this cell therapy might improve 
the treatment of kids with Hirschsprung disease. The problem is that 
neural stem cells with the right properties to correctly innervate the 
intestines only exist during fetal development. So where would we get 
the neural stem cells for therapy? We don't want to use aborted human 
fetal tissue. George Daley's recent results have raised the concern 
that if reprogrammed adult cells are not generated from peripheral 
nervous system stem cells that they might have difficulty making the 
correct types of neural cells to regulate intestinal function. Thus, 
the most prudent way of generating peripheral nervous system stem cells 
is by deriving them from human embryonic stem cells.
    I want to emphasize this point--we wish to use tissue-specific stem 
cells (often described as ``adult'' stem cells in the newspaper) for 
the therapy, but we will obtain them from embryonic stem cells. This 
illustrates why it is scientifically meaningless to frame this debate 
as a choice between adult and embryonic stem cells. We sometimes need 
embryonic stem cells to generate adult cell types for therapy.
    We are funded by the National Institutes of Health (NIH) to try to 
develop a cell therapy for Hirschsprung disease, using human embryonic 
stem cells to derive neural stem cells for transplantation. But our 
research has suffered from repeated delays. First, the awarding of this 
grant was delayed while NIH put in place its new embryonic stem cell 
research policy, after the repeal of the Bush administration policy. 
After the new NIH policy was established, we received the grant, but 
were unable to spend any of the money until NIH had the opportunity to 
review and approve embryonic stem cell lines for funding. Finally, 
lines were approved and we were able to start the research, then just 8 
months later the injunction was issued.
    In the first few days after the injunction was issued none of us 
knew exactly what research would be blocked, or how the ruling would be 
interpreted by NIH. During this period, I told my lab that if our 
funding were cut off as a result of the injunction, and if the 
injunction could not soon be lifted, that we would abandon our work on 
Hirschsprung disease. I have with me today Jack Mosher from my 
laboratory. The project I have been telling you about is Jack's work, 
and his salary comes almost exclusively from this grant.
    Jack completed his undergraduate work at Allegheny College in 
Pennsylvania, then a Ph.D at the University of North Carolina. He came 
to my lab in 2001 as a postdoctoral fellow and was ultimately promoted 
into a faculty position at the University of Michigan. He has dedicated 
the last 9 years of his life to studying peripheral nervous system 
development, culminating in this project, trying to translate our 
results to help patients. Yet in those early days after the injunction 
he did not know whether his work would survive the injunction, whether 
he would still have a salary, or what would happen to his career. Since 
the injunction, many students, postdoctoral fellows, and junior faculty 
have had similar conversations in scores of laboratories across the 
country.
    It turns out that because of the timing of our annual review, we 
received our second year of funding just before the injunction. As a 
result, our funding was not interrupted. But this is not the way in 
which funding decisions for medical research should be determined. 
American science is the envy of the world because it is a meritocracy 
in which there is fierce competition to fund the best ideas. As a 
consequence, American scientists lead the world in virtually every 
measure of scientific impact and America is the world's engine of 
scientific discovery.
    If we accept the idea that those who do not have the best ideas can 
obtain judicial relief that blocks NIH funding for the best ideas, to 
help the lesser ideas compete, this will erode the very heart of 
American competitiveness.
    We don't know yet whether the cell therapy we are attempting to 
develop will work, or whether it will ultimately be performed with 
embryonic stem cells, reprogrammed adult cells, or other cells. That's 
why they call it research. The point is that we're never going to find 
out until we do the research. Yet instead of devoting ourselves to 
trying to make a difference for kids with Hirschsprung disease, Jack 
and I now find ourselves talking about the uncertain future of 
embryonic stem cell research, whether legislative and judicial delays 
will continue on-and-off indefinitely, and whether his career would be 
better served by working in a different area.
    I'd like to leave you with one last story. Opponents of embryonic 
stem cell research frequently repeat the argument that this research is 
less promising than adult stem cell research because adult stem cells 
are already used to treat patients whereas embryonic stem cells are 
not. The problem is that adult stem cells have been studied for decades 
while we have only had human embryonic stem cells since 1998, 12 years. 
So let's examine this argument for a moment.
    The adult stem cell therapy that is routinely used clinically is 
blood-forming stem cell transplantation (formerly known as bone marrow 
transplantation) to restore the blood forming systems of patients after 
cancer therapy or to treat various diseases of the blood-forming 
system. This is indeed a great success: while it's not perfect it does 
save thousands of lives each year. What did it take to get to this 
point?
    After many years of research, the first bone marrow transplant 
among unrelated patients was attempted in 1955 by Donnall Thomas. All 
of the patients died. Dr. Thomas went back to the laboratory to figure 
out why he couldn't just randomly transplant bone marrow cells from one 
patient into another. He discovered that donor and recipient had to be 
matched, so that their immune systems didn't attack each other. The 
first successful bone marrow transplant between an unrelated donor and 
recipient was performed in 1969--14 years later. Thus if we were to 
take the advice of opponents of embryonic stem cell research, and 
abandon lines of research that do not lead to cures within 12 years, 
none of the adult stem cell therapies that they exalt would exist today 
and Donnall Thomas would never have won the Nobel prize.
    Science takes time, and the path to cures is uncertain and fraught 
with setbacks. American science is the envy of the world because it has 
fostered creativity and innovation, amidst constant competition and 
peer review to invest the public's limited resources in the most 
promising ideas. We owe nothing less to the patients suffering from 
incurable diseases. For this reason, we must support all forms of stem 
cell research so that no matter where the cures come from, we can get 
there sooner rather than later. I urge you to clarify the Dickey-Wicker 
amendment so that there can be no question regarding Congress' intent 
to fund the most meritorious science.

    Senator Harkin. Dr. Mosher, welcome, and thank you. I may 
even have a question for you when we get to the questions here.
    Now we turn to Dr. Peduzzi Nelson and welcome and please 
proceed.

STATEMENT OF JEAN PEDUZZI NELSON, Ph.D., ASSOCIATE 
            PROFESSOR, DEPARTMENT OF ANATOMY AND CELL 
            BIOLOGY, WAYNE STATE UNIVERSITY SCHOOL OF 
            MEDICINE, DETROIT, MICHIGAN
    Dr. Peduzzi Nelson. Thank you very much, Chairman Harkin, 
for the opportunity to present this information today.
    I am a translational neuroscientist from Wayne State 
University, and today I am--there are two types of stem cells, 
embryonic stem cells from embryos and adult stem cells. Today I 
am going to talk about adult stem cells, and we use the term 
``adult stem cells'' to mean not just stem cells from adults, 
but also from children, umbilical cord, from blood.
    What you have to understand is that the first human adult 
stem cell was isolated in 1992. Now, we do have a long history 
of doing bone marrow transplants, which contain stem cells, but 
adult stem cells are actually, in terms of looking at other 
diseases and injuries, a new field. And it was only in the late 
1990s did we realize the potential for other diseases other 
than cancer and various blood disorders.
    Yes, most of the Federal funding does go to adult stem 
cells, but the majority of that goes to old but very important 
studies in terms of treating cancer and blood diseases. The big 
disadvantage for adult stem cells is often there is not 
intellectual property. So the biotech industry that has a much 
larger budget for research than Federal funding is not 
interested in most cases in adult stem cells. And we only have 
a limited amount of Federal funding available.
    And where we are in adult stem cells--I am sure the members 
have seen previously that there are some examples of people 
being treated with adult stem cells where there is considerable 
improvement. But the research is going from isolated incidents, 
and I am going to present clinical trial data in respectable 
journals where we need to move through clinical trials to 
standard of care. And to move from a basic science study is 
relatively inexpensive, several hundred thousand dollars, but 
for each clinical trial, you need billions of dollars. So we 
need a lot more Federal support to move forward with adult stem 
cells.
    This is an example of one of the patients. Well, this would 
have been an example of one of the patients that was treated, a 
quadriplegic that was treated, using a procedure that was 
developed in Portugal by Dr. Carlos Lima and his team. And this 
is a picture. I think we do have a poster of this gentleman 
that I met several years ago. And he was treated with his own 
adult stem cells 2 years after his injury. And 2 years after 
his injury, this gentleman is now shown standing up without 
anyone supporting him. He is not waving, but he was in the 
video. And with only braces on his--a foot ankle brace. So he 
is standing up maintaining balance, and he can now walk with a 
walker. Amazing, a quadriplegic walking with a walker without 
assistance, and this is the progress.
    But this is not an isolated incident. If you look at the 
two publications that have been published in peer-reviewed 
journals----
    Senator Harkin. Dr. Peduzzi, could I just interrupt for a 
quick question?
    Dr. Peduzzi Nelson. Yes.
    Senator Harkin. Was he treated with what I would refer to 
as autologous stem cells?
    Dr. Peduzzi Nelson. Yes, he was. And these autologous stem 
cells were obtained from inside of his nose and used to treat 
his spinal cord injury.
    But this, as I said, is not an isolated incident. There are 
peer-reviewed publications and a larger number of patients, and 
I would love the opportunity to bring this forward in the 
United States after completing a safety study so patients do 
not have to go to other countries to have this done.
    Another example. This is Doug Rice, and he had several 
heart attacks and had chronic heart failure. And he was told in 
2002 that he had 2 years to live. He went to another country 
and had a treatment done, and he is alive and doing well. At 
the time he had the procedure done, he could barely walk. But 
this is also not an isolated example.
    This is a published, peer-reviewed article of a study where 
they used 191 patients who had adult stem cells and compared to 
200 patients with similar heart conditions. And the treated 
patients lived longer and also could exercise more.
    Now, I have to move to a somewhat gross picture, and I 
apologize for that. But this is corneal blindness, and this is 
the second leading cause of blindness in the country. And on 
the left side, it shows eyes of patients who had several 
surgeries that were unsuccessful and were blind in that eye. 
But using adult stem cells from their other eye, this shows the 
results several years later. This particular one was 112 
patients, and more than 75 percent of the time it was 
successful. And many of the patients regained normal vision in 
their eye.
    And let me just go to another example. This is the study, 
the published study, that was published of these 112 patients 
in the New England Journal of Medicine.
    I will go to one more patient. I am showing these sort of 
poster patients or poster examples, but now they are supported 
by results from clinical trials. In the middle between his 
parents is Joe Davis, and he had very severe sickle cell anemia 
and his parents were told that Joe might not live to his teens. 
So he had the procedure using his brother's umbilical cord 
blood cells, and Joe is absolutely doing fine right now and has 
no sickle cell symptoms.
    There have been two published studies for sickle cell, 
which is a very painful condition. This first study, 6 out of 7 
patients no longer have sickle cell symptoms.
    And another study--this particular study was by NIH 
scientists and published in the New England Journal of Medicine 
in 10 adults with sickle cell anemia. Most of these patients--9 
out of 10 of these patients--no longer had sickle cell 
symptoms.
    The last patient that I would like to show is Barry Goudy 
and he had MS. He went to Northwestern Memorial Hospital. His 
symptoms of MS have disappeared. And he was part of a larger 
study that was published in a peer-reviewed journal, in Lancet, 
and these patients showed significant functional improvement 
and no one got worse in this degenerative disease.
    Senator Harkin. Dr. Peduzzi Nelson, I have to ask you to 
wrap up. I would like to get to the last--we just have some 
more votes that just----

                           PREPARED STATEMENT

    Dr. Peduzzi Nelson. Okay. This is just another study 
supporting that. I will not talk about the amazing results in 
newly diagnosed juvenile diabetes in JAMA.
    But I would just like to conclude that we need more Federal 
funding. We need more NIH funding so patients do not have to go 
to other countries and so these amazing results that I 
presented can go to clinical trials and become standard of care 
for U.S. patients that need their support.
    Senator Harkin. Thank you very much, Dr. Peduzzi Nelson.
    [The statement follows:]

               Prepared Statement of Jean Peduzzi Nelson

    Thank you Chairman Harkin, Senator Cochran, and distinguished 
subcommittee members for the opportunity to present this information to 
you today. My name is Jean Peduzzi Nelson from Wayne State University. 
Please note that the testimony I am giving today is my own opinion and 
not necessarily that of the university. I am a translational 
neuroscientist who is working to bring using one's own olfactory 
mucosal adult stem cells for spinal cord injury, head injury, and 
radiotherapy damage.
    There are two major categories of stem cells: embryonic and adult. 
Human embryonic stem cells are derived from human embryos and remain 
controversial. I want to focus my comments on the science of adult stem 
cells that are treating patients for many diseases. This second 
category of stem cells can be obtained from adult tissues, as well as 
tissues from children. For my purposes, I will use ``adult stem cells'' 
to refer to these as well as stem cells from umbilical cord blood.
    I wanted to share with you pictures of some brave pioneers who 
first explored the potential of adult stem cell treatment. The progress 
of adult stem cells has gone so far beyond these particular patients to 
long-term follow-up results of numerous patients in peer-reviewed 
published clinical trials.
    Stem cells are cells that can generate lots of cells and, under the 
right conditions, become one of the many cell types in the body. Adult 
stem cells are stem cells obtained from adults, children, even infants 
and umbilical cord after birth. These include cells from the bone 
marrow, nose, fat tissue, umbilical cord, and other places. The great 
thing about these cells is that a person's own cells can be used which 
eliminates the problem of immune rejection and tumor formation 
sometimes observed with other types of stem cells. Adult stem cells are 
the best stem cells to replace lost or damaged cells in our bodies.
    The financial challenge with adult stem cells is that usually when 
you use your own cells, there is no intellectual property or patents. 
So, the biotech industry that invests billions in research often does 
not fund this research.\1\ Millions of dollars are needed to complete 
each clinical trial so all patients can benefit from a treatment, not 
the lucky few, and so that billions can be saved in healthcare costs. 
The National Institute of Health (NIH) has developed new programs to 
encourage translational research and clinical trials, but has a much 
smaller budget than private industry.\2\ Much of the funding for adult 
stem cells by NIH is directed at older, but important uses of bone 
marrow stem cells that were developed in the 1950s and 1960s for 
leukemia and other cancers. While bone marrow transplants have been 
used in patients for years, the successful isolation and 
characterization of adult stem cells is a very recent science. The 
first mouse adult stem cell was successfully isolated and purified in 
the laboratory in 1983.\3\ The first human adult stem cell was first 
successfully isolated and characterized in the laboratory in 1924.\4\ 
New uses of adult stem cells for other diseases and injuries only 
started in the 1990s, but have already reached patients with various 
diseases and injuries as I will demonstrate.
---------------------------------------------------------------------------
    \1\ ``In 2004, the top twenty companies spent a combined total of 
over $56 billion on research and development.'' York University (2008, 
January 7). Big Pharma Spends More On Advertising Than Research And 
Development, Study Finds. ScienceDaily. Retrieved September 10, 2010, 
from http://www.sciencedaily.com-/releases/2008/01/080105140107.htm.
    \2\ ``. . . growth in the National Institutes of Health (NIH) 
budget would slow sharply to just 2.7 percent in fiscal year 2004 from 
just-approved fiscal year 2003 level, to $27.9 billion.'' NIH Budget 
Growth Slows to 2 Percent in fiscal year 2004, AAAS R&D Funding Update 
on R&D in the fiscal year 2004 NIH Budget--REVISED AAAS Report XXVIII: 
Research and Development fiscal year 2004, http://www.aaas.org/spp/rd/
nih04p.pdf.
    \3\ Spangrude GJ, et al., Purification and characterization of 
mouse hematopoietic stem cells, Science 241, 58 (1988).
    \4\ Baum CM, et al., Isolation of a candidate human hematopoietic 
stem-cell population, Proc. Natl. Acad. Sci. USA 89, 2804 (1992).
---------------------------------------------------------------------------
    I would like to tell you about five patients who have been helped 
by adult stem cells. These patients were either part of a clinical 
trial, and their results are now published in a peer-reviewed journal, 
or sometimes a similar procedure was done in a clinical trial that is 
now published.
    The first patient is Silvio who I met several years ago. I have 
been working with a group in Portugal led by Dr. Carlos 
Lima.\5\-\6\ Dr. Lima, Dr. Pratas-Vital, Dr. Escada, Dr. 
Capucho, and Dr. Hasse-Ferreira have been using a person's own tissue 
from inside of the nose as a way of delivering adult stem cells. Silvio 
had a spinal cord injury at the base of his neck [cervical level 6/7, 
American Spinal Injury Association Impairment Scale (AIS) A, complete 
injury. Grade A is considered the worst, which indicates a ``complete'' 
spinal cord injury where no motor or sensory function is preserved in 
the sacral segments S4-S5.]. Silvio was left with no movement of his 
legs and minimal movement of his fingers. At 2 years after injury, he 
received his own adult stem cells and partial scar removal after 
intensive rehab failed to lead to an improvement.
---------------------------------------------------------------------------
    \5\ Lima, C., P. Escada, J. Pratas-Vital, C. Branco, C.A. 
Arcangeli, G. Lazzeri, C.A.S. Maia, C. Capucho, A. Hasse-Ferreira, and 
J.D. Peduzzi (2010) Olfactory Mucosal Autografts and Rehabilitation for 
Chronic Traumatic Spinal Cord Injury. Neurorehab & Neural Repair 24:10-
22.
    \6\ Lima, C., J. Pratas-Vital, P. Escada, A. Hasse-Ferreira, C. 
Capucho and J.D. Peduzzi. Olfactory mucosa autografts in human spinal 
cord injury: a pilot clinical study, J Spinal Cord Medicine, 29(3):191-
203, 2006.




    Today he can maintain standing position and wave without help. With 
a walker and short braces, he can walk more than 30 feet without anyone 
helping him. He can now move his fingers, which he could not do before. 
Because he was in a wheelchair for 2 years before treatment and could 
only move the chair using his wrists, a special rehab program called 
BIONT (brain initiated non-robotic/non-weight supported training) was 
used at Centro Giusto in Italy so he could learn to walk again. Dr. 
Arcangeli and Dr. Lazzeri have developed an effective rehab program 
that, when combined with adult stem cells, helps patients recover. 
BIONT therapy is being used on some U.S. patients who had this 
procedure in Portugal at Walk the Line in Detroit. With NIH and/or the 
Department of Defense (DOD) I would like to bring olfactory mucosal 
stem cell treatment to the people in the United States.
    This is much more remarkable than a treatment of an acute spinal 
cord injury within the first few weeks after injury. More than 15 
percent of the patients who are American Spinal Injury Association 
Impairment Scale (AIS) grade A improve in their classification in the 
first year after injury.\7\ If a treatment is given acutely or 
subacutely, it is difficult to separate normal recovery and effects of 
a treatment unless a large number of patients are enrolled in the 
clinical trial and randomly assigned to treatment or control. If a 
treatment is given at 1 year or greater after spinal cord injury, only 
5.6 percent of AIS A (32/571 patients) improve in grade from year 1 to 
year 5 after spinal cord injury.\8\
---------------------------------------------------------------------------
    \7\ Marino RJ, Ditunno JF Jr, Donovan WH, Maynard F Jr. Neurologic 
recovery after traumatic spinal cord injury: data from the Model Spinal 
Cord Injury Systems. Arch Phys Med Rehabil. 1999;80(11):1391-6.
    \8\ Kirshblum S, Millis S, McKinley W, Tulsky D. Late neurologic 
recovery after traumatic spinal cord injury. Arch Phys Med Rehabil 
2004;85:1811-7.
---------------------------------------------------------------------------
    Silvio is not an isolated case. Here are the two peer-reviewed 
publications from the Journal of Spinal Cord Medicine and 
Neurorehabilitation and Neural Repair which reveal that more than half 
of AIS A patients improved in grade compared to the normal 5 percent 
without treatment. When the adult stem cells are combined with an 
effective rehab program, 12/13 AIS A improved in AIS grade and all of 
the patients regained some muscle movement in their legs. These 
findings were documented with EMG and SSEP recordings.
    The next picture is Doug Rice who was told in 1998 that he had 2 
years to live due to chronic heart failure after multiple heart 
attacks. At that time he could hardly walk. He did not qualify for any 
U.S. clinical trials, so he went to Thailand to have a treatment with 
adult stem cells. The cells were sent to a company in Israel where the 
cells were purified and allowed to multiply, then sent back to Thailand 
for injection. Since that time, he has more energy and is enjoying 
life. However, this is also not an isolated incident. This year an 
article was published in the European Journal of Heart Failure 
reporting the followup of 191 patients who received adult stem cells 
from their own bone marrow compared to 200 patients with comparable 
symptoms.\9\ These adult stem cell treated patients lived longer and 
had a greater capacity to do exercises. Their heart functioned much 
better based on a large number of tests (left ventricular ejection 
fraction, cardiac index, oxygen uptake, and left ventricle 
contractility). This report of the STAR-heart study provides the 
controlled clinical trial data, and new trials are now proceeding in 
the United States.
---------------------------------------------------------------------------
    \9\ Strauer BE, Yousef M, Schannwell CM. The acute and long-term 
effects of intracoronary stem cell transplantation in 191 patients with 
chronic heARt failure: the STAR-heart study. Europ. J Heart Failure 
(2010)12:721-29.




    I have to apologize for the next picture. It isn't a photo of a 
single patient, but somewhat gross pictures of the eyes of three 
patients with corneal blindness from an article just published in the 
New England Journal of Medicine.\10\ Corneal disease is the second 
leading cause of blindness after cataracts in the world.\11\ Corneal 
transplants are commonly used, but the transplants are rejected in 
about 20 percent of the cases.\12\ On the left are pictures of the eyes 
of patients who had severe burns or damage to their eye and suffered 
from corneal blindness. These patients had surgery on their eyes, but 
these surgeries did not help. Several years later, adult stem cells 
were removed from the opposite eye and implanted in the damaged eye. 
The results of the adult stem cell transplant are shown on the right 
several years after the procedure. The patients went from barely being 
able to see hand movements to normal sight in these eyes. This 
procedure was successful in more than 75 percent of the 112 patients. 
Some of these patients were followed for 10 years. We need more 
clinical studies in the United States to treat U.S. patients with 
corneal blindness.
---------------------------------------------------------------------------
    \10\ Rama, P, S. Matuska, G. Paganoni, A. Spinelli, M. De Luca and 
G. Pellegrini. Limbal Stem-Cell Therapy and Long-Term Corneal 
Regeneration N Engl J Med 2010; 363:147-55.
    \11\ Whitcher, JP, M. Srinivasan, & MP Upadhyay. Corneal blindness: 
a global perspective. Bulletin of the World Health Organization, 2001, 
79 (3):214-221.
    \12\ Facts About The Cornea and Corneal Disease, NIH, National Eye 
Institute http://www.nei.nih.gov/health/cornealdisease/#4.




    The next patient is Joe Davis, Jr. Joe is the boy between his mom 
and dad; he was born with severe sickle cell anemia. Sickle cell anemia 
is a blood disease that affects 1/500 African Americans. The doctors 
thought that Joe might not live to see his teens. When Joe was 2 years 
old in 2002, he received a transplant of stem cells from his younger 
brother's umbilical cord. Joe no longer has sickle cell anemia. So, 
where are we now? About 72,000 people in the United States have sickle 
cell anemia that causes pain, chronic tiredness from anemia and severe 
infections, usually beginning when they are babies.\13\ In a published 
study last year in the New England Journal of Medicine that was 
supported by NIH, 10 adults were treated with adult stem cells from 
their brother or sister. Of these patients, nine no longer had symptoms 
of sickle cell anemia and were doing well at 4 years after their 
treatment.\14\ A similar study was published in 2008 showing that 6/7 
of the children with severe sickle cell anemia treated in a similar 
manner were without sickle cell symptoms when they were examined at 2-8 
years after treatment.\15\ It would be great if we could have everyone 
with sickle cell anemia treated.
---------------------------------------------------------------------------
    \13\ Anemia, sickle cell, http://www.ncbi.nlm.nih.gov/bookshelf/
br.fcgi?book=gnd∂= anemiasicklecell.
    \14\ Hsieh, MM, EM Kang, CD Fitzhugh, Allogeneic hematopoietic 
stem-cell transplantation for sickle cell disease. N Engl J Med 
2009;361:2309-2317.
    \15\ Krishnamurti L, Kharbanda S, Biernacki MA, Zhang W, Baker KS, 
Wagner JE, Wu CJ. Stable long-term donor engraftment following reduced-
intensity hematopoietic cell transplantation for sickle cell disease. 
Biol Blood Marrow Transplant. 2008 Nov;14(11):1270-8.




    The last picture is Barry Goudy who was suffering from multiple 
sclerosis (MS). He had numerous relapses and the medication was not 
helping his condition. He was part of a study conducted at Northwestern 
Memorial Hospital in Chicago and received his own stem cells in 2003. 
His MS symptoms disappeared in 4 months, and he continues to be symptom 
free today. Results were published last year by Burt and colleagues in 
Lancet.\16\ Patients had what is known as relapsing-remitting MS. These 
were patients who were still having relapses despite interferon beta 
treatment. All of the treated patients did not show the normal 
progressive worsening associated with MS, and a significant functional 
improvement was noted in these patients. In a similar study published 
this year, they describe the 1-year followup of six patients who showed 
improvement when their muscles were evaluated using 
electrophysiology.\17\ Their condition either stayed the same or 
improved in a disease that is characterized with progressive decline in 
function.
---------------------------------------------------------------------------
    \16\ Burt RK, Y Loh, B Cohen, D. Stefosky et al., Autologous non-
myeloablative haemopoietic stem cell transplantation in relapsing-
remitting multiple sclerosis: a phase I/II study. The Lancet 
Neurology--1 March 2009 (Vol. 8, Issue 3, Pages 244--253.
    \17\ Rice, CM, E A Mallam, A L Whone, P Walsh, D J Brooks, N Kane, 
S R Butler, D I Marks and N J Scolding. Safety and feasibility of 
autologous bone marrow cellular therapy in relapsing-progressive 
multiple sclerosis. Clinical Pharmacology and Therapeutics, May 5, 2010 
DOI: 10.1038/clpt.2010.44.




    The five pictures and their related clinical trials using adult 
stem cells show amazing progress for severe spinal cord injury, chronic 
heart failure, corneal blindness, sickle cell anemia, and multiple 
sclerosis. However, this is not an exhaustive list of the recent 
clinical trial findings using adult stem cells. I would just like to 
mention the amazing progress using adult stem cells in juvenile 
diabetes. A recent clinical trial report \18\ in the Journal of the 
American Medical Association found that the majority of the 23 patients 
who received adult stem cells achieved insulin independence in the 2-
year followup. Many may remember the news report of the person who 
received a new trachea using adult stem cells. An article published 
this year details the recovery of 20 patients with upper airway 
problems that received adult stem cells.\19\ Another break-through 
article was published this year in Blood which calls the use of adult 
stem cells ``. . . the gold standard in the frontline therapy of 
younger patients with multiple myeloma because it results in higher 
complete remission (CR) and longer event-free survival than 
conventional chemotherapy.\20\
---------------------------------------------------------------------------
    \18\ Couri CE, Oliveira MC, Stracieri AB, Moraes DA, Pieroni F, 
Barros GM, Madeira MI, Malmegrim KC, Foss-Freitas MC, Simoes BP, 
Martinez EZ, Foss MC, Burt RK, Voltarelli JC. C-peptide levels and 
insulin independence following autologous nonmyeloablative 
hematopoietic stem cell transplantation in newly diagnosed type 1 
diabetes mellitus. JAMA. 2009 Apr 15;301(15):1573-9.
    \19\ Macchiarini P, Rovira I, Ferrarello S. Awake upper airway 
surgery. Ann Thorac Surg. 2010 Feb;89(2):387-90; discussion 390-1
    \20\ Blade J, Rosinol L, Cibeira MT, Rovira M, Carreras E. 
Hematopoietic stem cell transplantation for multiple myeloma beyond 
2010. Blood. 2010 May 6; 115(18):3655-63.
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    Only with the help of NIH and the DOD Congressionally Directed 
Medical Research Programs, can these successful treatments reach all 
the people that desperately need them. I applaud Senator Harkin's 
efforts to increase the NIH budget in the past and ask all of the 
Senators and Representatives to make the people with diseases and 
injuries a major priority and put the patients first when considering 
funding stem cell research. These pioneers need to be joined by many 
other people to help those suffering from diseases and injuries. Adult 
stem cells aren't just showing great promise, but are treating people 
now. Much more of the limited funding needs to be directed at adult 
stem cells that are showing success right now.

    Senator Harkin. And now, Ms. Unser, welcome and please 
proceed.

STATEMENT OF CODY UNSER, FOUNDER, CODY UNSER FIRST STEP 
            FOUNDATION, WASHINGTON, DC
    Ms. Unser. Thank you, Chairman Harkin, for allowing me to 
testify and use my voice on behalf of millions of Americans 
living with debilitating diseases. I feel very honored and, to 
be honest, frustrated as to why we are here today.
    Ten years ago, my hero, my superman, Christopher Reeve, sat 
in his power wheelchair and using every breath he took, thanks 
to a machine, testified to Congress with the hope that 
embryonic stem cell research would be federally funded. Today 
in 2010 we are still fighting for this promising and hopeful 
research to continue.
    Embryonic stem cells are science based on hope, hope for 
improving the quality of life of millions of Americans by 
providing better treatment and eventually cures.
    My journey began 11 years ago. I was a healthy, 12-year-old 
kid who was very active and had big dreams. Everything changed 
on February 5, 1999. I cannot recall how it felt to put my feet 
on the floor, how I got dressed that morning, or what I had for 
breakfast, but what I do remember is that in a matter of 20 
minutes my body became paralyzed and my life drastically 
changed. I was playing basketball at school and suddenly could 
not catch my breath and my head started pounding with sharp 
pain. The school I was attending called the ambulance and while 
laying down in the locker room, my left leg became numb and 
tingly. I picked it up, put it back down, and I could not feel 
the floor. I was scared out of my mind, but I thought that 
whatever was wrong the doctors could fix.
    Transverse myelitis is an autoimmune disorder in which the 
immune system attacks the spinal cord causing inflammation that 
damages the cells that control sensory and movement of the 
body.
    After staying in the hospital for a couple of months, I 
went to rehabilitation where I learned how to do everything 
from a wheelchair, all the while having dreams of my feet 
imprinting in the sand.
    Today I am a 23-year-old woman who has learned to adapt to 
a life in a wheelchair and in a paralyzed body. Even though I 
live life to the fullest and look as though I am just sitting 
down in a wheelchair, I don't have to always worry about 
pressure sores from constant sitting. I worry about my 
osteoporosis advancing in my bones from not standing and 
bearing weight, which led to a fracture of my left femur. I 
worry about my scoliosis getting worse, a curvature of the 
spine common in people with spinal cord injuries. I have 
bladder and bowel complications and advancing nerve pain. But I 
am just one out of millions of Americans living with various 
diseases and conditions that no matter how hard we try affect 
how we live our lives.
    The first time hope actually meant something me to and 
became sort of my religion was when I saw what human embryonic 
stem cells can do. A year after I became paralyzed, my doctor 
and stem cell scientist, Doug Kerr, who was at Johns Hopkins at 
the time, showed me a mouse that was once paralyzed and now can 
bear its weight and take steps. At that moment, I realized that 
this is science I could not ignore, and it gave me a feeling of 
hope I wanted to fight for, which brings me to another point.
    It is frustrating to hear critics of this research say this 
is a path we cannot go down and adult stem cells hold just as 
much promise as embryonic stem cells do. Science is the pursuit 
of discovery and possibility. We should explore every 
opportunity and not count anything out because I cannot wait. 
And I know millions of Americans now and in the future cannot 
wait.
    In Christopher Reeve's testimony in 2000, he said, ``No 
obstacle should stand in the way of responsible investigation 
of their possibilities.'' I am here today to remove yet another 
obstacle in the path of this research, this answer, this hope.
    The political debate over this research is forcing many of 
our brilliant scientists to think twice about whether they 
should stay in this field. I know how dedicated and passionate 
they are about helping all of us find answers to our pain and 
suffering. If we keep dragging this debate back here to 
Washington, in Congress, and in the courts, more and more 
scientists will have no choice but to either find a different 
research avenue or move to another country where they can 
pursue the promise that embryonic stem cells possess. Once and 
for all, I urge Congress to pass unambiguous legislation that 
allows this research to move forward.

                           PREPARED STATEMENT

    I grew up around racetracks, and my family has won the 
Indianapolis 500 a total of nine times. The goal of every 
driver is to pass under the black and white checkered flag 
first. The meaning of the checkered flag is winning. Right now, 
I can see the flag waiting for me to go by, but with this 
current court ruling, I feel that I have been driving under a 
long, yellow caution flag. Today I came here to say that this 
research is real, promising, and hopeful to me and to others as 
we want so much to take that checkered flag and win our battles 
over diseases that constantly challenge our quality of life.
    Thank you very much.
    Senator Harkin. Thank you, Ms. Unser.
    [The statement follows:]

                    Prepared Statement of Cody Unser

    Thank you Chairman Harkin for allowing me to testify and use my 
voice on behalf of millions of Americans living with debilitating 
diseases. I feel very honored and to be honest, frustrated as to why we 
are here today.
    Ten years ago, my hero, my superman, Christopher Reeve sat in his 
power wheelchair and using every breath he took thanks to a machine, 
testified to Congress with the hope that embryonic stem cell research 
would be federally funded. Today, in 2010, we are still fighting for 
this promising and hopeful research to continue. Embryonic stem cells 
are science based on hope. Hope for improving the quality of life of 
millions of Americans by providing better treatment and eventually 
cures.
    My journey began 11 years ago. I was a healthy 12-year-old kid who 
was very active and had big dreams. Everything changed on February 5, 
1999. I can't recall how it felt to put my feet on the floor, how I got 
dressed that morning or what I had for breakfast, but what I do 
remember is that in a manner of 20 minutes my body became paralyzed and 
my life drastically changed. I was playing basketball at school and 
suddenly couldn't catch my breath and my head started bounding with 
sharp pain. The school I was attending called the ambulance and while 
laying down in the locker room my left leg became numb and tingly. I 
picked it up, put it back down and I couldn't feel the floor. I was 
scared out of my mind, but I thought that whatever was wrong the 
doctors could fix. Transverse Myelitis is an autoimmune disorder in 
which the immune system attacks the spinal cord causing inflammation 
that damages the cells that control sensory and movement of the body. 
After staying in the hospital for a couple of months I went to 
rehabilitation where I learned how to do everything from a wheelchair 
all the while having dreams of my feet imprinting in the sand.
    Today I am 23-year-old woman who has learned to adapt to a life in 
a wheelchair and in a paralyzed body. Even though I live life to the 
fullest and look as though I am just sitting down in a wheelchair, I 
have to always worry about pressure sores from constant sitting, I 
worry about my osteoporosis advancing in my bones from not standing and 
bearing weight which led to a fracture of my left femur. I worry about 
my scoliosis getting worse, a curvature of the spine common in people 
with spinal cord injuries. I have bladder and bowel complications and 
advancing nerve pain. But I am just one out of millions of Americans 
living with various diseases and conditions that no matter how hard we 
try affect how we live our lives.
    The first time Hope actually meant something to me and became sort 
of my religion was when I saw what human embryonic stem cells can do. A 
year after I became paralyzed, my doctor and stem cell scientist, Doug 
Kerr who was at Johns Hopkins at the time, showed me a mouse that was 
once paralyzed and now can bear its weight and take steps. At that 
moment, I realized that this is science I couldn't ignore and it gave 
me a feeling of hope I wanted to fight for. Which brings me to another 
point. It's frustrating to hear critics of this research say this is a 
path we can't go down and adult stem cells hold just as much promise as 
embryonic stem cells do. Science is the pursuit of discovery and 
possibility. We should explore every opportunity and not count anything 
out because I can't wait. And I know millions of Americans now and in 
the future can't wait. In Christopher Reeve's testimony in 2000 he 
said, ``No obstacle should stand in the way of responsible 
investigation of their possibilities''. I am here today to remove yet 
another obstacle in the path of this research, this answer, this hope.
    The political debate over this research is forcing many of our 
brilliant scientists to think twice about whether they should stay in 
this field. I know how dedicated and passionate they are about helping 
all of us find answers to our pain and suffering. If we keep dragging 
this debate back here to Washington, in Congress and in the courts, 
more and more scientists will have no choice, but to either find a 
different research avenue or move to another country where they can 
pursue the promise that embryonic stem cells possess. Once and for all 
I urge Congress to pass unambiguous legislation that allows this 
research to move forward.
    I grew up around racetracks and my family has won the Indianapolis 
500 a total of nine times. The goal of every driver is to pass under 
the black and white checkered flag first. The meaning of the checkered 
flag is winning. Right now, I can see that flag waving for me to go by. 
But with this current court ruling I feel that I have been driving 
under a long yellow caution flag. Today, I came here to say that this 
research is real, promising and hopeful to me and to others as we want 
so much to take that checkered flag and win our battles over diseases 
that constantly challenge our quality of life.
    Thank you very much.

    Senator Harkin. We now have two more votes, which I did not 
anticipate. I will try to get in a few questions. How much time 
do we have left? Eight minutes left?
    A couple of things. Dr. Morrison, in your statement, you 
alluded to claims made by some that adult stem cell research is 
more promising than embryonic stem cells because adult stem 
cells are already used to treat disease. We just heard a lot 
about that from Dr. Peduzzi Nelson. Could you expand on that? 
Should we be disappointed that embryonic stem cells have not 
yet yielded a cure? And how about all these pictures and things 
we just saw of people that have been cured by adult stem cells?
    Dr. Morrison. These arguments that you hear about focusing 
on adult stem cell research because they already treat people 
are meaningless arguments because human embryonic stem cells 
were first created in 1998. We have only had 12 years in which 
to work on them, whereas the adult stem cell therapies that are 
used clinically are related to bone marrow transplantation 
which was started in the 1950s. So we have had decades of work 
on adult stem cell therapies.
    Now, let me tell a very quick story. The first attempt at 
bone marrow transplantation between an unrelated donor and 
recipient was in 1955 by Donnall Thomas and his team. All the 
patients died. He went back to the laboratory to try to figure 
out why it was that he could not just randomly transplant bone 
marrow cells from one donor into another recipient and learned 
that you had to match the immune systems of the donor and the 
recipient so they did not attack each other.
    The first successful transplant from an unrelated donor and 
recipient was 1969, 14 years later.
    So if we applied this criterion of abandoning any form of 
research that does not lead to cures within 10 or 12 years, as 
has been suggested by opponents of embryonic stem cell 
research, then none of these adult stem cell therapies would 
exist today and, frankly, most of the medicines that we benefit 
from would never have been possible to develop.
    Senator Harkin. Dr. Daley, one line in your testimony. You 
said iPS cells and embryonic stem cells are different in 
important ways. I understand you have an NIH grant to examine 
this very issue that could be endangered, I understand, if 
Judge Lamberth's injunction were upheld.
    Again, tell me why this research is so important. What are 
the future discoveries that could be spurred by isolating the 
differences between these two types of cells?
    Dr. Daley. Yes. It is a major question to compare this new 
and very exciting and very powerful type of iPS cell against 
the embryonic stem cell, and we have one such grant. I am 
losing a lot of sleep over the future of that grant because 
when the injunction was in place, that grant was threatened. It 
was going to be pulled. It was not going to be renewed, and 
very promising projects that involve seven different 
institutions, the University of Miami, Boston University, Johns 
Hopkins, as well as Harvard Medical School, were all at risk.
    What our research has shown, our early research, primarily 
in mice and now also in humans, is that whereas our goal is to 
make iPS cells as close as possible to embryonic stem cells, 
despite our best efforts to date, there are still some 
differences. And understanding those differences is essential 
to understanding how those cells will behave in all of our 
research projects and ultimately for therapy.
    What we found is that after we turned the skin cells or the 
blood cells back to their embryonic state, they remember where 
they came from. Now, that can be an advantage. For us we are 
interested in treating blood diseases, and so we are migrating 
our work to work with iPS cells that are derived from the 
blood. But if you are interested in treating Hirschsprung 
disease or in treating liver disease, this fact that that 
memory exists may actually thwart your research.
    So fundamentally we are still so ignorant about how these 
new types of stem cells are going to function. We continue to 
depend on human embryonic stem cells.

                     AUTOLOGOUS STEM CELL TREATMENT

    Senator Harkin. Thank you very much.
    I have got some more. There are two more votes. I cannot 
hold you here any longer.
    Dr. Collins, Dr. Peduzzi's presentation see these people 
that have been cured--I have had people like that in my own 
office who have come in who have had autologous stem cell 
treatment in another country, and they come in and openly 
testified that whereas they could not walk, now they could a 
little bit. One person also had heart problems. What do I make 
of all this?
    Dr. Collins. So Dr. Peduzzi Nelson's examples are, in fact, 
exciting to see the potential that is here.
    Rob Califf who runs the clinical center at Duke once said 
something that I thought was kind of important, though, in all 
of this, that God gave us two gifts for understanding whether a 
treatment works or not: blinding and randomization. And if you 
have not applied those standards to an intervention, then you 
have to be skeptical because things happen that have nothing to 
do with the intervention. So the studies----
    Senator Harkin. What did you say again, Dr. Collins?
    Dr. Collins. Blinding, that is, the patients and the 
investigators cannot know whether that individual received the 
new treatment or some other placebo approach. And 
randomization, that is, patients get randomized to one or the 
other arm so that you do not have a bias in the outcome just 
based on their not being well matched.
    For all of us involved in medical research, until an effort 
has been put through that particular very stringent test, then 
one has to be a little concerned about whether what has 
happened is going to be generalizable. And that is what we 
want. We want things that you know will work for lots of 
people.
    The exciting research reported by Dr. Peduzzi Nelson for 
the most part has not yet reached that standard in terms of the 
spinal cords results that she talks about with these olfactory 
mucosal cells, although I think it is very exciting to see how 
that is. In fact, I understand there is an Australian study 
that has had difficulty replicating that. I am not, by this, 
saying that we should not be supporting that research. It is 
very exciting. We should be. But let us be clear about what we 
consider to be proof of success. Whether we are talking about 
human embryonic stem cells or iPS cells or adult stem cells, we 
have to be rigorous in our standards about when we are clear 
that we have established something confidently.
    Dr. Peduzzi Nelson. If I might make a comment.
    Senator Harkin. Dr. Peduzzi, I will give you a minute 
because I have got to go vote. I am sorry. Go ahead.
    Dr. Peduzzi Nelson. Just 1 minute. I would like to say that 
Dr. Collins is correct. And what I am saying is that we need 
the funds. We need funding from NIH and the Department of 
Defense, to bring these clinical trials to the next phase where 
there is blinding and there is randomization.
    I would like to make the second point. There is some 
confusion with the study in Australia. They in no way 
replicated the work in Portugal. They were using a different 
cell type.
    But beyond the fact about blinding and randomization, we do 
need that. In the case of the Portugal trial, there is a 
problem in that this is a surgical technique. They are actually 
putting the cells and tissue into the spinal cord. So you 
cannot go to the standard of having patients have a sham 
surgery. So that is part of the difficulty in verifying the 
technique.
    But all of these other clinical trials that I presented 
need to be replicated in the United States and they need to be 
brought on to the clinical trial where you do blinding of the 
patients, meaning that the people doing the investigation do 
not know if they received the treatment or not and the patient 
does not know that. So we need to move in that direction, and 
it is terribly expensive.
    Senator Harkin. Do you think that funding for embryonic 
stem cell research should be prohibited?
    Dr. Peduzzi Nelson. This gets to--I am here as a scientist 
and not here giving a personal opinion. As a scientist, what I 
came here to say is that in this country and other countries, 
we have seen some results that are, frankly, amazing, that are 
published in major journals that are clinical trials, and we 
need the funding for adult stem cells so they can become 
standard of care. It does not do anyone any good to treat 5 or 
10 patients.

                     ADDITIONAL COMMITTEE QUESTIONS

    Senator Harkin. I am sorry.
    Ms. Unser, again, I thank you very much for being here 
today. You are a very courageous young woman and thank you for 
picking up Christopher Reeve's mantel and moving ahead with it. 
I compliment you very much on that.
    I thank our whole panel. Thank you for being here. I am 
sorry. I could stay here and talk about this for another hour. 
I have got other things that I want to ask. I may submit 
questions to you in writing, and I would appreciate your 
responses to those.
    Dr. Collins, again, thank you very much.
    [The following questions were not asked at the hearing, but 
were submitted to the Department for response subsequent to the 
hearing:]

              Questions Submitted by Senator Thad Cochran

                           SCIENTIFIC HURDLES

    Question. In your testimony you said ``. . . our best window into 
human development is using human embryonic stem cells.'' In July, the 
Food and Drug Administration (FDA) authorized the first test--in 
humans--of an embryonic stem cell therapy. When these cells were 
tested, partially paralyzed animals walked. Dr. Collins, what is the 
most significant hurdle in translating current stem cell research into 
treatments like the one recently approved by the FDA?
    Answer. To use human embryonic stem cells (hESCs) for cell 
replacement and/or repair we must develop procedures that consistently 
produce a stem cell product that is appropriate for the specific use 
and safe for use in humans. Each particular disease or condition to be 
treated is likely to require a different cell therapy product. In some 
cases, scientists know the cell type required and the steps required 
for it to be produced. In others, they know the cell type required but 
not how to produce it and in yet others, investigators may not be sure 
what cell type would be most appropriate. Once the cell type has been 
identified and a protocol developed, scientists need to demonstrate the 
ability to produce enough of the desired cells consistently to allow 
comprehensive preclinical safety testing in animals, and support 
initiation of an early phase clinical trial in humans under FDA 
oversight. Significant hurdles during this stage of the process include 
manufacturing product of sufficient purity, tracking the cells inside 
the animal's body, and confirming that the cells integrate functionally 
into the target tissue. The timeframe for developing a stem cell 
therapeutic could easily be 10 years. For the few private companies 
making progress in the field of stem cell biology, such as Geron and 
ViaCyte, private sector funding has been consistent over a long period 
of time, while public funding has been unpredictable. If stem cell 
therapy is left to the private sector alone, development will likely be 
more restricted in breadth, access to research tools or results could 
be limited due to proprietary constraints, and innovative research may 
not be undertaken, thus hampering progress and threatening United 
States predominance in the field.
    Question. In your testimony, you said that human embryonic stem 
cells are a better model for how humans will respond to drug treatment 
than the current method using animal models. Can you expand upon this 
and provide a few examples?
    Answer. In the past, testing a drug or intervention on an animal 
model has been the best test that could be done before actually testing 
a drug on human beings in a clinical trial. In using an animal model, 
scientists are making an assumption that the human body is likely to 
respond to the drug or intervention being tested in a manner similar to 
that of the animal model's body, because humans and other animals are 
so similar in genetic makeup. However, scientists may now be one step 
closer to observing how the human body will respond to a therapy: we 
can use hESCs to generate the tissue of interest (heart, brain, skin, 
etc.) and then test the drug or intervention on those human cells for 
safety and effectiveness.
    For example, in the area of diabetes, scientists have identified 
numerous therapies that ``cure'' Type 1 diabetes in mice. Many of those 
treatments, however, that seemed so promising in the mouse have not 
proven to be effective in treating human diabetes. So, although animal 
models such as mice are still useful, they cannot tell us exactly how 
humans will respond. Scientists are making significant progress in 
learning how to coax hESCs into becoming mature human beta cells--the 
cells that produce insulin. They hope that these cells will be more 
useful tools to test potential diabetes drugs or other interventions 
and better predict how the human body will respond.
    In the area of heart disease, hESCs may also prove to be an 
invaluable tool. Although early stage clinical trials of drugs designed 
to improve heart function sometimes report positive results, the later 
stages are frequently halted due to unanticipated and negative side 
effects of the tested drug. Many of the drugs end up harming the very 
cells they were meant to help--the human heart cells, or 
cardiomyocytes. Scientists have now produced clinical grade 
cardiomyocytes from hESCs, and they hope that testing promising drugs 
on these cells prior to beginning clinical trials will speed safe and 
useful heart disease drugs to the many who need them.

                            FEDERAL FUNDING

    Question. If Federal funding is no longer available to support hESC 
research, would private or State funding be able to maintain the 
current pace of research?
    Answer. No. It would be extremely difficult for State or private 
funding in the United States to maintain the current pace of hESCs if 
Federal funds were no longer available.
    Currently, less than a dozen States have implemented funding 
programs for hESC research and the amount of research funding varies 
from State to State. When State research funds are available, the funds 
are restricted to scientists who conduct research within those States. 
However, scientists from all States are eligible to apply to the 
National Institutes of Health (NIH) for research funding and NIH awards 
grants based on the scientific merit of the research proposed. In 
fiscal year 2009, NIH made hESC research awards to institutions in 22 
different States.
    In addition, although there is no hESC policy in the United States 
that applies to both the public and private sectors, the NIH Guidelines 
for Human Stem Cell Research provide ethical standards for the States 
to follow if they choose.
    Without the central direction and coordinated research approach 
that the Federal Government can provide, many are concerned that the 
States' actions will result in duplication of research efforts among 
the States, variation in the level of ethics oversight, and ultimate 
loss of U.S. pre-eminence in basic hESC research, the foundation of 
translational and clinical hESC research.
    Long-term investment in basic research is necessary before 
scientific findings can be translated into clinical applications. For 
example, research is needed to understand the basic biology of hESCs 
before scientists can determine how hESCs can be coaxed into a 
particular cell type before stem cells can be used for drug testing or 
use in regenerative medicine. NIH is more likely to support these 
fundamental studies. Basic research must produce evidence of clinical 
relevance and demonstrate a potential market before the private sector 
will take up the research.

                     INTERNATIONAL COMPETITIVENESS

    Question. Medical research is projected to be one of the chief 
sectors for job growth over the next decade. What is the most 
significant hurdle for American scientists to remain competitive in the 
international stem cell arena?
    Answer. The most significant hurdle for American scientists to 
remain competitive in the international stem cell arena is the 
uncertainty of Federal funding for hESCs. In contrast, there is strong 
government support and investment in hESC in countries such as 
Singapore, India, China, and the United Kingdom. A 2008 study ranked 
the United States as a low performer in hESC research as compared to 
its leadership in other areas of emerging, but noncontroversial, 
biomedical research.\1\ Notably, the top four high-performing countries 
in hESC-related research (United Kingdom, Israel, China, and Singapore) 
all have supportive government policies for this field. The uncertainty 
in Federal funding for hESC research discourages established 
investigators from pursuing promising leads since they cannot count on 
stable funding for their best projects. Outstanding young scientists 
are reluctant to focus their efforts on promising hESC research when 
they may not be able to continue because of changes in funding policy.
---------------------------------------------------------------------------
    \1\ Levine, A.D. ``Identifying Under- and Overperforming Countries 
in Research Related to Human Embryonic Stem Cells'' Cell Stem Cell 2, 
June 2008, pp. 521-524.
---------------------------------------------------------------------------
                                 ______
                                 
               Question Submitted by Senator Patty Murray

                            RESEARCH FUNDING

    Question. In your testimony, you mentioned that the National 
Institutes of Health (NIH) has invested more than $500 million in human 
embryonic stem cell (hESC) research since 1998. You also mentioned that 
stopping Federal investment of this research mid-stream would result in 
wasting the funds that have been put in accounts or already drawn down. 
You stated this wasted amount will amount to $270 million. This 
inconsistent policy of Federal support has effects on research budgets 
and planning. As a researcher, have you seen how this policy has 
affected budgets for promising research? Could you expand a little bit 
on how looking for other types of funding when Federal support stops 
and starts affects the continuation of research?
    Answer. Speaking as both NIH Director and a researcher with my own 
laboratory, even a temporary suspension of funds can jeopardize ongoing 
research projects. When a laboratory experiment or clinical study is 
interrupted, it cannot be easily restarted. Such experiments may 
involve biological materials such as cell lines growing in lab 
incubators that must be managed daily to encourage growth and prevent 
contamination. Valuable laboratory animals serving as models of a wide 
range of human diseases and disorders that are being used to test new 
therapies could be lost due to the lack of funds to pay personnel to 
care for them. Once critical research tools and reagents--including 
unique materials that have taken years to develop--have been lost for 
lack of funding, it may take months or years to recreate them, if that 
is even possible. In clinical research projects, it can be very 
difficult to maintain the willingness of participants to stay involved 
with research. In cases where clinical interventions are being tested, 
this could pose severe ethical concerns about benefits and risks to 
those who have received only part of the scheduled protocol. In 
addition, laboratory personnel whose jobs depend on grant funds may be 
let go and the best investigators, including promising young 
investigators, may abandon that particular line of research or possibly 
move to other countries that have more predictable support for hESC 
research.

                         CONCLUSION OF HEARING

    Senator Harkin. Thank you all for being here. I am sorry I 
have to run.
    [Whereupon, at 12:17 p.m., Thursday, September 16, the 
hearing was concluded, and the subcommittee was recessed, to 
reconvene subject to the call of the Chair.]


              MATERIAL SUBMITTED SUBSEQUENT TO THE HEARING

    [Clerk's Note.--The following testimonies were received by 
the Subcommittee on Labor, Health and Human Services, and 
Education, and Related Agencies for inclusion in the record.
   Prepared Statement of the American Association for Cancer Research
    The American Association for Cancer Research (AACR), the world's 
oldest and largest professional organization dedicated to advancing 
cancer research, represents more than 32,000 cancer researchers, 
physician-scientists, other healthcare professionals, and survivors and 
patient advocates. On behalf of AACR, I thank you, Chairman Harkin and 
members of the Senate Appropriations Subcommittee on Labor, Health and 
Human Services, and Education, and Related Agencies, for holding this 
important hearing on the future and promise of human embryonic stem 
cell research. The AACR appreciates the opportunity to share its views 
on this issue.
    There is vast potential for stem cell research to improve the 
prevention, diagnosis, and treatment of cancer and many other diseases. 
Human embryonic stem cell research, in particular, may lead to new 
biological insights that offer previously unknown avenues for the 
development of promising new therapies for cancer patients. As stated 
in our 2005 policy statement on stem cell research,\1\ the AACR 
believes that reasonable, ethical exploration of the full spectrum of 
stem cell biology is a crucial component of scientific discovery.
---------------------------------------------------------------------------
    \1\ American Association for Cancer Research. Responsible 
Exploration of the Full Spectrum of Stem Cell Biology is Essential to 
the Advancement of Cancer Research. Position Statement, 2005. http://
www.aacr.org/home/public-media/aacr-press-releases/press-releases-
2005.aspx?d=482.
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 THE COURT INJUNCTION IS A SETBACK FOR SCIENTIFIC DISCOVERY AND CANCER 
                                RESEARCH

    Scientists who were recently given new opportunities under 
President Obama's Executive order \2\ to pursue important research 
questions using human embryonic stem cell lines could now be stopped in 
their tracks. The recent decision by the Federal District Court of the 
District of Columbia to block Federal funding for human embryonic stem 
cell research underscored the instability faced by scientists working 
in this promising field. The injunction created mayhem for scientists, 
who in the blink of an eye became unsure whether they could legally 
continue their experiments funded by the National Institutes of Health 
(NIH). A whole cadre of young scientists interested in pursuing this 
area of science may be discouraged from doing so due to concerns about 
funding stability. The AACR is deeply concerned that the lack of 
clarity on Federal funding for human embryonic stem cell research will 
significantly affect the ability of the United States to be a leader in 
this cutting-edge field of science that has real potential to save 
lives. United States scientists already face a distinct disadvantage in 
this field compared to their colleagues in countries such as Great 
Britain and Australia with more progressive, yet still ethically 
responsible, policies. While the injunction temporarily was lifted, 
great uncertainty remains as the case goes to the appellate court.
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    \2\ On March 9, 2009, President Barack H. Obama issued Executive 
Order 13505 Removing Barriers to Responsible Scientific Research 
Involving Human Stem Cells.
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   HUMAN EMBRYONIC STEM CELL RESEARCH HOLDS MUCH PROMISE FOR CANCER 
                                PATIENTS

    Stem cell research is part of a multi-faceted approach to 
understanding the biology of cancer and developing new ways to combat 
the 200 diseases collectively called ``cancer.'' Potentially paradigm-
shifting research may be developed from embryonic stem cell research, 
as scientists are just now learning what potential these stem cells 
hold and how they differ from the less-controversial adult stem cells.
    For example, recent scientific discoveries have shown that human 
cancer cells often display features that are reminiscent of human 
embryonic stem cells and that the more a cancer cell resembles an 
embryonic stem cell, the more aggressive its behavior. Indeed, it is 
only now being appreciated that the initiation and progression of many, 
if not all human cancers, involves deregulation of the very same genes 
and pathways that are necessary and responsible for normal human 
embryonic development. Inappropriate activation of these pathways in an 
adult cell can overtake its development and drive creation of a tumor.
    Early studies in the laboratories of numerous cancer researchers 
are showing that if these genetic and epigenetic errors are corrected, 
the growth of the cancer can be slowed or even reversed. However, 
successful translation of these exciting laboratory discoveries into 
advances for patient care requires that we better understand the 
differences between normal embryonic stem cell and cancer biology. To 
achieve this, it is absolutely imperative that this fundamental 
research, which has already led to so many significant discoveries, be 
allowed to continue. This research de facto depends on laboratory-based 
investigations of human embryonic stem cells.
    Another important advancement in cancer research has been the 
discovery that certain tumors arise as a consequence of genetic 
mutations in normal adult stem cells. For example, leukemia can arise 
when mutations occur in normal hematopoietic (blood) stem cells, and 
brain tumors can arise as a consequence of mutations in normal neural 
stem cells. The ability to isolate normal hematopoietic stem cells from 
bone marrow has fueled discovery into the origins of leukemia and is 
leading to the development of novel therapies to target leukemia stem 
cells. However, because of the relative rarity and inaccessibility of 
other adult stem cells, very little is yet known about their normal 
biology or how they morph into cancer cells. Cancer researchers are 
harnessing the pluripotency and regenerative power of embryonic stem 
cells to generate these rare adult stem cells in the laboratory.
    As a renewable source of neural, neural crest, pancreatic, liver, 
and other tissue-specific stem cells, embryonic stem cells are--for the 
first time--providing cancer researchers with the tools to study 
differences between normal adult stem cells and cancer stem cells. 
Already these studies are generating novel insights into tumor biology 
and identifying potential therapeutic targets that could be exploited 
to selectively kill cancerous stem cells.
    The benefits of this research are applicable especially to the 
pediatric population. Given that fully two-thirds of childhood cancer 
survivors are afflicted with long-term side effects from cancer 
treatments that negatively impact their health and well being, it is 
imperative that we strive to develop new therapies for pediatric 
cancers that spare normal stem cells and developing tissues. The 
promise of human embryonic stem cells as tools for scientific discovery 
provides hope for these children and for all patients who are afflicted 
with brain tumors, bone and soft tissue sarcomas, neuroblastoma, 
malignant melanoma, pancreatic, liver, and other solid tumors that all 
too frequently resist current therapies.

    AACR SUPPORTS SOUND, ETHICAL AND RESPONSIBLE STEM CELL RESEARCH 
                                POLICIES

    The AACR believes that human embryonic stem cell research must be 
conducted in accordance with policies that are sound, ethical, and 
responsible. As with any scientific investigation, explorations of stem 
cell biology must be pursued in strict accordance with such policies to 
safeguard the welfare of research donors and recipients. Individuals 
donating biological materials for research--including somatic cells, 
gametes and embryos--need to give their fully informed and voluntary 
consent through a mechanism uncompromised by financial incentive.
    The NIH has exerted significant effort to ensure that this 
promising research, like all NIH research, is conducted in a manner 
consistent with established ethical principles. After a thorough and 
transparent process involving extensive public input, the NIH put forth 
guidelines last July that stipulate the assurances and supporting 
documentation that must accompany requests for NIH funding for research 
using human embryonic stem cells. The guidelines also expressly 
prohibit funding for research projects using lines derived for the 
purpose of research through processes such as somatic cell nuclear 
transfer, in vitro fertilization or parthenogenesis. Neither Obama's 
Executive order nor the NIH guidelines permit Federal funding to be 
used for the generation of new stem cell lines.
    In considering its support for research utilizing human embryonic 
stem cells, the AACR recognizes and shares the universal sentiment that 
the human embryo deserves respect. Research involving human embryonic 
stem cells must serve important research aims that cannot be reached by 
other means. Moreover, we agree with the internationally accepted 14-
day limit on the developmental age of blastocysts from which the 
embryonic stem cells are derived.
    Although research using human embryonic stem cells raises many 
important ethical considerations, the majority of Americans believe 
that the potential for research to yield significant advances in 
patient care warrants responsible conduct of research. A 2008 Time 
magazine poll showed that nearly three-quarters of Americans support 
embryonic stem cell research using cells derived from embryos that will 
be discarded following in vitro fertilization procedures.\3\ Enforcing 
strict guidelines with appropriate oversight will ensure that such 
research is conducted according to the highest ethical standards.
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    \3\ SBIR Research poll for Time magazine. June 2008. http://
www.pollingreport.com/science.htm. Accessed September 8, 2010.
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                               CONCLUSION

    The AACR believes that stem cell research can be conducted in a 
manner consistent with established ethical principles, and strongly 
supports responsible explorations of the full spectrum of stem cell 
biology, including the use of human embryonic stem cells, for 
meritorious scientific research and therapy development.
    The AACR has been moving cancer research forward since its founding 
in 1907. The AACR and its more than 32,000 members worldwide strive 
tirelessly to carry out its important mission to prevent and cure 
cancer through research, education, and communication. Responsible 
embryonic stem cell research holds tremendous promise to deliver new 
therapies to patients suffering from cancer, as well as many other 
diseases such as heart disease, Parkinson's, diabetes, Alzheimer's, 
HIV/AIDS, and spinal paralysis.
                                 ______
                                 
        Prepared Statement of the Americans for Cures Foundation

    Honorable Senators Tom Harkin and Thad Cochran, members: Thank you 
for this opportunity to provide testimony on a subject which affects 
100 million Americans with a chronic (incurable) disease or 
disability--and everybody who pays the medical bills.
    The costs are staggering: last year, chronic illness cost America 
$1.65 trillion, more than all Federal income taxes ($1.2 trillion) 
combined. The suffering is incalculable.
    These are not empty statistics, but members of your family and 
mine: people like my son.
    On September 10, 1994, Roman Reed was playing college football. At 
middle linebacker he was having a great game: 11 solo tackles, a diving 
one-hand interception, a forced and recovered fumble.
    And then, the accident. There was a hideous sound, like an axe 
handle breaking on a rock. In an instant our son was paralyzed from the 
shoulders down. He was 19.
    The doctors gave us no hope.
    ``He will never walk again, nor close his fingers; almost certainly 
no children'', they said.
    We would not accept that diagnosis then, and we do not accept it 
now. We worked to find a cure.
    With the leadership of Fremont Assemblyman John Dutra, we passed a 
California law, Assembly Bill 750, the Roman Reed Spinal Cord Injury 
Research Act of 1999.
    On March 1, 2002, I held in my hands a rat which had been 
paralyzed, but which walked again, thanks to embryonic stem cells--as 
my son watched from his wheelchair.
    This was the famous experiment to re-insulate damaged nerves in the 
spine. Geron is taking it to the world's first human trials of 
embryonic stem cells, recently approved by the Food and Drug 
Administration (FDA). Ten newly paralyzed young men or women will be 
offered a chance my son did not: to maybe get better, through embryonic 
stem cell research.
    ``Roman's law'' funded the first use of the Presidentially approved 
embryonic stem cell lines. And, importantly, the Federal Government 
backed us up. For our total expenditure of $14 million over 9 years, we 
brought in an additional $60 million in follow-up grants and matching 
funds from the National Institutes of Health (NIH), new jobs and 
revenue. But it was not enough.
    For a cure to come, not only for paralysis, but also the dozens of 
incurable diseases afflicting so many, the entire field of regenerative 
medicine had to advance.
    In 1942, research connected with the medical aspects of radiation 
sickness from the atomic bomb revealed that bone marrow transplants had 
healing properties. That was the beginning of adult stem cell research, 
which has proved extremely useful in the treatment of forms of cancer 
and blood disease.
    But it is not the exclusive answer to all chronic illness and 
injury.
    Embryonic stem cells which build every portion of the body are also 
important.
    The difference between adult and embryonic stem cells is like the 
difference between gift certificates and cash money--one can only be 
spent in certain places, the other is acceptable everywhere.
    For example, adult stem cells heal surface wounds slowly, leaving a 
scar. Embryonic stem cells build the entire human body. The difference 
is power is extraordinary.
    For the field of regenerative medicine to advance, both types of 
cells are needed. Each is different and has different powers and 
purposes.
    But there are subtle dangers to be aware of with adult stem cells. 
They cannot always do what embryonic stem cells can. Sometimes adult 
cells which have been experimentally turned into useful cells revert to 
their original adult stem cell state. One attempt to turn adult stem 
cells into nerve cells did not last, and after a few days, the rats 
which had the cells implanted developed excruciating pain, so they 
gnawed off their paws. (A replication of this study was done by Dr. 
Candace Floyd, UC Davis.)
    An approach I regard as a failure is the attempt to use Olfactory 
Epithelial Glia (OEGs) to restore spinal cord function, basically 
reaching a scalpel up into the nose and scraping off part of the brain, 
which is then spread like jelly on the injured spine. I have spoken 
with a recipient of that treatment who described (after an expenditure 
of approximately $40,000) the essential failure of it: the paralyzed 
person regained a patch of skin sensation on his elbow, so that he 
could feel his sleeve go on, when they dressed him in the morning.
    Much has been said about ``adult stem cell treatments for 70 
diseases'', but this is misleading at best. Prescribing aspirin for 
cancer may be a treatment, but it is not a cure.
    The ancient scientist Galen spread pigeon dung on the spines of 
paralyzed gladiators. It was a treatment, but hardly a successful one.
    The idea of adult stem cells being ready to be the sole standard of 
treatment is not only unwise but cruel, imposing something unreliable 
in place of the possibility of actual cure.
    California's Bob Klein began an initiative, Proposition 71, the 
Stem Cells for Research and Cures Act. I was proud to serve on the 
board of directors of that successful effort.
    But even when 7 million voters approved the $3 billion stem cell 
program, lawsuits were hurled against us: frivolous in their grounds, 
but devastating in their consequences.
    For almost 2 years the full program was held up. Research delayed 
is research denied. Who knows what might have been discovered during 
that time, if we had our program fully operational?
    But we prevailed, and today the California Institute for 
Regenerative Medicine (CIRM) is the pride of our State and a friend to 
all the world.
    Recently, four major grants, $20 million each, were awarded by the 
CIRM for embryonic stem cell research: Lou Gehrig's disease, stroke, 
juvenile diabetes, and age-related blindness were chosen. Each is an 
attempt to do the impossible with the invisible: to try and heal a 
malady incurable since the dawn of time.
    Cures the CIRM develops will benefit everyone; not only the 
individual families whose suffering will end, but also the economies of 
every nation, struggling to pay mountains of medical debt. All will 
benefit.
    But we need the Federal Government to help with the enormous costs 
which wait beyond initial research: the ``valley of death'' which faces 
all new medical discoveries: the costs of turning theory into therapy, 
all the way from bench to bedside, may approach $1 billion. 
California's program has a budget of approximately $300 million a year: 
we can't do it alone.
    March 9, 2009, a day of joy. Roman, Gloria and I were in the room 
when President Barack Obama reversed the Bush restrictions. Now, at 
last, the Federal Government would take its rightful place, leading in 
the quest for cure.
    But another obstacle arose. The case of Sherley v. Sebelius may 
shut down Federal funding of the research so many patients and families 
have worked to advance.
    The argument is often made by ideological opponents that embryonic 
stem cell research is a form of abortion.
    This is false. How can there be an abortion, when there is no baby?
    There is no pregnancy in embryonic stem cell research. Nothing is 
placed in the womb. It is biologically impossible for an unemplanted 
blastocyst to become a child. It is living tissue, like a wiggling 
sperm, but not a life. It cannot possibly become a child without the 
nurturing protection of a mother's womb. No mother, no baby: this is 
unarguable fact.
    Congress had a full and vigorous debate on Federal funding for the 
research: and approved it twice. The Stem Cell Research Enhancement 
Acts of 2005 and 2007 passed both houses with strong majorities. 
President Bush exercised his prerogative and vetoed both bills, but the 
will of Congress was crystal clear.
    The Senate, the House of Representatives, and the President of the 
United States support Federal funding of embryonic stem cell research.
    If research funding is blocked for ideological reasons, we abandon 
a principle: that every American family deserves the best medical 
treatment science can provide.
    Denying cure condemns 2 million paralyzed Americans like my 
paralyzed friend Karen Miner to a life sentenced incarcerated in a 
chair; it diminishes hope for those who suffer cancer and leukemia, 
which killed my mother and my sister Patty; and it slows the growth of 
jobs in America's shining new industry: biomedicine. Nine years ago, 
September 5, 2001, I provided testimony for Senator Edward Kennedy's 
similar hearings on scientific freedom for stem cell research. I 
conveyed my son Roman Reed's request, asking that the Senate:

    ``Take a stand: take a stand in favor of medical research; take a 
stand-so one day everybody can.''

    Roman and his wife Terri, and their three children, Roman Jr., 
Jason, and Katie--send that message again.
    Finally, I would be remiss if I did not cite one of America's 
greatest advocates.
    The late Christopher Reeve sent a dictated letter to our family. It 
said: ``One day, Roman and I will stand up from our wheelchairs, and 
walk away from them forever.''
    Cure did not come in time for the paralyzed Superman, but we still 
believe in his great dream. Our champion has fallen, but the flame of 
his faith still lights our way. He always said, we must ``go forward''. 
And we will go forward: because America has picked up the torch.
                                 ______
                                 
    Prepared Statement of the California Institute for Regenerative 
                                Medicine

    In 2004, 7 million Californians, accounting for 59 percent of the 
electorate, approved Proposition 71, The Stem Cell Research and Cures 
Initiative; creating the California Institute for Regenerative Medicine 
(CIRM), the State stem cell institute. In the ensuing 5 years, 
scientists and clinicians empowered by CIRM have made extraordinary 
advances in medical research.
    As the largest United States based funder of human embryonic stem 
cell research during the Bush administration, CIRM has a unique 
understanding of the importance of stable funding for therapies derived 
from human embryonic stem cell research. At least four grants for 
therapies derived from human embryonic stem cells are currently headed 
towards human clinical trials, including: Type 1 Diabetes, Stroke, 
Macular Degeneration (age-related vision impairment or blindness), and 
Amyotrophic Lateral Sclerosis (ALS--Lou Gehrig's Disease).

  NATIONAL INSTITUTES OF HEALTH (NIH) GRANTS FOR HUMAN EMBRYONIC STEM 
CELL RESEARCH COVER CRITICAL WORK ON WHICH CIRM/CALIFORNIA RESEARCH IS 
                               DEPENDENT

    Although California-based embryonic researchers receive substantial 
financial commitments from CIRM, the NIH's backing is crucial for many 
of our grantees. If the Federal Government stopped funding embryonic 
stem cell research, many of our grantees most promising work would be 
in jeopardy. With 50 percent of our stem cell researchers responding, 
more than 31 of our grantees that perform embryonic stem cell research 
have indicated that they also received NIH funding covered by NIH 
regulations. The reported total value of these grants is $45.5 million. 
Virtually all reported there would be negative impact on their research 
if NIH funds were cut.
    More importantly, halting this research will have a devastating 
impact on the future of the field and the United States' leadership 
position in biomedical research. With a 50 percent response rate, 16 of 
our grantees reported the need to eliminate or reduce postdoctoral 
positions, if NIH funding is not permanently restored. America's best 
and brightest scientists are unlikely to enter this promising field if 
Federal funding is cut off or stagnant.
    While we are thrilled California is among the worlds leaders in 
biomedical research, California cannot drive the field alone. It is 
imperative for the NIH, the leading funder of biomedical research in 
the world, to fund this vitally important field. Collaboration between 
scientists at different institutions around the world is imperative if 
we are going to develop therapies and cures to fight some of today's 
most debilitating diseases. The collaboration among many of these 
scientists depends on U.S. scientists receiving NIH funds.

  VALIDATING THE POTENTIAL FOR THERAPIES DERIVED FROM HUMAN EMBRYONIC 
                               STEM CELLS

    In October 2009, CIRM awarded $230 million for 14 unique 
multidisciplinary Disease Team Research Awards. The goal of these 
awards is to develop new medical therapies from stem cell research to 
reduce the suffering from chronic disease and injuries to cure these 
conditions, if possible. According to an international peer review 
panel of 15 scientists (all from outside of California) the Disease 
Team grants and loans, have all demonstrated ``compelling and 
reproducible evidence'' that ``demonstrates that the proposed 
therapeutic has disease- (or injury-) modifying activity. The project 
is sufficiently mature, such that there is reasonable expectation that 
an Investigational New Drug (IND) filing'' for a phase 1 human trial 
``can be achieved within 4 years of the project start date.''
    As previously stated, four of these projects utilize embryonic stem 
cell research to treat some of today's most harmful conditions, 
including: Type 1 Diabetes, stroke, macular degeneration, and 
amyotrophic lateral sclerosis (ALS--Lou Gehrig's Disease).

                            TYPE 1 DIABETES

    This public-private disease team partnership between Novocell Inc. 
and the University of California San Francisco (UCSF) has developed 
methods to make large-scale batches of replacement beta cells from 
human embryonic stem cells (hESC). The team has demonstrated that these 
hESC-derived beta cells cure experimental diabetes in mice and rats. 
Additionally, they have devised strategies to reduce the risk that 
recipients will see these implanted hESC-derived beta cells as foreign 
cells and subsequently reject them. The team now plans to complete the 
manufacturing, efficacy, safety testing required to generate the 
necessary data for Food and Drug Administration approval to test in 
phase 1 clinical trials.

                                 STROKE

    Led by renowned Stanford and University of California Los Angeles 
(UCLA) researchers, this team has produced preliminary evidence on the 
use of cells derived from human embryonic stem cells as a poststroke 
treatment to improve recovery in the weeks and months following a 
stroke. The team has developed a technique that to restrict the 
potential of embryonic stem cells to neural stem cells that 
differentiate only into cell types that are normally found in the 
brain. When these neural stem cells are transplanted into the brains of 
mice or rats 1 week after a stroke, the animals are able to regain 
strength in their limbs. Based on these findings, the Stanford led team 
proposes to further develop these neural stem cells into a clinical 
development program for stroke in humans at the end of this grant 
period.

   MACULAR DEGENERATION (AGE-RELATED VISION IMPAIRMENT OR BLINDNESS)

    The multidisciplinary team led by researchers at the University of 
Southern California and the University of California--Santa Barbara 
have produced preliminary evidence on the use of human embryonic stem 
cells to replace dysfunctional or destroyed retinal pigment epithelial 
cells to slow or reverse the disease. They plan to coax human embryonic 
stem cells to differentiate into a monolayer of retinal pigment 
epithelial cells that can be transplanted into the eye. The replacement 
RPE cells will function normally to support and protect the light-
sensitive cells of the retina and prevent further degeneration and 
vision loss.

                       ALS--LOU GEHRIG'S DISEASE

    This San Diego based team (from the Salk Institute for Biological 
Studies, University of California San Diego, and the Ludwig Institute 
for Cancer Research) plans to protect surviving neurons in people 
diagnosed with ALS from further degeneration. The strategy involves 
targeting glial cells, which are neuroprotective cells that surround 
and support neurons. A type of glial cell called an astrocyte is found 
in both the brain and spinal cord and acts as a regulator of glutamate 
surrounding motor neurons. The team intends to grow human embryonic 
stem cell-derived astrocyte precursors that will be transplanted 
directly into the spinal cord environment to prevent further 
neurodegeneration caused by ALS. The work, which is based on mouse 
experiments, should be effective in both familial and sporadic ALS.

                          ALZHEIMER'S DISEASE

    Alzheimer's disease, the most common cause of dementia among the 
elderly and the third-leading cause of death, presently afflicts more 
than 5 million people in the United States, including more than 500,000 
in California. University of California Irvine (UCI) received an early 
translational grant from CIRM aimed at developing a development 
candidate for treating Alzheimer's disease. Their proposed studies, 
utilizes embryonic stem cells to develop a novel and promising strategy 
for creating an effective therapeutic. Their preliminary studies 
indicate that stem cell biology may provide a significantly more 
effective therapy for the disease than any current pharmaceutical 
products. These results, however are preliminary, and will require 
years of additional research to confirm the potential.
    We have a moral obligation as citizens of the United States to 
support the dedicated scientists, clinicians, and patient advocate 
organizations to pursue the best scientific approaches across the 
scientific field of stem cell research, including human embryonic stem 
cell research--to reduce the suffering of our families and friends and 
families around the world.
                                 ______
                                 
    Prepared Statement of the Student Society for Stem Cell Research

    On behalf of the Student Society for Stem Cell Research (SSSCR), 
our chapters across this great Nation, and our membership worldwide, we 
urge Congress to act expeditiously to address recent events regarding 
Federal funding of human embryonic stem cell research. The recent 
preliminary injunction against human embryonic stem cell research in 
Judge Lamberth's district court is deeply disturbing and threatens the 
education and training of thousands of students. More specifically, 
graduate students working on human embryonic stem cell projects 
supported by the National Institutes of Health (NIH) are in danger of 
having their financial support entirely cut off. Our constituency is 
directly affected by the court's ruling. Many of us will not be able to 
continue our biomedical programs if the injunction holds and Congress 
fails to act. Simply stated, we will lose our means of financial 
support and potentially our careers. The impact on the American people 
will be devastating as our country risks losing scores of developing 
scientists working on the most promising medical science in human 
history.
    On March 9, 2009, SSSCR and the more than 3,500 members of our 
network were elated when President Obama issued Executive Order 13505, 
entitled ``Removing Barriers to Responsible Research Involving Human 
Stem Cells.'' It is our assertion that the order expressed the will of 
the American people, Congress, and in particular our generation. 
Medical research and clinical advances that would alleviate human 
suffering and restore quality of life is a humanitarian concern of 
greatest importance to us. Each year, at hundreds of U.S. institutions, 
students enter undergraduate, graduate, and medical school programs 
designed to fulfill the needs of the biomedical industry. The 
biomedical industry is one of the fastest-growing industries nationally 
and internationally. Within the biomedical industry, stem cell research 
has attracted the excitement of students, researchers, doctors, and 
companies. In 2009, the State of California passed Bill 471, ``The 
Biomedical Training and Stem Cell Research Education Act'' to ensure 
that enough workers would be trained to meet the growing demands of the 
regenerative medicine industry. Students believe that the medical 
promise of cellular regeneration using stem cells is unparalleled in 
human history. This excitement is demonstrated by our decision to 
choose medical research projects focusing on stem cell applications, in 
which the vast majority of graduate student stipends are funded by NIH 
grants. The accelerated development of this new field, unprecedented 
State bills to support the research and career training of scientists 
and doctors, and the influx of commercial investments are all 
indicative of a trajectory consistent with our intuitive assertion that 
stem cell research will revolutionize medicine. However, absent the 
temporary stay, the recent preliminary injunction turns the field on 
its head and is immediately devastating to career development in 
regenerative medicine, threatening our generation's long term objective 
of finding cures to intractable medical conditions. Federal funding of 
human embryonic stem cell research is the will of the people, necessary 
for biomedical advance, critical to America maintaining its competitive 
advantage, morally and ethically acceptable, and integral to the 
education and training of the next generation of scientists and 
doctors. Human embryonic stem cells (hESCs) are unique in their use as 
biomedical research tools and for their current and eventual clinical 
application. HESCs are not supplanted by any other cell source, 
including adult stem cells or induced pluripotent stem cells. 
Therefore, SSSCR urges Congress to act swiftly and decisively to 
legislatively fix our current public policy debacle on funding hESC 
research
    In the interest of maintaining a strong, healthy, and competitive 
Nation we present to Congress the following arguments:
    Federal Funding of Human Embryonic Stem Cell Research is the Will 
of the American People and Congress.--Poll after poll since the early 
2000's has unanimously demonstrated a majority support for hESC 
research and Federal funding of the research. State led campaigns in 
California, Missouri, and Michigan have sided with the research. In the 
most recent poll on the subject conducted in August 2010 by 
Research!America, it was found that 70 percent of Americans favor 
expanded Federal funding of research using human embryonic stem cells. 
Gallup polls from 2005 to 2009 have shown support for fewer 
restrictions on hESC research to range between 52-60 percent. In 2006 
and 2007, Congress passed Stem Cell Research Enhancement Acts in both 
the House and Senate specifically removing Federal funding restrictions 
on new stem cell lines. It has been the unambiguous interpretation of 
three administrations, the NIH in 1999, congressional votes in 2006 and 
2007, and Senate appropriations since 2002, that Federal funding of 
hESC research does not violate the Dickey-Wicker amendment. Five 
hundred forty-six million dollars in Federal funds has been invested in 
hESC research, since 2002, and nearly a decade of scientific advance is 
in jeopardy. Projects may be lost forever and millions of taxpayer 
dollars wasted. The students engaged in these projects will very likely 
have to change their discipline or their careers entirely due to losing 
their research stipends.
    HESC is Morally and Ethically Acceptable.--It is SSSCR's contention 
that a society's highest moral obligation is to treat the sick 
medically and with dignity. When a research path presents hope to 
millions of patients with debilitating conditions, the only dignified 
approach by society and government is to provide that hope by pursuing 
promising medical research in an expeditious and ethical manner. SSSCR 
feels that the reality of the potential for excess IVF embryos to 
generate offspring is often clouded by ideology from those opposing the 
research. Scientifically, many of the embryos that would be donated to 
research for the generation of new human embryonic stem cell lines are 
enviable for implantation and could never lead to a successful 
pregnancy. The supply of IVF embryos far exceeds the demand for 
reproductive purposes. In a famous RAND corporation study, it was found 
that more than 400,000 IVF embryos were still in storage, dating back 
to the 1970s. This fact underscores that hESC research does not 
necessitate the destruction of any embryo nor has the research 
prevented a single pregnancy. The generation of IVF embryos for 
reproductive purposes results in excess embryos that will be stored 
indefinitely or destroyed. The creation of stem cell lines for research 
is a subsequent, determining act that chooses humanitarian benefit over 
biomedical waste. Furthermore, prior congressional legislation and the 
``New Guidelines on Human Stem Cell Research'' have carefully addressed 
ethical considerations by mandating approved lines to have been donated 
under informed consent, without financial incentive, and for the embryo 
to have been created with reproductive intent, but no longer desired 
for such purposes. Therefore, SSSCR feels that it is our society's 
moral obligation to conduct hESC research and that the Government has 
put in place an appropriate framework for students to ethically 
continue our research projects and to pursue our passion for finding 
cures.
    NIH Funding is Critical to the Education and Training of the Next-
generation of Scientists and Doctors.--The vast majority of biomedical 
research conducted at our universities is carried out by graduate and 
medical students who depend on a mentor's grant to fund their stipend, 
which covers living expenses while completing the biomedical research 
program. For fiscal year 2010, the NIH funded $131 million for hESC 
research. In total, there are 223 hESC NIH-funded research projects 
estimated to support 1,300 jobs. However, the impact can spread beyond 
these projects. One example is the UCSF Medical Scientists Training 
Programming involving 88 students earning joint M.D. and Ph.D. degrees. 
The entire program is in jeopardy of losing NIH funding, since mentors 
working with hESCs cannot be separated out based on the interdependency 
of the award and the applicant. Twenty-four NIH-funded projects that 
are up for $54 million in annual renewal on September 30, 2010, is in 
jeopardy if a stay in not continued at the projected September 27 
hearing. In future fiscal years, projects up for annual renewal are 
also in danger of losing all funding. In all these cases, the immediate 
impact of an injunction on Federal funding of hESCs is the imminent 
loss of students' stipends. Current students will be financially forced 
out of the field to find alternative salary, while new students will be 
dissuaded from entering the field, and America will begin to lose its 
competitive advantage in science and medicine. It is imperative that 
Congress takes action to establish the policy for hESC research 
funding, so that we never again jeopardize the future and training of 
our young scientists and doctors in this field.
    HESCs are Unique in Their Medical Promise and are not Replaced by 
Other Types of Stem Cells.--Despite 50 years of research using adult 
stem cells, severe limitations have not been overcome, such as growing 
them in sufficient numbers for clinical use and continued failure to 
treat nonhematological tumors. Pluripotent cells offer a distinct 
advantage for neurological conditions where brain biopsies can only be 
used for diagnostic purposes and autopsies do not yield viable nerves. 
A recent medical advance in reprogramming adult cells back to a 
pluripotent state, called induced pluripotent stem cells (iPSCs), is 
very promising; however, iPSCs do not replace the need for hESCs in 
basic research and clinical application. The industry standard for iPSC 
cells is not well-established, resulting in a great deal of inter-lab 
variability, and characterization continues to be based on hESC 
comparison. Clinically, iPSC cells remain very hazardous and are 
unlikely for FDA approval anytime soon. The reprogramming process 
involves hazardous viral gene delivery methods and in some cases known 
cancer causing genes. While the field hopes to resolve these issues, 
it's too early to project confidently their use in the clinic. Much 
more basic research needs to be done on both iPSC and hESCs to 
understand their biological differences and similarities. Geron has 
received FDA approval for clinical trials using hESCs to treat spinal 
cord injury. Advanced Cell Technologies is expected to receive FDA 
approval to use hESC-derived retinal cells in clinical trials to treat 
eye disease. IPSCs do not replace hESCs. Clinical trials with hESCs 
have already received FDA approval, while iPSCs remain clinically 
hazardous with the current technology.
    Our generation has a great responsibility to society to advance 
science and medicine. In order to realize the potential of regenerative 
medicine and maintain our global scientific leadership, our young 
scientists and doctors in training must receive the financial support 
to continue their programs. The NIH is critical in this aim and thusly 
Federal funding for hESC research must be safeguarded and maintained. 
We urge Congress to sort out the legal wrangling and provide our 
generation the chance to use hESCs in regenerative medicine to discover 
cures for devastating medical conditions.

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