[Senate Hearing 109-191]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 109-191
 
         EXPLORING THE PROMISE OF EMBRYONIC STEM CELL RESEARCH

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

                                HEARING

                               before the

                       SPECIAL COMMITTEE ON AGING
                          UNITED STATES SENATE

                       ONE HUNDRED NINTH CONGRESS

                             FIRST SESSION

                               __________

                             WASHINGTON, DC

                               __________

                              JUNE 8, 2005

                               __________

                            Serial No. 109-8

         Printed for the use of the Special Committee on Aging




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                       SPECIAL COMMITTEE ON AGING

                     GORDON SMITH, Oregon, Chairman
RICHARD SHELBY, Alabama              HERB KOHL, Wisconsin
SUSAN COLLINS, Maine                 JAMES M. JEFFORDS, Vermont
JAMES M. TALENT, Missouri            RUSSELL D. FEINGOLD, Wisconsin
ELIZABETH DOLE, North Carolina       RON WYDEN, Oregon
MEL MARTINEZ, Florida                BLANCHE L. LINCOLN, Arkansas
LARRY E. CRAIG, Idaho                EVAN BAYH, Indiana
RICK SANTORUM, Pennsylvania          THOMAS R. CARPER, Delaware
CONRAD BURNS, Montana                BILL NELSON, Florida
LAMAR ALEXANDER, Tennessee           HILLARY RODHAM CLINTON, New York
JIM DEMINT, South Carolina
                    Catherine Finley, Staff Director
               Julie Cohen, Ranking Member Staff Director

                                  (ii)




                            C O N T E N T S

                              ----------                              
                                                                   Page
Opening Statement of Senator Gordon Smith........................     1
Opening Statement of Senator Herb Kohl...........................     3
Opening Statement of Senator Susan Collins.......................     4
Opening Statement of Senator Ron Wyden...........................     5
Opening Statement of Senator Thomas Carper.......................     6
Prepared Statement of Senator Hillary Rodham Clinton.............    16

                                Panel I

Chris Dudley, diabetes advocate, and former center, Portland 
  Trailblazers, Portland, OR.....................................     8
Larry Goldstein, Ph.D., University of California-San Diego, 
  School of Medicine, San Diego, CA; representing the American 
  Society for Cell Biology.......................................    20
Douglas A. Doerfler, president and chief executive officer, 
  MaxCyte, Gaithersburg, MD, on behalf of the Biotechnology 
  Industry Organization..........................................    32
John D. Gearhart, Ph.D., Johns Hopkins University, Department of 
  Medicine, Institute for Cell Engineering, Baltimore, MD........    40
Su-Chun Zhang, M.D., Ph.D., assistant professor of Anatomy and 
  Neurology, Stem Cell Research Program, Waisman Mental 
  Retardation Center, University of Wisconsin-Madison, Madison, 
  WI.............................................................    49

                                APPENDIX

Statement submitted by the John Hopkins University...............    57

                                 (iii)




         EXPLORING THE PROMISE OF EMBRYONIC STEM CELL RESEARCH

                              ----------                              --



                        WEDNESDAY, JUNE 8, 2005

                                       U.S. Senate,
                                Special Committee on Aging,
                                                    Washington, DC.
    The committee met, pursuant to notice, at 2:05 p.m., in 
room SD-G50, Dirksen Senate Office Building, Hon. Gordon H. 
Smith (chairman of the committee) presiding.
    Present: Senators Smith, Collins, Kohl, Wyden, Lincoln, 
Carper, and Clinton.

     OPENING STATEMENT OF SENATOR GORDON H. SMITH, CHAIRMAN

    The Chairman. Good afternoon, ladies and gentlemen. I would 
like to welcome you all to what is sure to be an interesting 
and highly informative hearing of the Senate Special Committee 
on Aging: exploring the promise of embryonic stem cell 
research.
    Stem cell research is one of today's most exciting and 
rapidly advancing fields in modern medicine. It holds the key 
to potentially unlocking the secrets of diseases that have 
mystified scientists for years, namely, Alzheimer's, 
Parkinson's, diabetes, cardiovascular disease, and more. This 
becomes particularly important as our Nation's population ages 
and more and more of our seniors become afflicted with ailments 
that take a great toll on the families of loved ones, as well 
as on the individuals themselves.
    Scientists are just beginning to scratch the surface of the 
knowledge and benefits that can be reaped through a thorough 
understanding of stem cells and their potential for creating 
breakthroughs in therapeutic disease treatment.
    This hearing will examine some of the most important 
progress being made in the area of embryonic stem cell 
research, the need for new stem cell lines, and the reasons 
these additional lines should receive Federal support.
    Among the elderly, diseases such as diabetes, Alzheimer's, 
Parkinson's, and cardiovascular ailments are among the most 
prevalent and the most costly to the Federal budget and family 
budgets. Together, the estimated annual direct and indirect 
costs of caring for patients with these diseases is $650 
billion. Alzheimer's alone is a disease that afflicts one in 
ten Americans over the age of 65, and nearly half of all 
persons over the age of 85. As baby boomers begin to age, the 
prevalence of Alzheimer's is expected to grow by 350 percent, 
from 4 million Americans today to an estimated 14 million by 
2050, which will make it one of the most costly diseases in our 
society.
    The impact on Medicaid and Medicare and our private health 
care system will be enormous. However, if we can find a way to 
delay the onset of Alzheimer's by just five years, we could 
reduce the number of cases and spending on the disease by more 
than half, by more than 50 percent. In addition, diabetes, 
neurodegenerative, and cardiovascular diseases also happen to 
be areas for which stem cell therapy seems most promising. 
Although a limited number of human embryonic stem lines are 
eligible for use in federally funded research, many scientists 
are concerned about the usefulness of these lines. While some 
claim a total of 78 embryonic stem cell lines are listed, in 
reality only 22 lines are currently available to researchers. 
Furthermore, scientists have serious concerns about the 
quality, longevity, and availability of these existing lines 
because they were grown in culture dishes coated with mouse 
cells which have contaminated them. These mouse cells helped 
them to generate, but they also eventually create 
contamination.
    At the time this method was created, the mouse cells were 
necessary. However, in a dramatic new achievement earlier this 
year, scientists were successfully able to maintain stem cell 
lines without using animal feeder cells. In order to allow 
researchers the opportunity to fully explore the possibilities 
and promise of stem cells, we must ensure they have expanded 
access to these new uncontaminated stem cell lines.
    The Stem Cell Research Enhancement Act, S. 471, introduced 
by Senators Specter, Hatch, Harkin, Kennedy, Feinstein and 
myself, and H.R. 810, introduced by Congressman Castle and 
Congresswoman DeGette would allow research to receive Federal 
funding for the study of embryonic stem cells derived from 
excess embryos created for fertility treatments and willingly 
donated by patients. Last month, H.R. 810 passed the House on 
May 24, 2005, by a vote of 238-194, and it is now time for the 
Senate to act.
    I am also currently working on legislation titled ``The 
Stem Cell Research Investment Act,'' which would buildupon S. 
471 to promote cutting-edge research to fight devastating 
chronic diseases and health conditions. Modeled after 
California's recently passed Proposition 71, the bill 
encourages States to issue up to $30 billion in zero-interest 
bonds to fund their own stem cell research initiatives and 
provides bondholders a Federal tax credit in light of interest 
payments. As with S. 471, such funding could only be used for 
embryonic stem cell research that uses embryos that are bound 
for destruction from fertility clinics donated by patients.
    In the field of medicine, there is no such thing as a 
Republican science or a Democratic science. There is just 
science. New advances in technology have allowed us to 
understand the nature of the human body like never before, and 
with it the ability to prolong life and to cure disease. 
Responsible research grounded in the roots of scientific 
principles and conducted with the ultimate goal of saving life 
must be allowed to flourish. We owe a moral obligation to the 
sufferers of these debilitating diseases and their loved ones 
to provide our best and brightest scientists with the tools 
they need to undertake their quest in a safe and ethical 
environment free from unnecessary Government barriers but with 
moral parameters.
    I eagerly await the testimony of our experts who understand 
the financial, emotional, and physical costs of these diseases 
and who are among the leaders in cutting-edge research that is 
being done in this field. It is my hope that by the end of 
today's hearing we will have a greater understanding of 
embryonic stem cell research and a deeper appreciation of the 
incredible potential of this exciting branch of medicine.
    It is my privilege now to turn to my colleague and friend, 
Senator Herb Kohl, the ranking member of this committee. 
Senator Kohl.

          OPENING STATEMENT OF SENATOR HERBERT H. KOHL

    Senator Kohl. We thank you, Mr. Chairman, for holding this 
very timely hearing today, and we welcome our witnesses who 
will be testifying today. Every family in America has 
experienced the tragedy of watching a loved one suffer through 
a deadly or debilitating illness. Diseases like Parkinson's and 
Alzheimer's take a terrible toll on families' lives and 
livelihoods. While we have made great strides in biomedical 
research in recent years, we still do not have all the keys to 
unlock the secrets of disease, and that is why the potential of 
embryonic stem cells is so exciting. Embryonic stem cells have 
the ability to develop into virtually any cell type in the 
human body. Scientists tell us that harnessing the power of 
these cells could one day lead to new treatments and maybe even 
cures for a number of diseases that afflict American families.
    Important research is being done every day on stem cells, 
and I am proud that some of this research is being done at the 
University of Wisconsin in Madison, which was the first to 
isolate human embryonic stem cells. So we are pleased to have 
Dr. Su-Chun Zhang from the university's Waisman Center here 
today to testify on the ground-breaking work that he and his 
colleagues are doing.
    We all understand, of course, that this research is not 
without controversy. We respect the concerns that some people 
have about the use of embryonic stem cells in research, and we 
agree that we must closely monitor this research to ensure that 
it is done ethically. However, scientists and disease advocates 
are warning us that the current limits on Federal funding for 
stem cell research are seriously inhibiting our potential to 
find new cures. Without expanded Federal support, we do risk 
slowing down the tremendous progress that could be made to 
alleviate human suffering. It would not be right for the 
Federal Government to turn it back on the discoveries that 
expanding stem cell research promises.
    Now more than ever it is important to grasp this 
opportunity in an ethical manner by making sure that 
potentially life-saving research does keep moving forward. So 
we look forward to hearing from our expert panel today and 
hearing their recommendations. Again, we all thank you, Mr. 
Chairman, for holding this hearing.
    The Chairman. Thank you, Senator Kohl.
    We are joined by two of our colleagues, Senator Collins of 
Maine and my colleague from Oregon, Senator Wyden. So, Senator 
Collins, you are next, and then Senator Wyden.

         OPENING STATEMENT OF SENATOR SUSAN M. COLLINS

    Senator Collins. Thank you, Mr. Chairman.
    I want to begin by commending you for holding a hearing on 
an issue of profound importance to this country. The title for 
this hearing that you have chosen is highly appropriate. 
Embryonic stem cell research does indeed hold tremendous 
promise to treat and possibly even cure a vast array of 
devastating diseases and conditions. It is a promise that must 
be explored. From Alzheimer's to Parkinson's and ALS, to 
cardiovascular disease, diabetes, and cancer, this research 
offers great hope to our seniors and their families.
    For some seniors, these devastating diseases can turn their 
last years of life into a time of suffering and misery. The 
potential of this research to relieve this suffering and misery 
is far too great for us not to explore it. But this research 
will not just benefit our seniors. As the founder and the co-
Chair of the Senate Diabetes Caucus, I am particularly excited 
about the promise that stem cell research holds for a cure for 
juvenile diabetes. This disease has had such a profound effect 
on more than 1 million American children and their families. It 
condemns far too many of them to a future of heart disease, 
stroke, kidney failure, blindness, and amputation.
    We simply cannot ignore the potential that stem cell 
therapy holds for these young people, and indeed in two weeks' 
time, I will be chairing the biennial Children's Congress where 
children with diabetes come from every State in the Nation. It 
is so important because they put a human face on this debate.
    Of course, we know that this research offers the 
possibility of recovery or at least better treatment to people 
of all ages who have suffered devastating spinal cord injuries. 
We are now engaged in a great national debate over whether this 
vital research can proceed at a vigorous pace given the 
administration's decision to make Federal funding available 
only for that research using embryonic stem cell lines that 
existed before August 2001. As the chairman indicated, many 
scientists contend that the existing lines are contaminated 
with mouse cells and that severely compromises their potential 
therapeutic values for use in humans. I look forward to hearing 
the testimony from our experts today on that crucial issue.
    This debate is often portrayed as a choice between 
scientific advancement on the one hand and medical ethics on 
the other. I believe that that is a false choice. I believe 
that we can advance this vital research and at the same time 
maintain the highest ethical standards.
    Last month, the House of Representatives took an important 
step in resolving this debate. The legislation passed in the 
House would expand the current restriction on Federal funding 
for embryonic stem cell research to the more than 400,000 cells 
unused in fertility clinics that would be made available by 
willing donors. I would point out that these are cells that 
otherwise would be discarded. Isn't it far better to put them 
to work to help advance this research?
    The House legislation is the result of bipartisan 
cooperation and compromise. As the chairman indicated, there is 
similar legislation in the Senate which we have cosponsored 
along with our colleague Senator Specter. This legislation also 
makes available to researchers that vast number of unused 
cells, and it respects the ethical considerations that are such 
an important part of this research.
    I believe that the ethical thing to do is to move forward 
with this research, and I want to again thank the chairman for 
his leadership in making this research possible.
    The Chairman. Thank you, Senator Collins. I think the issue 
is often cast as a Republican or a Democrat issue. Actually, I 
think we have proven that this is a human issue.
    Senator Wyden.

             OPENING STATEMENT OF SENATOR RON WYDEN

    Senator Wyden. Well, thank you, Mr. Chairman. I would like 
to characterize it as an Oregon issue as well because you have 
done a tremendous job in terms of leading the committee. I also 
want to say how much I appreciate Chris Dudley being here. 
Chris Dudley is involved in just about every good cause in our 
home State, and we are just thrilled to have him and appreciate 
all he has done.
    Mr. Chairman, I am going to have to be in the Intelligence 
Committee and I think a couple of other committees in the next 
10 minutes, and I am not going to be able to stay. I just 
wanted to come by and make a point or two on behalf of the 
leadership that you are showing.
    The principal thing that I wanted to touch on is that the 
opponents of embryonic stem cell research seem to be arguing 
now that the reason this research should not go forward is that 
there are not enough medical discoveries or cures using 
embryonic stem cells. I guess if you follow that kind of logic, 
some of the opponents of embryonic stem cells would have 
criticized the Wright brothers for not launching a moon flight 
when they took their very first flight.
    The fact of the matter is you have got to let science 
advance. Scientific research takes time and money to develop 
treatment and cures, and the fact is that embryonic stem cell 
research has been hamstrung by limiting the cell lines and not 
giving Federal funds to non-approved stem cell lines.
    So I think we also ought to note that there is progress 
being made in adult stem cells. It is important that that work 
continue. But in the Commerce Committee, we examined some of 
the limits on what adult stem cells could do, and it is clear 
that the country ought to go farther.
    Now, I would be shocked, I guess, to say that politics is 
involved. We all remember that line from ``Casablanca.'' But 
suffice it to say nobody can be naive. There is politics in 
this debate, and I would just hope that people would pause a 
little bit because in an age when there is not a lot of 
bipartisanship, when there are not enough legislators who are 
doing what Chairman Smith is doing, what Senator Collins is 
doing, this is a piece of legislation where there is true 
bipartisan support. I think that that is the case because this 
country wants science to be science. This country does not want 
science to be seen through a political prism. So what you have 
are Senators who want to pursue science in that kind of 
approach.
    I commend you, Mr. Chairman, and I apologize to our friend, 
Chris Dudley, for not being able to stay throughout the 
afternoon. But I know that the Oregon juggernaut, with the 
chairman and Chris Dudley, is going to prosper in terms of 
advancing this cause, and I thank you both for all you are 
doing, and my colleagues as well.
    Thank you.
    The Chairman. Thank you, Ron.
    We have been joined by Senator Carper of Delaware, so we 
welcome your statement if you have one.

         OPENING STATEMENT OF SENATOR THOMAS R. CARPER

    Senator Carper. I am not from Oregon. [Laughter.]
    But I have been to Oregon, and I must say I liked it a lot. 
In fact, I thought about going to graduate school there when I 
got out of the Navy in California, and they told me they would 
never let me be a Senator, Governor, or any of that stuff. So I 
decided to find a smaller State. But we are glad that you are 
here and look forward to hearing from you.
    I have a statement I would like to enter for the record, 
but let me just make a brief personal comment if I may.
    My mom passed away a couple of months ago. She had 
Alzheimer's disease and congestive heart failure and arthritis 
and all kinds of maladies. She lived to be about 82, a full 
life, but the last 6 years she was in a wonderful facility in 
Ashland, KY, called Woodland Oaks, where they took great care 
of her. She lived close to my sister and closer to my mother's 
sister. But she had Alzheimer's disease, and it sucked away her 
vitality and a special part of her life in her later years. Her 
mom had had Alzheimer's disease. Her grandmother had 
Alzheimer's disease as well.
    As we focus on the issues of stem cell research and try to 
apply the research as we develop it, I think of my mother. I 
think of my mother's father, who had Parkinson's disease. I was 
in born in West Virginia, and my grandfather was a butcher. He 
was kind of an amazing guy, and he lived to be about 85 years 
old. But he would drive to work at Patton's Market, which is 
one exit off the West Virginia Turnpike from Robert C. Byrd 
Drive. My grandfather would drive up to Harper Road, and his 
hands were shaking just like this. I always remember when I was 
a little boy going to visit him and wondering how will he ever 
go into the butcher shop and not cutoff a finger or a thumb. He 
would get in the butcher shop and he was like a rock for the 
rest of the day until it came time to go home. Then he would 
have what we would call palsy or the trembles.
    I remember my grandfather who made the best of the hand 
that he was dealt and stayed with it for a long, long time. Not 
everybody, including a fellow I had a chance to spend some time 
with at lunch on Monday in Philadelphia, not everybody is as 
lucky as my grandfather to be able to go that long and that 
hard.
    Everybody here, probably everybody on the panel, I guess 
everybody in the room, can talk about their own mom or dad or 
their aunt or uncle or their grandparent and how their lives, 
their quality of life has been diminished because of their 
battle with a disease that I think can be tamed, can be cured, 
or at least delayed through the kind of research that would be 
enhanced by the legislation that my Congressman, Mike Castle, 
has introduced and championed in the House. I am pleased to 
cosponsor it here in the U.S. Senate.
    So I would say, Mr. Chairman, thank you for pulling this 
all together and for letting a couple of guys who are not from 
Oregon say a few words and to say hello and good work. Thanks. 
Welcome, Chris.
    The Chairman. Thank you, Senator Carper, and we will 
include your full statement in the record as well.
    [The prepared statement of Senator Carper follows:]

              Prepared Statement of Senator Thomas Carper

    Mr. Chairman, thank you for holding this hearing on the 
potential of stem cell research, a topic that is very important 
to many people in Wisconsin. I commend you and the Ranking 
Member for providing leadership on this issue.
    I am pleased that the Aging Committee is providing a forum 
for some of the country's leading researchers to speak about 
the progress they are making with embryonic stem cell research, 
and the limitations that currently impede their ability to 
further advance this research.
    I am especially proud that Dr. Su-Chun Zhang of the Waisman 
Center at the University of Wisconsin is here to describe 
firsthand his experience in using embryonic stem cells to 
further understand degenerative diseases and medical conditions 
such as Parkinson's, ALS, MS and spinal cord injuries. I thank 
Dr. Zhang for his contributions to these efforts, and for 
agreeing to take time from his valuable work to appear before 
this Committee.
    Dr. James Thomson at the University of Wisconsin first 
broke ground in this amazing research, and with the help of 
talented researchers such as Dr. Zhang, Wisconsin continues to 
be a proud leader in this field.
    Embryonic stem cell research holds the potential for better 
understanding, and possibly developing cures and treatments 
for, many fatal and debilitating diseases and medical 
conditions. That is why I have cosponsored S. 471, the Stem 
Cell Research Enhancement Act of 2005 introduced by Senators 
Specter and Harkin. This bill would help our nation's 
researchers unlock that potential by increasing the quantity 
and quality of stem cells lines available for research.
    There is much work that needs to be done to further 
understand the role that embryonic stem cells can play in 
providing answers to some of the most troubling medical 
diseases and conditions that affect so many Americans. Limiting 
our ability to find these answers when researchers are only 
starting to make headway would be a huge step backwards for the 
many Americans who could benefit from this groundbreaking 
research.
    Embryonic stem cell research could very well be the gateway 
to finding treatments or cures for diabetes, heart disease, 
ALS, spinal cord injuries and other medical conditions that 
millions of Americans currently suffer from. I will continue to 
support this incredibly important science which would expand 
our research horizons, and bring hope to so many people.
    I was very pleased to see stem cell legislation pass the 
House, and I am proud to be part of the bipartisan effort to 
expand federal funding for embryonic stem cell research in the 
Senate. It is my hope that the Majority Leader will soon bring 
this bill to the Senate floor for a vote, and that the Senate 
will overwhelmingly pass this legislation.

    The Chairman. It is not a requirement to be from Oregon to 
be heard today, but our first witness is from Oregon, and he 
will be joined by a second panel of very distinguished 
scientists and physicians who will hopefully illuminate us on 
the promise that embryonic stem cell research may offer.
    To introduce Chris Dudley, I think many of our audience 
would recognize and remember him from his stellar career as an 
NBA center. He was with the Portland Trailblazers through some 
of their brightest days, and after this last season, they could 
use your help again, Chris. But as the owner of the Milwaukee 
Bucks is right here, he probably does not want to see you 
return.
    Chris Dudley understands firsthand the enormous financial, 
physical, and emotional costs of a particular disease, that is, 
diabetes. He was diagnosed with that affliction at age 16, and 
yet he nevertheless went on to achieve remarkable success in 
athletics.
    Chris' desire for every child to succeed regardless of 
their economic, education, or health liabilities inspired him 
to create the Dudley Foundation in 1994, which includes the 
Chris Dudley Basketball Camp for Kids with Diabetes in 
Vernonia, OR. His personal story will underscore the importance 
of exploring all scientific avenues, including human embryonic 
stem cell research, to prevent and find a cure for diabetes.
    Chris, thank you for coming this long way to participate in 
this hearing.

   STATEMENT OF CHRIS DUDLEY, DIABETES ADVOCATE, AND FORMER 
          CENTER, PORTLAND TRAILBLAZERS, PORTLAND, OR

    Mr. Dudley. Thank you. Good afternoon, Senator Smith and 
members of the committee. Thank you for the invitation to 
appear before your committee today to tell you about how living 
with juvenile diabetes has affected my life and the lives of so 
many children that I have met over the years through my 
foundation and at my basketball camp.
    My name is Chris Dudley, and I played professional 
basketball for 16 years with Cleveland, New Jersey, the 
Portland Trailblazers, and the New York Knicks. I am the proud 
father of three wonderful children ages 6, 5, and 3 and husband 
to wife Christine. I also have juvenile diabetes.
    I was diagnosed at the age of 16. I had the classic 
symptoms of excessive thirst and having to go to the bathroom 
constantly. My uncle also has diabetes, so my dad recognized 
the symptoms, luckily, and brought me home a test kit, and it 
showed that my blood sugar was extremely high. We immediately 
went to the hospital, and I was diagnosed with diabetes.
    When I first heard the news, I was devastated. I did not 
really know enough about the disease, and I was terrified that 
I would no longer be able to play basketball. In fact, my dad 
tells the story that the first question I asked was would I 
still be able to play basketball.
    I was fortunate that the doctors and nurses said that I 
would be able to continue to play if I was careful about 
monitoring my blood sugar, and this to me was a tremendous 
relief. I thought if I can keep playing, I can go forward.
    This is not always the case. Many times kids with juvenile 
diabetes are not encouraged to keep playing sports because of 
fears of what can happen, especially from low blood sugars. I 
was also fortunate to have a supportive family that encouraged 
me to continue to play basketball and not let diabetes stop me 
from doing what I loved.
    After my diagnosis, I really looked up to people like Bobby 
Clarke, Hall of Fame hockey player for the Philadelphia Flyers, 
who had diabetes and also of a triathlete--I was then living in 
San Diego--who had diabetes. I felt at that time it was a 
tremendous help for me to realize that if Bobby Clarke can play 
Hall of Fame hockey and this other person can run a triathlete, 
then I can play JV basketball.
    Ever since that time, I have been an outspoken advocate for 
encouraging kids with diabetes to pursue their passions--
whether it be sports or other activities--provided that they 
take care of their diabetes. That being said, that provision is 
a hard one. Diabetes is such a hard disease because you have to 
stay on top of it every hour of every day, or you can face 
serious complications. It is a disease that is 24/7 and takes 
no breaks. Diabetes never stopped me from playing basketball, 
but by no means was it easy. There were many times when the 
disease did hinder my performance.
    When I was playing in the NBA, I would have to test my 
blood sugar 14 times on a game day and take multiple insulin 
shots. When you are preparing to play in front of 20,000 
people, you want your sugar--blood sugar level--to be as close 
to the ideal as possible. This is very difficult to do, and 
some days no matter how hard you try, it is never perfect. It 
took a lot of practice and monitoring, but I was able to play 
to the best of my abilities regardless of my diabetes. I was 
fortunate that my teammates were also supportive. It was 
through my teammates that I realized how widespread this 
disease really is. I was amazed at how many teammates would 
have a father, brother, sister, uncle, grandfather who had some 
connection to diabetes. I was fortunate that I always felt 
there was a great understanding and appreciation of what I had 
to go through every day just to be able to play basketball.
    I also had my battles with diabetes. In college, I was in a 
car accident. After working out, my blood sugar dropped 
dangerously low and I ran into a parked car. That is one of the 
dangers of what can happen when you have diabetes, and with 
working out your blood sugars can drop dramatically. I have had 
diabetes for 24 years, and I have had that constant worry about 
the long-term risks and what the disease is doing to my body.
    Now that I am retired from the NBA, my passion is my 
family--my three children--and advocating on behalf of research 
to get us to a cure for juvenile diabetes as soon as possible 
and enabling kids who already have diabetes to be able to 
pursue their dreams. I started the Dudley Foundation in 1994 
and the Chris Dudley Basketball Camp for Kids with Diabetes in 
Vernonia, OR, in 1994. At the camp, I see firsthand what these 
kids--some of them very young--have to go through every day. 
Some struggle much more than others, not because they are being 
lazy about monitoring their blood sugars, but because it is 
just more difficult for some kids to keep their sugar levels in 
range, as hard as they try. At that age, you have hormones, you 
have stress, colds. Anything can throw your blood sugars out of 
whack.
    When I talk to kids with diabetes and work with them at 
camp, I walk a fine line. I want to show them that the diabetes 
does not have to stop you from doing whatever it is that you 
want to do; but, on the other hand, I know that it's not easy 
for them and that they will never get a day off from this 
disease. It is not easy for their parents either. Parents of 
the kids who come to my camp tell me that it is the only week 
throughout the entire year that they can sleep through the 
night without having to get up to check on their kids and check 
their blood sugar levels. To me that is just amazing as parent.
    I worry every day that one of my kids will be diagnosed 
with juvenile diabetes. Even though I have been very blessed in 
my life and have been able to achieve some great things even 
with diabetes, this is not the life I want for my children. I 
am missing my 6-year-old son's kindergarten graduation to be 
here today, but I explained to him that being in Washington was 
my opportunity to help people understand why a cure for 
diabetes is so important. I want this cure for the children who 
come to my camp, my children and your children.
    Last August, I received an award, a Freedom Corps Award, 
from President Bush for my camp and the foundation. I had the 
opportunity to travel with President Bush and Leery Bush in 
Portland that day. I told them what it was like to live with 
juvenile diabetes and the struggles the kids who come to my 
camp face every day. I also told them that even though I share 
and empathize with some of their same concerns, I believe that 
there is an ethical compromise that will allow the tremendous 
potential of stem cell research to flourish. Research is the 
only avenue to the cures and therapies for diabetes and many 
other diseases, and we should pursue this promising research 
aggressively within an appropriate ethical framework.
    I want to be able to tell the children I see--and I see a 
lot of them--with diabetes and tell them with a straight face 
that in this great country we are doing everything possible to 
find a cure and that help is on the way.
    Mr. Chairman, thank you again for this opportunity. It has 
been an honor to appear before you today.
    The Chairman. Chris, just as an Oregonian, I just have to 
tell you how proud I am of what you do and your basketball 
camp. Obviously, these kids that you take into the camp--do 
they all have childhood diabetes?
    Mr. Dudley. Yes, they do. That is a requirement of camp.
    The Chairman. That is a requirement.
    Mr. Dudley. Yes.
    The Chairman. Would you just repeat once again the comment 
of the parents, that they are saying this is the one week in 
their life that they do not have to worry about their 
children's blood condition during the middle of the night.
    Mr. Dudley. Absolutely. One of the greatest fears of 
parents is that during the night while their child is asleep, 
they will have a blood glucose reaction; their blood sugar will 
drop during the night. So the parents get up and check them 
during the middle of the night. Especially with the younger 
ones, parents tell us that this is the only week that they can 
sleep through the night because they leave these kids with us 
for the week. It is really two camps in one. It is a basketball 
camp with a basketball staff, and it is a diabetes camp with a 
diabetes staff, and doctors and nurses and counselors. We test 
them during the night, and we take care of everything. In fact, 
the parents have to check their children into the camp, and 
then they check them out, and they meet with our doctors.
    To get back to your point, they feel comfortable leaving 
their kids with us during that week, and this is the only week 
of the year that some of them actually sleep through the night.
    The Chairman. How many kids do you have?
    Mr. Dudley. We have 75 kids, and that is the most we are 
able to have because of just handling the medical requirements, 
boys and girls, 10 through 17. We have figured out that this is 
the tenth year. Throughout the years, we have had a child I 
think from every State in the Union except----
    Senator Carper. Not Delaware?
    Mr. Dudley. Mississippi. No, we have had Delaware. 
[Laughter.]
    Mississippi is the one we have got to work on. I think it 
was Mississippi and one of the Dakotas. But we have had pretty 
much the whole United States covered.
    The Chairman. Yours is the only camp in the country of this 
kind?
    Mr. Dudley. It is the only camp in the country of its kind 
that is basketball and diabetes. One of the more--there are 
other diabetes camps, but they are more along the lines of 
regular camp, arts and crafts, and this is more of a sports 
camp we go after it. It is a real basketball camp, and the kids 
go hard, and they learn how to handle their diabetes while 
going hard.
    The Chairman. Well, we are just thrilled that this hearing 
can help spread the message of the remarkable service that you 
provide, and I cannot thank you enough. I do want to ask you 
one question. I understand you describe yourself somewhat, as I 
do myself, a pro-life Republican, and sometimes you are asked 
to justify that. I obviously have my own reasons for being 
supportive of embryonic stem cell research. Could you share 
with the committee why you think it is not inconsistent to be 
pro-life and pro-embryonic stem cell research?
    Mr. Dudley. Yes, I can, and that is obviously not an easy 
issue. When you say pro-life--I try not to get painted into 
either corner but--because I feel like there are exceptions. 
But for the most part, I am a Christian, and I believe in life, 
as I am sure we all do here today. It becomes a difficult 
question, and I have to wrestle with it when taking a stand for 
this. I think there are--I think the fact that these are 
embryonic cells that are going to be discarded anyway, that it 
is just a shame and a lost opportunity to not protect the life 
that is already with us today.
    The Chairman. Thank you very much, Chris.
    Senator Kohl.
    Senator Kohl. Thank you very much, Mr. Chairman, and it is 
a particular delight, as I pointed out to you before, that we 
convene today, Chris, to have you here, to break bread and to 
make peace with you for all the torment that you inflicted on 
my team over your years in the NBA. [Laughter.]
    As I said to Chris, the only redeeming feature of Chris' 
career with respect to us is that he could not make a free 
throw. [Laughter.]
    Mr. Dudley. Thanks for bringing that up.
    Senator Kohl. Otherwise, he was and is a great guy and a 
great player.
    For those of us who do not know all that much about 
diabetes in terms of its daily treatment, you talked about 
during your career--is it still true today?--that you need to 
monitor your condition? Explain that one. Do you need to keep 
in touch with physicians?
    Mr. Dudley. Yes.
    Senator Kohl. How does that work for a person with 
diabetes?
    Mr. Dudley. I take anywhere from four to six insulin shots 
a day, test my blood sugar six to ten times a day. I think I 
did a little--I got the calculator out last night and figured 
that over my lifetime I have taken over 35,000 shots of 
insulin. So it is a disease that does not go away.
    Senator Kohl. It does not go away.
    Mr. Dudley. It does not go away.
    Senator Kohl. Is that true about virtually all people who 
have diabetes?
    Mr. Dudley. Well, there are two types of diabetes. There is 
Type I, which is what I have, which is commonly called juvenile 
diabetes, where my pancreas does not produce any insulin. Type 
II, which is also called adult onset, although that has changed 
because now it is becoming more common with younger and 
younger--it is really becoming an epidemic in this country. 
Their pancreas produces some insulin, but either not enough or 
their body is resistant to it. A lot of times they can take 
pills, exercise, diet, and then it becomes shots. So there are 
two types. But my body does not produce insulin and will not 
produce insulin.
    Senator Kohl. Currently is there any hope out there beyond 
embryonic stem cell research?
    Mr. Dudley. Well, that is a tough question because people 
are working on transplants and they are working on different 
areas, and there is hope, but it is not a near-term hope. You 
know, I have had diabetes since 1981, and you always have to be 
careful about saying there is too much hope because a lot of 
people with diabetes have thought that we were that close to a 
cure many times, and that is a tough thing, especially at a 
younger age, to be told that is coming around the corner and 
then it does not come. So, yes, I think there is hope, but how 
close, I don't know. This seems to be the most promising, the 
greatest potential Senator Kohl. Out there right now.
    Mr. Dudley. Out there right now.
    Senator Kohl. So that it is fair to say for those with 
diabetes stem cell research is a huge, huge part of their 
hopeful future.
    Mr. Dudley. Oh, absolutely. I think with diabetes and a 
number of diseases, there is such a tremendous potential with 
stem cell research that, yes, it is a huge part.
    Senator Kohl. Thank you.
    Mr. Dudley. Thank you.
    Senator Kohl. Good to see you again, Chris.
    Mr. Dudley. Good to see you, too.
    The Chairman. Senator Collins.
    Senator Collins. Thank you.
    Mr. Dudley, when I heard your statement about diabetes 
being 24/7, I was reminded of the first time that I met a 
family with a son who is age 10 who had diabetes. He looked up 
at me, and he said that his greatest wish was that he could 
just have one day off from his diabetes. He said, ``If only I 
could take Christmas off or my birthday off.'' That just 
touched me so deeply. In fact, it led me to be the founder of 
the Diabetes Caucus in the Senate, to see this 10-year-old boy 
having to struggle with the treatment of his disease and never 
being able to take a day off. So I think your testimony just 
reminded me so much of that.
    It is one thing to ask an adult to struggle with a disease 
24/7, but to ask a little child to have to bear that just is so 
painful. It is one reason that I have been such an advocate of 
stem cell research.
    The issue that I want to bring up with you is whether you 
could help us better understand--and I know subsequent 
witnesses will--why stem cell research holds particular promise 
in the treatment of juvenile diabetes. Is it the hope that 
there could be islet cells produced from stem cells that could 
then be transplanted? Or what is the theory that makes stem 
cell research particularly important in the treatment of 
diabetes?
    Mr. Dudley. Yes, I would like to hit two points, and the 
first with your last point. I think it is becoming that islet 
cells are the Holy Grail, so to speak, but I better--because we 
have these three gentlemen here, I think they would be better 
served going into the detail on that. I don't want to be 
exposed by them later. [Laughter.]
    On your first part about the children, I really--for me I 
want a cure personally, but that is not why I am here, because 
I have lived with it. I want it for the children, because I get 
so touched by dealing with the parents and the children at 
camp, it is like you said. I have had that same experience, and 
it really just does, you know, melt you down. I really believe 
we need to do everything possible to cure it for this children, 
because they want just one day, you know, to have a normal day.
    You know, I realize--and it is so difficult for them to 
deal with it on a constant level. One of the benefits from the 
camp that I did not know about or did not know how great it 
would be until I started the camp was a lot of these kids come 
from cities or towns or schools where they are the only one 
with diabetes. They feel so alone out there. They are just 
battling this and going through it, and that is one of the 
greatest benefits of the camp, was for them to see that there 
are other kids who are wearing the same shoes with them, are in 
the same boat. I think that really helped them. But they are 
all just great, great kids, and I think maybe it is something 
about having to deal with adversity at such a young age. But 
they are tremendous kids, and there is nothing that--I mean, 
there is nothing that would be better than to cure this disease 
and let them live a normal life. I cannot imagine a greater 
thing. I really appreciate your help, what you have done 
through the years for the Diabetes Caucus.
    Senator Collins. Thank you, and thank you for your 
testimony today.
    Mr. Dudley. Thank you.
    The Chairman. If we find that cure, Chris, I assume that 
the basketball camp will just be more broadly attended.
    Mr. Dudley. Exactly. It will be a little bigger.
    The Chairman. Senator Carper.
    Senator Carper. Thanks, Mr. Chairman.
    Before I ask some serious questions, let me ask one that is 
not so serious. Who are you putting your money on in the NBA 
finals? [Laughter.]
    Mr. Dudley. You know, my pick before it started was San 
Antonio, so I have got to go with them. I have got to keep 
going with them.
    Senator Carper. All right. We are going to see some good 
defense.
    Mr. Dudley. We are going to see a lot of good defense.
    Senator Carper. That is for sure. None of them can make 
foul shots, though.
    Mr. Dudley. No--well, a couple of them can.
    Senator Carper. A more serious question. Going back to your 
childhood, I understand you were diagnosed when you were 16 
with juvenile diabetes.
    Mr. Dudley. Yes.
    Senator Carper. Did you have some inkling beforehand? You 
did not just wake up someday and say, ``Boy, I am thirsty and I 
have got to go to the bathroom.'' Kind of just tell us how it 
happened.
    Mr. Dudley. I was working, playing basketball every day 
after school, and going to school. It was after the season was 
over. All of a sudden, I just had to start drinking and I was 
just so thirsty. I would come home and, I mean, literally drink 
half a gallon of whatever it was. Usually, especially in 1981, 
that ``whatever it was'' was Gatorade or Kool-Aid or whatever 
and it had sugar in it, so that made the situation worse. By 
drinking this, you just have to go to the bathroom every 5 
minutes and race into the bathroom and not feeling well and 
just know something is definitely wrong. I was fortunate that--
my dad's brother has diabetes. I was fortunate in the fact that 
my father----
    Senator Carper. He had juvenile diabetes?
    Mr. Dudley. Yes, he had juvenile diabetes and had struggled 
with it. Fortunately, technology has gotten a little better, 
but he had really struggled with it. My father knew that those 
were some symptoms, and he went to the drug store and got a 
little home tester and saw that my blood sugar level was sky 
high and raced me to the hospital. I was fortunate that we 
caught it when we did then. There are cases where, because you 
are so thirsty, people pass out and go into a coma because they 
just keep drinking, and what they are drinking has sugar in it 
and is just making the situation worse and worse. The reason 
you are so thirsty is your body is trying to flush out that 
sugar, and so it is really just trying to do it but it cannot.
    Senator Carper. OK. I think you said that for kids who have 
juvenile diabetes, their pancreases do not create insulin. Your 
pancreas created no insulin or small amounts or diminishing 
amounts over time? How did it work?
    Mr. Dudley. It is my understanding that it produces no 
insulin. It just for whatever reason stops producing insulin. 
Where people with Type II diabetes produce some insulin, are 
capable of producing insulin, Type I diabetics to my 
understanding, there is no insulin; or at least if there is, it 
is so negligible it does not make a difference.
    Senator Carper. For a person whose pancreas produces no 
insulin, how would, in theory, embryonic stem cell research 
applied actually make a difference?
    Mr. Dudley. Well, again, I think this will get touched on 
later, but I believe it is going to be to transplant islet 
cells which help produce the insulin. So by having those 
transplants, they will be able to make my body have the 
capability of producing insulin.
    Senator Carper. You mentioned at some point in your 
testimony about flying out, I think you said, to Oregon with 
President and Mrs. Bush?
    Mr. Dudley. I was in Portland, OR, and when the President 
traveled in different States, they recognize--it is part of the 
Freedom Corps Act, and I was recognized for the State of 
Oregon. So I met him and the First Lady in August of last year, 
and I brought out one of my campers and we went out and met the 
President. Then I was able to travel with him a little bit--not 
with him but with the party. I was able to talk to the First 
Lady a little bit about it. She had just gone through a tragic 
loss, I believe, at that time. I think it was her father. So we 
were able to talk about the issue, and she was very sympathetic 
and understanding. Obviously there are differences, but it is 
kind of my belief that this is not a partisan issue. It is a 
scientific issue, and there has got to be a way of figuring it. 
There has got to be a compromise in there somewhere that makes 
sense to help people.
    Senator Carper. Some of us think that the compromise 
crafted by my friend and colleague in the House, Mike Castle, 
comes pretty close to a fair compromise. I do not know how 
familiar you are with this, but----
    Mr. Dudley. You know, I have looked at it, I mean the rough 
sketch of it, and I think it makes a lot of sense. I really do. 
I think my feeling is that if you are going to take a hard line 
against it, then you have got to get rid of in vitro in the 
first place, that it is all or nothing; that if you are doing 
in vitro, then it makes sense to use these excess embryos for 
scientific research. If you follow the argument that every 
embryo should be used, then we should not have in vitro in the 
first place. That is kind of where I fall on it.
    Senator Carper. One last question if I could, Mr. Chairman, 
and this may be a question that would be better directed to our 
next panel of witnesses. But let me just run it by you, and if 
you have any thoughts on it, fine. If not, we will just hold it 
in abeyance for now. But with regards to adult and cord blood 
stem cells, I am a layman at this sort of thing, as others are 
in the room, but any thoughts with respect to the kind of 
benefits that they may be able to provide and may not be able 
to provide in comparison to embryonic stem cells?
    Mr. Dudley. Yes, sure. From what I understand, they hold 
potential but not nearly as much, that they are not as--I don't 
know the terms, but multifaceted. They are not as able to 
become as many things. There are a lot more issues with them, 
and it is not something that I think should be either/or. I 
think it should be both. I think you should look at both. I 
think you have to go with the most promising, which is what we 
are talking about today, but that does not mean drop the other 
ones, because adult stem cell I believe has been around for 
quite some time, and it has some purposes but does not hold 
nearly the potential that what we are talking about today does.
    Senator Carper. Well, by watching you testify, I get to see 
some of the next panel of witnesses behind you, and it is 
interesting watching them nodding their heads. You might have 
gotten it right.
    Thanks so much for being here, and thank you for the 
example you provide for all of us in what you are doing for a 
lot of kids.
    Mr. Dudley. Thank you for having me.
    The Chairman. We have been joined by another First Lady, 
the Senator from New York. Senator Clinton, if you have a 
statement and/or questions, we would be happy to receive them.
    Senator Clinton. Mr. Chairman, I would just ask unanimous 
consent to submit my statement for the record.
    The Chairman. Without objection.
    [The prepared statement of Senator Clinton follows:]

          Prepared Statement of Senator Hillary Rodham Clinton

    I'd like to thank Senators Smith and Kohl for convening 
today's hearing on the promise of embryonic stem cell research. 
Like them, I am a cosponsor of S. 471, the Stem Cell Research 
Enhancement Act of 2005. The House companion bill, H.R. 810, 
passed that chamber last month, and I would urge Senate 
leadership to bring it to the floor as quickly as possible.
    When the promise of embryonic stem cell research became 
apparent in the 1990s, the Clinton Administration, working 
through the National Bioethics Advisory Commission and the 
National Institutes of Health (NIH), examined the ethical and 
medical issues involved with such research.
    In September 1999, the National Bioethics Advisory 
Commission released its report, ``Ethical Issues in Human Stem 
Cells Research.'' In this report, it recommended that research 
using cells from embryos created, but not used for, infertility 
treatment, should be eligible to receive federal funding.
    By August of 2000, the NIH had released guidelines for 
research using stem cells. These guidelines would have allowed 
funding for research from lines derived from embryos 
voluntarily donated, with no coercion or financial incentives, 
by couples who had determined, after informed consent, that 
such embryos would not be implanted or otherwise used in their 
fertility treatments--in short, embryos that would, if not used 
for research, be discarded.
    And these recommendations are followed in S. 471, which 
provides for the funding of research conducted in an ethical 
manner according to these guidelines--that is, research on 
lines derived from embryos created for fertility treatments and 
voluntarily donated by parents.
    But, because of this Administration's policy, which 
prohibits federal funding of research on any stem cell lines 
created after August 9, 2001, we are prohibited from funding 
research that would meet the high ethical standards developed 
by both the NIH and independent scientists.
    Instead, federally funded scientists, some of whom are here 
today to testify about their work, are limited to using 
slightly over 20 stem cell lines, instead of the 78 lines 
originally thought to be available. And not all of these lines 
are suitable for research. Some of them may be contaminated 
with mouse feeder cells, which can increase the risk of 
creating strains of diseases which can more easily pass from 
mice to humans.
    It's clear that the Administration's policy is far more 
restrictive than it appeared when first announced. And the 
limited number of cells available for federal funding means 
that we are not fully achieving the promise of these cells for 
research into many chronic, debilitating and fatal conditions.
    And this delay is hurting millions of Americans--those 
living with Parkinson's, Alzheimer's and diabetes, as well as 
their friends, families and caregivers. We have the potential 
to develop treatments, even cures, for these diseases, but we 
can't move forward if we don't have new cells.
    The Administration's stem cell policy is not just limiting 
our ability to discover new treatments for diseases. It is a 
case where ideology is impending science. Without access to 
federal funds for embryonic stem cell research, our scientists 
are falling behind, as researchers in South Korea and other 
countries make advances that our scientists simply cannot.
    I look forward to hearing both the scientific and the 
patient perspective from our panelists today, and I hope that 
with their input, we will learn more about the promise of this 
research to create medical breakthroughs for many diseases.

    Senator Clinton. Mr. Dudley, I want to thank you for your 
very thoughtful testimony and, more than that, for your example 
in your work. You have great intellectual and moral authority 
in how you are addressing this issue, and I agree with you that 
we have a tremendous opportunity here. Starting back in 1999, 
the National Bioethics Advisory Commission came up with the 
formulation that we are discussing now about using excess 
embryos that were created through in vitro fertilization so 
long as the donors with informed consent agreed that they could 
be used for scientific research as opposed to being destroyed. 
The NIH guidelines in 2000 really ratified the Bioethics 
Commission report.
    I think that there is a growing consensus in the country 
that really does cross every kind of line one can imagine, 
particularly partisan lines, that this is a very promising area 
that there is an appropriate ethical framework for us to follow 
in engaging in this research. S. 471, which a number of us 
sponsor, which would put into law the advice of the NIH and the 
Bioethics Commission of 5 and 6 years ago, I think would be a 
tremendous step forward. I believe if we can be successful in 
making that case and passing legislation, as the House recently 
did, I think you will really be entitled to share much of the 
credit for that. We can then hope that we can get about doing 
the research and finding the cures for diabetes and other 
diseases.
    So I just want to thank you for taking your personal story 
and your celebrity and telling it to the world and running the 
camp for the children. It means a great deal. We are very 
grateful to you.
    Mr. Dudley. Well, thank you very much.
    The Chairman. Chris, thank you so very much. God bless you 
and your work.
    [The prepared statement of Mr. Dudley follows:]
    [GRAPHIC] [TIFF OMITTED] 23759.001
    
    [GRAPHIC] [TIFF OMITTED] 23759.002
    
    The Chairman. We will now call forward our second panel. We 
are very fortunate to have a very distinguished group. Lawrence 
S. Goldstein is a Ph.D. from the University of California-San 
Diego, School of Medicine; and Doug Doerfler is the president 
and CEO of MaxCyte in Gaithersburg, MD, testifying on behalf of 
the Biotechnology Industry Organization; and then there will be 
John Gearhart, a Ph.D. from Johns Hopkins University, 
Department of Medicine, Institute for Cell Engineering, 
Baltimore, MD; and Su-Chun Zhang, M.D., Assistant Professor of 
Anatomy and Neurology, Stem Cell Research Program at the 
University of Wisconsin.
    We welcome you all.
    We have by video Lawrence S. Goldstein. We will start with 
him and then go to you, Doctor. So, Dr. Goldstein, if you can 
hear me, we thank you for taking the time to participate in 
this hearing, and we will be pleased to receive your testimony 
now.
    We are just working on the sound a little bit. Now we have 
got it. Go right ahead.

 STATEMENT OF LARRY GOLDSTEIN, PH.D., UNIVERSITY OF CALIFORNIA-
SAN DIEGO, SCHOOL OF MEDICINE, SAN DIEGO, CA; REPRESENTING THE 
               AMERICAN SOCIETY FOR CELL BIOLOGY

    Dr. Goldstein. You can hear me?
    The Chairman. We can hear you.
    Dr. Goldstein. Great. Mr. Chairman, members of the 
committee, I want to thank you for inviting me to testify 
today, and in particular I want to thank you for letting me 
testify by videoconference. I am a professor of cellular and 
molecular medicine at UC-San Diego. I am an investigator with 
the Howard Hughes Medical Institute. My research that is 
relevant to today's hearing is focused on understanding the 
molecular mechanisms that are used to move vital materials 
inside neurons, brain cells, and we study and are trying to 
learn how failures of those movements contribute to the 
development of diseases such as Alzheimer's disease, 
Huntington's disease, Parkinson's disease, and perhaps others, 
including mad cow disease.
    Now, before I tell you about my science, I do want to just 
take a moment and thank you, Senator Smith, and your colleagues 
there for your longstanding support of the Federal investment 
in biomedical research and, in particular, for your leadership 
in developing Federal funding, we hope, for broader areas of 
human embryonic stem cell research. I respect your courage on 
this issue. I know it is not easy.
    With respect to the science, I want to discuss how my 
research is trying to take advantage of the enormous scientific 
and medical opportunity provided by human embryonic stem cells. 
I do want to be cautious. I want to stress that scientific 
progress in the fight against these diseases, particularly 
Alzheimer's disease, is very difficult. This is a hard problem, 
and sometimes our advances are agonizingly slow, even when we 
have the best tools available to us. Importantly, it is very 
hard to guarantee the rate at which we can progress. 
Nonetheless, I and many of my colleagues think that human 
embryonic stem cells potentially hold the key to major advances 
in the search for new understanding of and new treatments for 
these terrible diseases.
    Now, for many diseases, including, for example, juvenile 
diabetes, as Mr. Dudley just testified about, there is great 
enthusiasm for using human embryonic stem cells to replace 
cells that are lost in disease. For Alzheimer's disease, 
however, I think that there may be an even more powerful 
approach to the use of human embryonic stem cells to develop 
new understanding and new therapies. That is what I want to 
talk about today--another way of taking advantage of this 
enormous scientific opportunity.
    Now, before doing that, I need to explain why it is so hard 
to learn what happens, what goes wrong in brain cells in brain 
diseases such as Alzheimer's disease. The bottom line in a 
sense and an important basic principle is that we can rarely, 
if ever, do the kinds of biochemical and cellular experiments 
on brain cells of human patients while they are still alive and 
while they are still in the earliest and, we hope, treatable 
stages of the disease. I think you can understand why a patient 
might not be willing to give their brains to experiments before 
they have died of the disorder. They still need their brain, 
after all.
    So much of what we learn and can learn about the basic cell 
biology and biochemistry of brain cells that have this disease 
comes from studying brain cells from people who have died of 
the disease already and, hence, were in late stages. The 
problem is that we then end up studying the cells in the brain 
after most of the damage has already happened. If you think 
about it, this is in some ways like trying to detect and 
prevent, to learn to detect and prevent plane crashes by 
studying the pattern of wreckage on the ground after planes 
have already crashed. There is a great deal that is missing.
    What we really need in a sense is the black box. We need 
the black box to reveal what went wrong in the earliest stages 
of the disease, the nature of the cellular changes and 
malfunctions, so that we can then learn to treat or prevent 
these diseases. So in our search for understanding of 
Alzheimer's disease, we are effectively looking for the black 
box of this disorder.
    The question then is how to find that because, after all, 
we need to learn what those early changes are so that we might 
learn to fix them.
    Now, a very important approach that we have used for the 
past decade in the fight against Alzheimer's disease is to take 
advantage of the existence of very rare forms of the disease 
that are caused by large genetic changes that give rise to what 
we call hereditary Alzheimer's disease. These large genetic 
changes are in many cases known. So what we can do is we can 
take these large genetic changes, and we can introduce them 
into laboratory animals such as mice. We can then study the 
brain cells from these mice and learn what cellular changes and 
what changes in the brain happen in these mice that have these 
large-scale genetic changes that cause Alzheimer's disease in 
people.
    The problem is that while we have learned a great deal from 
this approach--and, indeed, there are many ideas that my lab 
and others have generated from this approach--I am sure you 
realize that people are not just big mice, and there are many 
important differences in the physiology of our cells and our 
brains that lead us to need to test ideas that come from 
studying mice in human people, human patients, and particularly 
if we are going to develop treatments and drugs. Of course, the 
question then is how to do that, and that is where human 
embryonic stem cells provide what I think is going to be an 
incredibly important tool for doing this. This is what we are 
trying to do now in my laboratory.
    What we are trying to do is to learn to take these human 
embryonic stem cells and invert them into the brain cells, the 
types of brain cells that die and fail to function properly in 
the earliest stages of Alzheimer's disease. Some of the 
properties of these cells make it possible for us to make the 
genetic changes, the large genetic changes in these cells that 
cause hereditary Alzheimer's disease in people. So what we can 
then do with these brain cells in a dish that have Alzheimer's 
disease because of the genetic changes is to study them at 
their earliest stages and test our ideas that come from 
studying mice. Ultimately, we think, as we learn which ideas 
are truly correct, which I hope we will do in the next few 
years, we can use these cells, we believe, to begin testing and 
developing new drugs that we can use to treat this terrible 
disorder, because as you know, we have very little in the way 
of drugs to treat Alzheimer's disease.
    Now, there is a second problem in treating and 
understanding diseases such as Alzheimer's disease where human 
embryonic stem cells also have a major contribution to 
potentially make, and that comes from the observation that most 
Alzheimer's disease is what we call sporadic. It is not caused 
by the large genetic changes that are strictly hereditary. 
Instead, it appears to occur almost randomly. However, there is 
a great deal of evidence that suggests that each one of us has 
different genetic susceptibility or potential genetic 
resistance to the development of this disorder. We think that 
there are many small genetic changes that each of us harbors in 
different combinations that interact together or interact with 
the environment to cause us to either develop or not develop 
this disease.
    The problem is that we do not have a way to study this 
major form of the disease in animals. It is a huge limitation. 
These embryonic stem cells, however, potentially give us a way 
to crack that problem because each different embryonic stem 
cell line has different combinations of these small genetic 
changes. We think that we can convert those cells, these 
different cell lines, to the brain cells that malfunction in 
this disease and study how those small genetic changes lead to 
the different cell behavior, cell function in the disease that 
causes those symptoms. This is where the availability of many 
different embryonic stem cell lines may turn out to be crucial 
in the fight against this disorder because we can begin to 
evaluate how our different genetic constitutions confer 
susceptibility or resistance to this disorder and potentially 
teach us how to predict which people will respond to different 
types of treatment or for whom a particular drug will not work 
and, thus, help us both in the development of clinical trials, 
the development of drugs, and not treating people with drugs 
that are not going to help them.
    Now, obviously, these are very difficult goals. We have to 
work very hard to get there, and we are going to need far more 
than a single scientist laboring in San Diego to make the kinds 
of breakthroughs that are going to be needed on this one 
disorder.
    I want to close with just saying that the ideas that I have 
just described and the approaches that I have just described 
for Alzheimer's disease will, I believe, be very valuable in 
the fight against Parkinson's disease, Huntington's disease, 
Lou Gehrig's disease, and other neurodegenerative diseases 
where we do not even understand them well enough to give them a 
name. But I think in the future, if my colleagues and I have 
the opportunity to do this, we are prepared to devote our lives 
to solving these problems using these methods.
    Thank you for listening to my testimony today, and I would 
be happy to answer questions when you have them.
    [The prepared statement of Mr. Goldstein follows:]
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    The Chairman. Dr. Goldstein, I think in the interest of the 
time we have on this video link, my colleagues and I will ask 
questions to you now before going to our next panel members.
    Dr. Goldstein, obviously California has passed a bond 
initiative, and it is not illegal to do embryonic stem cell 
research in the United States. Has that already provided you 
additional lines? Is that working? Even if it is working, is 
there value in the Federal Government playing a role through 
NIH and other of our research institutions?
    Dr. Goldstein. Yes, Senator, that is an excellent question. 
California is just getting ready and beginning to establish the 
mechanisms for issuing funding. But I think there are many 
important roles--in fact, a much more important role for the 
Federal Government to play relative to the State governments.
    First of all, even the availability of State funding in 
California will not solve many of the problems. The Federal 
Government has been a longstanding funder of basic biomedical 
research since the Second World War. This is the genius of our 
system, that the Federal Government has funded research 
throughout our Nation, and most of our best scientists are 
funded by Federal funds--their equipment, their labs. They all 
have Federal funds in them. So what we are faced with in 
California is potentially building separate facilities, which 
we can do, but it is an enormous waste of resources that could 
go instead to the fight against this disease.
    Second, as much as I love California and as much as I 
believe that the very best scientists in the world are here in 
California, I will concede that there are other excellent 
scientists in other States around the Nation.
    The Chairman. Would they be in Oregon? I am just curious. 
[Laughter.]
    Dr. Goldstein. There are some very excellent scientists in 
Oregon. Now, we may try to recruit them from Oregon if 
something is not done to enable them to work in this important 
field. The Federal Government has an enormous role to play in 
ensuring that our best scientists, regardless of where they are 
located, can participate in the fight against this disorder, 
because, again, we do not want just one scientist doing this. 
We want dozens, hundreds, taking advantage of these 
opportunities and making progress.
    The Chairman. Relative to other countries who may be 
pursuing this--specifically in Europe and Japan I am aware of--
what is the importance of the U.S. Government participating 
along with other nations? The same logic holds?
    Dr. Goldstein. Well, I think it is partly the same answer, 
and it is partly that in the drive to develop uniform 
international ethical standards, both for the treatment of 
human subjects and for the development of therapies, I think 
that the United States has a very important leadership role to 
play, which it has played historically.
    I will also note that there is an enormous economic 
interest here. The United States has a positive balance of 
trade in this area of its economy, and to be honest and blunt, 
if I look ahead 10 years from now, I would rather have my 
children selling therapies to the rest of the world as opposed 
to buying them from the rest of the world. I would be loath to 
see the United States cede its historical lead in this field.
    The Chairman. Doctor, I understand that over 100 new stem 
cell lines have been developed since August of 2001 and that 
some of these lines are disease specific. Can you tell me what 
it means to have a disease-specific stem cell line and what 
this means to a researcher like yourself?
    Dr. Goldstein. Well, for the case of Huntington's disease, 
that is perhaps the easiest to understand because I gather that 
one of those lines or a few of those lines have the major 
genetic change that causes Huntington's disease, a terrible 
disorder where the brain malfunctions and people have movement 
and cognitive disturbances.
    The ability to develop brain cells from those cells that 
have those genetic changes will let us understand what fails 
early in the disorder and could be an incredibly important tool 
in solving the problems of that disease.
    Similarly, for Alzheimer's disease one approach is to 
introduce known genetic changes. But, of course, the ability to 
have stem cell lines that are patient or person specific allows 
us to compare how that person's disease has developed in their 
adult state with how we can study the biology of those brain 
cells that have the identical genetic constitution in the 
laboratory and really learn what are the nature of the cellular 
changes that cause the dysfunction, that cause the failure that 
leads to the inability of these people to remember, to think, 
to speak, any other terrible symptom with the disorder.
    The Chairman. Are there specific experiments that 
scientists want to do but cannot do now because of the 
limitation on federally approved lines?
    Dr. Goldstein. I think that nobody knows the answer to 
that, but I think in some ways it is a question of rate. The 
current system in my view--and I will be blunt about this--is 
incredibly clumsy. It turns scientists such as myself into 
lawyers and accountants to navigate the very complicated 
licensing, patenting, and, of course, separation issues if one 
wants to work with more than just the approved cell lines. Of 
course, I do want to work with more than the approved cells 
lines. We have established methods for doing that in my 
laboratory. I am in a very unusual and fortunate situation 
because of the Howard Hughes Medical Institute that I can do 
that. But most of my colleagues cannot, and so they are, as has 
often been said in this debate, working with one hand tied 
behind their back. Of course, you can make progress with one 
hand. I would rather have two.
    The Chairman. Thank you, Doctor.
    Senator Kohl.
    Senator Kohl. Dr. Goldstein, in your opinion, what will 
happen to your research and that of others in our public 
institutions if embryonic stem cell research is forced to be 
conducted largely in the private sector or in other countries?
    Dr. Goldstein. Well, I think that those are two somewhat 
different issues. The private sector I think is unlikely to 
tackle many of these problems in the way that we will in the 
academic sector. The kind of approach that I described in 
trying to understand and develop new treatments for Alzheimer's 
disease is in many ways a longer-time-frame approach than the 
private sector with its quarterly reports and its inexorable 
bottom line can do. They will have the kinds of limitations 
that academic scientists such as myself will not have. So I 
think if you limit this to the private sector, it will 
eventually happen perhaps, but it will happen much slower. I 
think the issue really is one of time and delay.
    Suppose, for example, that it takes 10 years to get to the 
point where we have some new drugs from this approach. That is 
a long time. On the other hand, if we delay 5 years before 
initiating that approach or we have in place restrictions that 
add 5 years to that time line, that is millions of people who 
will suffer and die before we have an opportunity to treat 
them. You add delay on at the end. That is the inexorable and 
terrible problem of the political situation that exists in this 
country now with this vital area of research.
    Now, with respect to other countries, I have a great deal 
of respect for my colleagues in other countries. But I also 
have national pride. I believe in the enormous energy and 
creativity of the American scientific community, and I believe 
that things will happen much, much faster with the 
participation, the full participation of the scientists in this 
Nation.
    Senator Kohl. States like California and my own State of 
Wisconsin are moving forward with their own initiatives to 
encourage and provide funding for stem cell research in the 
absence of a strong Federal policy. With no coordinated Federal 
oversight or strategy, are we at some risk for creating 
duplicative research efforts in the different States?
    Dr. Goldstein. I think that is absolutely a danger, as well 
as a danger of not being able to freely exchange materials, of 
a patchwork of national regulation where what we can do in one 
place is different from what we can do in another place. I 
mean, it creates an enormously complicated playing field.
    I think you have to remember that science is not the sort 
of ivory tower scientist laboring in isolation. We are a very 
interactive profession, and that interaction allows progress to 
proceed more rapidly because we are very open with 
communication of our ideas and our materials in most cases. 
When Government policies restrict that interaction and our 
ability to exchange materials and ideas, things go very much 
more slowly and incredibly inefficiently. It is a waste of 
valuable human and financial resources to proceed in that 
manner.
    Senator Kohl. Thank you very much, Mr. Chairman.
    The Chairman. Senator Clinton.
    Senator Clinton. Thank you very much, and thank you, Dr. 
Goldstein.
    I co-chair the Senate's Alzheimer's Task Force, and I want 
to thank you for the work that you are doing in this area. In 
your opening testimony, you discuss sporadic Alzheimer's 
disease which may be caused by a combination of genetic and 
environmental factors. Can you please discuss the ways that 
stem cell research may help us understand how genetic changes 
are effected by our environment? Will this type of research 
result in information that can contribute to our public health 
efforts to try to prevent diseases?
    Dr. Goldstein. That is an excellent question, Senator 
Clinton, and I think you have really summarized the value of 
this approach. Using human genetic methods in large human 
populations, there are a great number of small genetic changes 
that have been identified in different genes where there is 
statistical evidence that those small changes may predispose 
someone to the development of this disease either on their own 
or perhaps in combination with environmental factors. But it is 
extremely difficult, if not impossible, to evaluate how those 
genetic changes affect the behavior of the human brain cells 
that fail in Alzheimer's disease. So the availability of brain 
cells that have those changes, which we believe we can get 
through the manipulation of these human embryonic stem cells, 
will let us study how those brain cells differ from people who 
did not develop the disease, whose genotypes or genetic 
constitution did not lead them to develop the disease. We can 
compare the behavior of those cells to cells that have the 
large-scale genetic changes and ask what are the physiological 
similarities and differences and, yes, the identity of those 
genes--for example, a gene involved in cholesterol metabolism 
or a gene involved in the response to foreign pathogens in the 
immune system. Studying how those things behave in culture 
gives us important clues about the environmental insults that 
may tip a cell over the edge into the disease stage because of 
its unique genetic constitution.
    Senator Clinton. Dr. Goldstein, I appreciate your 
describing for us the problems that American scientists are 
having because of the lack of any Federal policy that really 
not only provides permission but a framework in which work can 
continue on embryonic stem cells.
    There is a flip side to that which I find equally 
disturbing and it does not get much attention. Isn't it the 
case that right now, in the absence of any regulation other 
than the President's Executive Order confining research to a 
limited number of stem cell lines and prohibiting any Federal 
funding from being used for any further experimentation, there 
are no rules governing what the private sector does? Right now 
we have sort of an open door to private sector research. For 
all we know, if someone has enough money, they could be engaged 
in reproductive cloning as we speak. They could be engaged in 
all kinds of research that we might find ethically and morally 
abhorrent. But because we do not have a legal framework, we 
have no prohibitions against what goes on in the private 
sector.
    Am I correct in that conclusion?
    Dr. Goldstein. I think that you have made an excellent 
point, and with the exception of a few States, such as 
California, that have put in place strong legal frameworks for 
proceeding, strong legal prohibitions on the kinds of 
activities you describe, both in the public and private sector. 
Nationally, we lack that kind of uniform framework, and, 
indeed, there is a vacuum in many places where unethical or 
perhaps unreasonable things could proceed in the private 
sector.
    You know, with recombinant DNA, gene splicing technology 
provides us with a good example of how the opposite can happen, 
which is that when the Federal Government plays an active, 
involved, and informed role, the public sector develops 
guidelines, regulations, and practices that we practice in the 
public sector. The fact is that even in the absence of direct 
law and regulation, the private sector companies are staffed by 
people that come out of our laboratories. As scientists, we 
actually embrace regulation. It is not that we want to operate 
in a completely uninhibited way. We welcome reasonable 
regulation. We want to participate in the development of those 
regulations so that they are workable as well as ethically and 
financially reasonable. The private sector in my experience is 
happy to follow along and adopt those, either directly through 
the process of law and regulation, or indirectly, as happened 
in recombinant DNA, because our people who were trained in the 
public sector moved into the private sector.
    So I hope that we can find a way through this impasse so 
that we can develop standards, perhaps regulation, that allow 
the public and private sectors to operate with an agreed-upon 
set of guidelines.
    I will just add, of course, that even in the absence of 
Federal regulation, the National Academy of Sciences just 
released a wonderful set of recommended guidelines and 
regulation which I hope, even in the absence of Federal 
regulation--although I hope there will be some--our 
institutions are moving to adopt in many cases, and those 
guidelines I commend to the committee to have a look at because 
they are excellent.
    Senator Clinton. Thank you, Dr. Goldstein.
    The Chairman. We have been joined by Senator Lincoln of 
Arkansas. Senator Lincoln?
    Senator Lincoln. Thank you, Mr. Chairman, and thank you, 
Dr. Goldstein.
    Just briefly, I know that in some of the testimony from our 
other panelists here, there is some mention about the impact of 
embryonic stem cell research and its positive impact on the 
research using umbilical cord and bone marrow. At the 
University of Arkansas Medical Sciences now in Little Rock, we 
have become a leader in some of the blood stem cell 
transplants. I visited one of the research physicians and 
really was amazed at how much progress we have made in that 
area of adult stem cell transplants and the abilities that 
exist there.
    Do you have any comments to elaborate on how embryonic stem 
cell research will further the development of this other type 
of research?
    Dr. Goldstein. Yes. Although this is not my specific area 
of focus and expertise, there are two areas where I know there 
is enormous interest and promise for embryonic stem cell 
research to make an impact on cord blood and blood-forming stem 
cell transplants such as those you have described. Those kinds 
of transplants, as you know, are enormously valuable. If you 
have a disease that can be treated in this way, it is an 
enormously powerful way to do it.
    However, there are many people for whom we cannot find 
genetic matches to give them a cord blood or bone marrow 
transplant. For those people, we will need perhaps embryonic 
stem cell-derived cells for transplant in the future unless the 
genetic match issues can be solved in some way.
    The second thing is that one can learn a great deal, if you 
will, about the properties, the behavior of cellular materials, 
and the ability to use them in different ways, much as you 
might study the properties of metal if you were trying to learn 
how to fix automobile engines. That tells you a great deal 
about how to approach these problems. Human embryonic stem 
cells have a great deal to teach us about how cord blood stem 
cells and other stem cells can work in the therapeutic setting 
and perhaps how they can fail in the disease setting.
    So scientists such as myself would never say that one 
should do one or the other. I am an enormous fan of adult stem 
cell research for the areas where it can make an enormous 
impact. I would not say that we should not do that. However, it 
is, again, the one-hand-tied-behind-your-back problem. We will 
do better with two hands, and those two hands can work together 
in order to solve some of these terrible problems.
    Senator Lincoln. Thank you so much.
    Thanks, Mr. Chairman.
    The Chairman. Thanks, Senator Lincoln.
    Dr. Goldstein, you have been a tremendous help to us and 
have added measurably to the debate that is going on on Capitol 
Hill, and we thank you so much for your time and your expertise 
and all the work that you are doing. All the best.
    Dr. Goldstein. Thank you, Mr. Chairman.
    The Chairman. We will turn now to our next witness, Doug 
Doerfler of the Biotechnology Industry Organization and 
president and CEO of MaxCyte in Gaithersburg, MD. The mike is 
yours.

STATEMENT OF DOUGLAS A. DOERFLER, PRESIDENT AND CHIEF EXECUTIVE 
     OFFICER, MAXCYTE, GAITHERSBURG, MD; ON BEHALF OF THE 
              BIOTECHNOLOGY INDUSTRY ORGANIZATION

    Mr. Doerfler. Thank you. Good afternoon, Mr. Chairman and 
members of the committee. My name is Doug Doerfler. I am 
president and CEO and founder of MaxCyte, a biotechnology 
company located in Gaithersburg, MD.
    The Chairman. You may want to pull the mike a little closer 
to you.
    Mr. Doerfler. I am also here representing the Biotechnology 
Industry Organization, better known as BIO.
    Thank you for the opportunity to present testimony today on 
the promise of embryonic stem cell research and in support of 
Senate bill 471, the Stem Cell Research Enhancement Act of 
2005.
    My company uses cell-loading and gene delivery technologies 
to develop cell-based therapies. Because many diseases and 
disabilities are caused by cellular malfunction, medical 
breakthroughs in the treatment of serious diseases and 
disabilities can be developed through these cell-based 
technologies. At MaxCyte, we are working to develop therapeutic 
products for treating pulmonary disease, oncology, infectious 
disease, autoimmune disease, diabetes, and other neuroscience 
diseases.
    I want to make two points at the outset of my testimony. 
First, my company does not perform embryonic stem cell 
research. Second, BIO supports all types of stem cell research, 
including research using cord blood and adult stem cells.
    Mr. Chairman, I am here today to say that to help speed the 
development of all types of cell-based therapies, we need 
expanded Federal support of embryonic stem cell research. That 
is why BIO supports S. 471.
    Scientists have found that existing cell lines are not 
genetically diverse, are difficult to grow, and may be 
contaminated with animal proteins. Your bill appropriately 
makes more cell lines eligible for Federal funding while 
creating a framework to ensure that research is performed 
ethically.
    In particular, BIO strongly supports development of NIH 
guidelines. We believe this is an important step and is similar 
to the way the Asilomar Conference helped ease public anxiety 
and spur the development of recombinant DNA technology during 
the 1970's.
    This committee has heard and will continue to hear about 
the potential benefits of embryonic stem cell research 
regarding cures and treatments for diseases and disabilities. 
Embryonic stem cells have the potential to be turned into any 
of the body's cell types, meaning they could possibly be 
developed into replacement cells and tissue for patients whose 
own cells are malfunctioning. This has not yet been shown to be 
true for adult stem cells.
    It is that potential that has thus far generated the most 
enthusiasm amongst the scientific community because it shows 
promise toward developing breakthrough treatments for a variety 
of intractable diseases, including various cancers, diabetes, 
Parkinson's, Alzheimer's. We need to know these answers.
    However, I would like to discuss other reasons to support 
this research.
    It is important to emphasize that embryonic stem cell 
research will have a positive impact on all types of 
therapeutic research and will further the development of cell-
based therapies.
    Embryonic stem cell research will lead to greater 
scientific understanding of cell differentiation. This cell 
differentation is the process by which cells change from a stem 
cell to perhaps a nerve cell, a brain cell, or a blood cell to 
perform certain critical functions in our body.
    In addition, if this bill is enacted, more genetically 
diverse cell lines will be available for funding. Scientists 
will then be able to learn more about how and when genetic 
anomalies cause cells to malfunction. This will help 
researchers understand the root causes of many diseases and, 
therefore, lead to the development of truly breakthrough 
therapies.
    Expanded support of embryonic stem cell research could also 
go a long way toward reducing the time and expense needed for 
drug development. New chemical or biological compounds meant to 
treat diseases could be tested in specific human cells prior to 
their use in live human beings, accelerating development, 
reducing costs, and reducing adverse events in patients.
    Mr. Chairman, I have heard opponents of your bill say that 
it is not necessary to expand Federal support for stem cell 
research because many States are moving forward with their own 
programs.
    Nothing could be further from the truth. There is no 
substitute for increased commitment from the NIH. In addition 
to funds, the NIH provides the infrastructure and a uniform set 
of rules for the scientific community--especially as basic 
research is turned into therapies. Forcing companies to deal 
with a patchwork of State regulations and requirements will 
create huge inefficiencies and confusion that will hamper 
capital formation and inhibit critical collaborations and slow 
development of treatments for patients.
    In conclusion, embryonic stem cell research holds the 
promise to dramatically improve scientists' ability to develop 
cures and treatments for disease. Whether these therapies are 
the direct result of this research or come about due to the 
advances in scientific knowledge that will come from this work, 
our Nation must increase its commitment.
    BIO supports your legislation, Mr. Chairman, because it 
will expand Federal support for this important research.
    Thank you for the opportunity to present this testimony 
today, and thank you for your courage in handling this very 
sensitive issue.
    [The prepared statement of Mr. Doerfler follows:]
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    The Chairman. Well, thank you so much for your presence 
here today and your contribution to our committee.
    Mr. Doerfler, this is not the first time new medical 
technology was considered controversial. There are a number of 
medical technologies considered routine today that were 
initially criticized and opposed. I am thinking specifically of 
recombinant DNA. Do you recall that that was criticized in 
earlier days as well?
    Mr. Doerfler. That is right. That recombinant DNA was the 
focus of a number of concerns, certainly in Boston where people 
were concerned about DNA being placed in drains of apartment 
buildings. The response was the scientific community stepped 
forward with the Asilomar Conference back in the early 1970's, 
and the best and the brightest lawyers, business people, 
scientists, and ethicists created a set of guidelines that were 
then used with NIH to create the framework by which recombinant 
DNA technology was able to not only start but flourish and has 
resulted in some incredible breakthroughs in the treatment of 
serious diseases.
    The Chairman. As I understand your testimony, you do not do 
embryonic stem cell research.
    Mr. Doerfler. We do not.
    The Chairman. But you do adult stem cell research. But you 
are here testifying on behalf of stem cell research to be 
expanded in other areas. Is that because you recognize that 
adult stem cells may work for some diseases and embryonic may 
work for other kinds of diseases?
    Mr. Doerfler. I am not sure we understand how broadly adult 
stem cells can be used, and one way of learning about that is 
being able to study embryonic stem cells, understanding how 
these cells differentiate into pancreatic cells or nerve cells, 
so we can directly take the learning from embryonic stem cells 
differentiation and apply them to cells like umbilical cord 
stem cells or bone marrow-derived stem cells. It is an 
important area that we need to know. We just do not have the 
background yet to understand how cells differentiate and what 
we need to do to proliferate these cells, expand these cells 
without them differentiating, which is a huge problem in the 
stem cell area.
    The Chairman. Thank you.
    Senator Kohl.
    Senator Kohl. No questions.
    The Chairman. Thank you very much, Doug. We appreciate your 
time and your contribution again.
    Our next witness is John Gearhart, Ph.D., Johns Hopkins 
University, Department of Medicine, Institute for Cell 
Engineering. Thank you for being here, sir.

STATEMENT OF JOHN D. GEARHART, PH.D., JOHNS HOPKINS UNIVERSITY, 
    DEPARTMENT OF MEDICINE, INSTITUTE FOR CELL ENGINEERING, 
                         BALTIMORE, MD

    Mr. Gearhart. Thank you, Mr. Chairman, for the invitation. 
I think one of the advantages of testifying toward the end is 
that many of your points were already taken, but I think there 
is one area that I can be of service to the committee. My 
laboratory has been working on stem cells for the last 13 
years. We were one of the two labs in the country to report the 
isolation of very unique stem cells from the earliest stages of 
human embryos.
    Since that time, in 1998, we have developed a very large 
research program at Johns Hopkins centered in the Institute for 
Cell Engineering, and I think what I would like to present to 
you is what is going on in an institute such as ours on a daily 
basis with respect to human embryonic stem cell research, adult 
stem cell research, and umbilical cord blood research.
    Our goal is not to promote one form of stem cell research 
over another. Our goal is to try to find cell-based therapies. 
We want to see what works, what does not work.
    One of the difficulties in any stem cell research program 
at this point in time--and we will talk specifically about 
embryonic stem cell research--is that we have now cells in a 
laboratory dish that are capable of forming any of the over 200 
different cell types that are present in your body. We do not 
want 200 different cell types present in that dish. We want 
cardiomyocytes, the heart muscle; we want dopaminergic neurons, 
those cells that are needed for Parkinson's disease; we want 
motor neurons.
    How do we get them? Now, here is this cell that has these 
capabilities. How do we direct it to form what we want and in 
the numbers we need to do any kind of graft for a therapy?
    So with these 200 different cell types, I think I have a 
graduate student working on each one, or a post-doc, and there 
are problems. We can to some degree generate all these 
different cell types, but it is a matter of efficiency. We rely 
upon basic science information that has been obtained over the 
years, principally through the NIH funding, on how in our 
bodies from the very earliest stages of embryogenesis a motor 
neuron becomes a motor neuron or a heart cell becomes a heart 
cell.
    We try to take that information and utilize it in a dish to 
say, well, we know at this point in time it is seeing this type 
of a growth factor or any number of combinations of things, and 
we try to recapitulate the steps to get us to the endpoint. In 
some of these cases, we have succeeded in generating various 
kinds of cells with high efficiency. In our lab, we have been 
interested in cardiac tissue, in motor neurons. We are also 
interested in dopaminergic neurons for Parkinson's disease. We 
are also interested in blood cells.
    But let me tell you what takes place now. We are able, for 
example, to grow large numbers of human heart muscle cells. We 
can do this now through embryonic stem cell technology. We are 
now in the process of grafting these cells into various animal 
models, whether it is congestive heart failure, heart attack, 
to see if these cells will function following transplantation. 
The same is true of dopaminergic neurons for Parkinson's 
disease or motor neurons, which you will about hear from Dr. 
Zhang.
    This now is a whole other level of issues that we must 
confront and solve, and that is, how do we introduce these 
cells? Do they stay where you put them? Do they form tumors? Do 
they maintain their function over periods of years, which is 
what really we are targeting in humans? Do we have animal 
models that will model human disease, and how effective are 
they?
    So we have our cells in rodents, we have our cells in fish, 
we have our cells in monkeys--all with the idea of how well 
will these cells function to cure or to ameliorate any of these 
disease processes. You say, well, this is great. It is great. 
This is a major step in the direction of moving toward human 
trials. But there is much work to do to get it to the human 
trials.
    Where have the limitations come from? Well, quite frankly, 
it is from Federal funding into this area. We are very limited 
in what we can do with Federal funding in our laboratories. Dr. 
Goldstein made an important point that I hope you did not miss. 
If we are using the approved lines for NIH funding in our 
laboratories, they must be handled in a very, very different 
way logistically than lines that we use from Harvard or from 
Singapore in that it must be clear that everything that touches 
those cells, including the technicians that are using it, has a 
straight line and only a straight line to the Federal funding 
source, that there is no crossover into other areas of where 
that Federal money is going. That logistically is a nightmare. 
Most of us have to build separate laboratories to make sure 
that those walls are there. This is a major, major limitation.
    The lines themselves that we use with the Federal sources, 
they certainly are proving to be valuable in many areas of 
research. I am not discounting that. But there are limitations. 
We find, for example, in some of these lines that we cannot 
isolate a specific cell type out of those existing lines, and 
we have to go to lines that were generated at Harvard or 
outside of this to find the cell type of interest that we are 
looking for. This is a major limitation.
    Again, this whole issue of how much flexibility an 
investigator has among laboratories in the same institution, 
some working on the human approved lines with NIH--sorry, not 
human approved, but the approved lines for Federal funding, and 
down the hall or next door to us we have a lab working on 
another type of cell line. There is just a logistical nightmare 
with this as it now exists.
    Still, I would commend you and commend the Members of the 
House for trying to expand the current policy. I think the 
generation of new lines is extremely important for our work. We 
will, I think, be more assured that these lines will have 
utility, that they will be safe, and provide us a broader base 
as far as the genetics are concerned within those cells.
    So, again, I thank you for trying to expand that. I wish I 
could nudge you to even expand it in other directions, if we 
could, and perhaps we could talk about that at another time. 
But at this point in time, it is essential that this bill be 
passed, and it is essential, I think, as far as national policy 
is concerned, that we have a better policy covering more 
aspects of the work.
    I will stop there. I would love to get into our issues with 
our investigators in other countries and how we are dealing 
with that competitively, et cetera. But I again thank you for 
your support on behalf of S. 471.
    [The prepared statement of Mr. Gearhart follows:]

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    The Chairman. Dr. Gearhart, I came from a luncheon of my 
Republican colleagues where there was--and in the interest of 
confidentiality, I am not going to mention anybody's names, but 
there were a lot of pro-life Senators in that room with 
different opinions on this issue. One of my colleagues, who 
will remain anonymous, made the point that there is a new type 
of embryonic stem cell research that does not destroy the 
embryo. I do not know what the name of it is or I would tell 
you that, but you probably know what I am talking about because 
it has been in the newspaper. The point he was making is that 
this argument will be moot in the very near future if that type 
of research is the one that goes forward.
    Can you speak to that?
    Mr. Gearhart. Oh, I would love to.
    The Chairman. Is that person wrong on that?
    Mr. Gearhart. Well, look, one of the most disheartening 
aspects of being in these stem cell battles--I have been there 
for 7 or 8 years--is the distortion of facts around any aspect 
of either other avenues that can work, the claims around the 
adult stem cells, for example, of doing everything that an 
embryonic stem cell can do. This is just very disheartening, 
and I would like to quote one of your former great Senators 
from the standpoint that, he was fond of saying you are welcome 
to your own opinion but not your own facts. This is the case:
    We saw recently published in the Washington Post an article 
by Rick Weiss in which he summarized several new alternatives 
to the use of embryos or the destruction of embryos in 
embryonic stem cell research. None of this is published. Some 
of it is in the very earliest stages of research. If you read 
this carefully--and I would tell you I have read some of the 
manuscripts that have come out of this. I probably should not 
say that. This is not--the frequency of success in this is 
extremely low. There are many holes in the experimental designs 
and the outcomes of what people have even presented at meetings 
up to this point.
    If we were to wait around for this to work, I mean, to say, 
yes, I would love just to take a single cell, one cell off an 
embryo and say that we can generate an embryonic stem cell line 
out of that, leaving the remainder of the embryo to be used for 
reproductive purposes, the frequency and success of this is so 
low you would have to sample from hundreds of embryos. No one 
is going to permit this in any kind of a protocol before an 
IRB, internal review board. It is not going to be that way.
    So I would just say to you I hope it would work.
    The Chairman. But it is not the silver bullet that it was 
presented as being?
    Mr. Gearhart. It is not the silver bullet, and if we were 
to wait around any longer before we really established robust 
ES work in this country, we are really going to be behind.
    The Chairman. That was my supposition to what was being 
said and argued very strenuously, but there was, you will be 
pleased to know, lots of Senators arguing in a different 
direction, in the direction you are advocating.
    You are from one of the great medical schools in our 
Nation, and I don't know that Maryland has passed any bond 
initiative like California's. What does the range of the CA-
initiative mean to Johns Hopkins?
    Mr. Gearhart. Well, we came, interestingly, within one vote 
of a filibuster in the Senate of Maryland of perhaps passing 
legislation. It was not a bond issue. It was $23 million for 
embryonic stem cell research.
    I think you could argue the point in two ways. I wish that 
States did not have to get into this. I mean, money is scarce. 
States can do many things with it. But because of the national 
political scene, we have to get in it. I think we have to get 
in it not only to support our researchers, but I think where 
those States are going to be successful is they are going to 
serve as catalysts for bringing in biotechnology that is going 
to be there for decades.
    This is what is happening in California. People are looking 
there. I mean biotech companies. The other thing which I am 
personally upset about is that they are coming to members in 
our research group and saying, How about a job in an 
environment with money? You do not have to worry about these 
things. For particularly young investigators, this is a major 
draw. I think we are going to see this not only in California, 
I believe New York is going to be in play, New Jersey is in 
play, other States are going to be in play. I think we are 
going to get partitioning out of not just stem cell research 
but, as I say, there is going to be a movement to where there 
is a progressive outlook on biomedical research, and it would 
be characterized by those States. So it does have an impact, I 
think, both in getting our work done and having the personnel 
that we would like.
    One other point. For us to get this technology to the 
clinic, it is not just going to reside in the academic setting. 
We are going to have to be partnering with biotech companies, 
pharmaceutical companies, because it is going to be expensive 
to get it into the clinic. To go through trials and things like 
this, we are going to need the partnerships with biotechnology 
and pharmaceuticals, and if they are going to other places 
where this is more appropriate, we are not going to have it.
    The Chairman. Frankly, the money and where people live that 
are doing the research is a very, very secondary consideration, 
but I think you have just told me that Johns Hopkins, Harvard, 
Oregon Health Sciences University, you know, they are going to 
be out of the business in a sense. They are certainly going to 
be in the back benches of this effort if the Federal Government 
does not participate financially and by creating ethical 
boundaries.
    Mr. Gearhart. One of the arguments that is made is why 
isn't it just handled by the private side. I do not see where 
all the money would come from. I mean, we are now--yes, we are 
the recipients, as other institutions are, of millions of 
dollars for some of this research. But this cannot go on 
forever. Clearly, I think at the state-of-the-art of the work, 
at the very early stages, we need the Federal support which 
historically has come.
    The Chairman. Senator Kohl.
    Senator Kohl. Thank you, Mr. Chairman.
    Your comments, your questions, and your responses have been 
quite informative and have really added to the depth of the 
dialog, and we appreciate very much your being here.
    Mr. Gearhart. Thank you, Senator.
    The Chairman. Thank you very much, Doctor. We appreciate 
very much your presence.
    I think it would be appropriate for me to allow your 
Senator to introduce you.
    Senator Kohl. Thank you, Mr. Chairman. Our next witness, 
Dr. Su-Chun Zhang, is a researcher at the University of 
Wisconsin-Madison at the renowned Waisman Center. He is known 
worldwide in the science community for recent scientific 
breakthroughs that successfully coaxed stem cells into becoming 
human motor neurons, the nerves responsible for movement 
throughout the entire body. Dr. Zhang's research illustrates 
the enormous potential of embryonic stem cell research in 
treating and curing diseases affecting the lives of Americans 
suffering from Parkinson's, ALS, and other disorders. So we 
welcome you here, and we are looking forward to your testimony.

STATEMENT OF SU-CHUN ZHANG, M.D., PH.D., ASSISTANT PROFESSOR OF 
  ANATOMY AND NEUROLOGY, STEM CELL RESEARCH PROGRAM, WAISMAN 
  MENTAL RETARDATION CENTER, UNIVERSITY OF WISCONSIN-MADISON, 
                          MADISON, WI

    Dr. Zhang. Thank you, Mr. Chairman, for inviting me to 
testify before you about the recent progress in the area of 
human embryonic stem cells. Since I am the last one, most of 
the points have been made. But I just would like to tell you 
how I got into embryonic stem cell research, how we feel about 
the current state of embryonic stem cell research in this 
country.
    I used to work on brain stem cells, one kind of the so-
called adult stem cells. The hope was to use these brain stem 
cells to produce specialized brain cells to repair neurological 
disorders. But it turned out that even though these brain stem 
cells can produce nerve cells, they actually have very limited 
capacity to produce very specialized nerve cells like dopamine 
neurons, motor neurons, or oligodendrocytes that are lost in 
multiple sclerosis patients and other patients.
    That was one of the major reasons why I contacted Dr. 
Thomson, Jamie Thomson, who was the first person, along with 
Dr. Gearhart, to establish the first human embryonic stem cell 
lines in the world. We started, but because of the sensitivity 
of embryonic stem cell research, actually I waited for a year 
and a half in order to set up a separate small and very 
rudimentary laboratory outside of the institute in order to 
conduct this kind of research.
    Like many laboratories in this country, my own laboratory 
was not able to use these cells until 2002, after the 
President's decision on the Federal funding on human ES cells. 
But if you look back just the past three years, the progress 
that has been made in the area of embryonic stem cell research 
is quite enormous.
    It has already been shown that many cell types can be 
produced from human embryonic stem cells, including heart 
muscle cells, brain cells, or blood cells. In particular, at 
least in the literature, peer-reviewed literature, it has been 
shown that the dopamine neurons can be very efficiently 
produced from these embryonic stem cells, including from my own 
lab, and motor neurons which control our movement can also be 
produced from these cells. There is a recent report showing 
that another type of cells which are making myelin sheath, and 
are lost in multiple sclerosis patients, can also be produced 
very efficiently.
    So by just looking at this very short period of time in 
terms of the progress made in this area, it is quite 
unprecedented in the history of science, and it speaks itself 
of the great potential of embryonic stem cells.
    Furthermore, actually recently it already began to show 
that these cells may work in repairing some of the damages in 
animal studies. I think what has been shown lately in animal 
studies, if you transplant some of these embryonic stem cell-
derived nerve cells, they can help animals that have motor 
neuron disease or Parkinson's disease or even spinal cord 
injury. Of course, a lot more work needs to be done in order 
for this technology to be actually used in patients.
    Now, if you look back at the past two or three years or 
beyond, there are also problems. The problem is that we have 
only a limited number of stem cell lines to work, and plus 
these cell lines were originally derived from growing on animal 
cells. Now more and more investigators want to use these cell 
lines. So the number of lines are simply not sufficient for the 
community to work, at least using Federal dollars.
    Further, if you want to use some special stem cell lines, 
for example, some cell lines that have genetic defects which 
will allow us to understand what is going wrong and how to 
correct them, these are still not allowed to be used using 
Federal dollars. For example, my institute works on mental 
retardation and genetic disorders, and we are very keen to use 
these cell lines. But if I take these cell lines and I already 
have Federal money, it just is so difficult to separate these 
Federal dollars away from the private funding. So the reality 
is the current situation, current rule that you can work on 
these stem cell line using Federal dollars, actually not only 
slows down the research using stem cells, but also interferes, 
actually affects the effective use of private funding. If I am 
going to use private funds to work on the cell lines currently 
not in the (NIH) registry, I have to again go back to what I 
did several years ago to set up a separate lab outside of the 
area I am working on. So these problems really hamper the area 
of research.
    That is why we really want to urge the Senators to consider 
changing the current rule. I think you already mentioned that 
you will.
    Finally, before I came here, some of my graduate students 
grabbed me: ``You have to deliver another point.'' That is, we 
Americans actually led the world by first establishing this 
human embryonic stem cell work, including the pioneers, Dr. 
Thomson and Dr. Gearhart here. Yet we should not be left out. I 
told them yesterday, I said the Senators are much smarter than 
us. They want Americans to lead the way, and will not let us 
down in leading the world in this area of promising research, 
which could potentially be saving life and improving health for 
all Americans.
    [The prepared statement of Dr. Zhang follows:]
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    [GRAPHIC] [TIFF OMITTED] 23759.017
    
    The Chairman. Dr. Zhang, to that point, are there other 
nations that are ahead of us now in this area?
    Dr. Zhang. In some areas, for example, the cloning of 
human-specific cell lines, because these techniques have been 
already there, here in Wisconsin or in Johns Hopkins.
    The Chairman. You are referring to the therapeutic cloning.
    Dr. Zhang. Yes.
    The Chairman. But how about in the embryonic stem cell?
    Dr. Zhang. Well, sir, as I mentioned, currently we only 
have limited numbers of cell lines available.
    The Chairman. Can you name a country that is proceeding on 
embryonic stem cell research? Is Great Britain?
    Dr. Zhang. Great Britain, Japan, South Korea, Singapore, 
Australia, also including China, Israel, many, many other 
countries.
    The Chairman. Dr. Gearhart, do you have a sense of where 
they are relative to where we are?
    Mr. Gearhart. Yes there are certain ways in science that 
you measure where progress is being made. One of the best 
measures, Senator, is looking at where publications are coming 
from, just the number of publications and the quality, you 
could argue, also, of that. Initially, as was pointed out, this 
work started in the U.S., and we saw U.S. investigators 
publishing most of the papers. The last few years we have seen 
a tremendous upswing in papers coming from these other 
countries, just in sheer volume and in quality. Good work is 
being done by these investigators. So that is one measure that 
we take as to, you know, where this work is being done.
    The second is looking at the investment, either through the 
government or through the private side, and we are seeing 
hundreds of millions of dollars dumped directly into embryonic 
stem cell research. This does not deal with the cloning issues 
at all in whether it is Singapore, whether it is China, Israel. 
We can go down the list, and this is another measures of just 
looking at where the investment is.
    We also now look at where the investigators are going to do 
this work. Now, before California was in play, you know, 
students, post-docs, were leaving the country. Now they may go 
to California, which would be the same difference, perhaps. But 
the issue is it is in play now. But I am telling you that when 
our students were finishing and looking for jobs, looking for 
what we call post-doctoral fellowships, they are looking at 
other countries.
    The Chairman. Dr. Zhang, you mentioned that the President's 
decision to allow embryonic stem cell research to proceed on 
these 78 approved lines has developed some real progress on the 
issue.
    Dr. Zhang. Yes.
    The Chairman. But as I have read, the way in which they 
were allowed to be generated through mouse cells ultimately 
makes it impossible for them to take this page and just xerox 
it, you know, ad infinitum, and that they are just played out.
    Dr. Zhang. Yes, it will make it very difficult for you to 
translate what progress you made using the current lines to 
application to the clinical side.
    The Chairman. So we are really at a dead end at this point 
in making progress.
    Dr. Zhang. Exactly.
    The Chairman. Do all of you agree with that in terms of the 
lines that are available, they are played out at this point, in 
terms whether we can do more?
    Dr. Zhang. We can still use the existing lines to do basic 
research, but in order for the translation from basic to 
clinical side, we need cleaner lines that are derived using 
different technology. The reality is this technology is being 
developed. As mentioned earlier, Dr. Thomson's lab in Madison 
already established a method that you can grow these human 
embryonic stem cells without the animal cells as supporting 
feeder cells. In other words, you can potentially generate new 
stem cell lines that will be free of animal contaminants.
    The Chairman. So what we have done to date has been of 
value, but the potential of these lines has been maximized. Is 
that what you would say, Dr. Gearhart?
    Mr. Gearhart. I agree with that. From a pure utility 
standpoint in the laboratory, to work on lines that have to be 
grown on other types of cells makes it just very difficult to 
identify specific components that are critical for 
differentiating these into some specific types. So we would 
rather work on lines that had no feeder layers--these are now 
available--or have never seen an animal product. When you say 
animal product, you are generally talking about a mixture of 
stuff, we do not know what is in it, but they sure grow well. 
We would like to define what is in it, and that is now what is 
being done with some of these other lines that are available in 
what Jamie has recently done. This is an extremely valuable 
reagent that should be eligible for Federal funding if we want 
to progress.
    The Chairman. Just for my own research, I understand that 
adult stem cells may actually really work well for some sorts 
of afflictions, but that it is more difficult to coax adult 
stem cells into specific cells. Is that accurate?
    Dr. Zhang. Yes.
    The Chairman. Can you access specialized cells for advanced 
diagnosis and treatment of various diseases? Can you tell me 
what are adult stem cells most promising to help, and what are 
embryonic stem cells most likely to cure?
    Dr. Zhang. For some adult stem cells, like blood stem 
cells, they have been used in the clinic for years to treat 
anemia and leukemia and other disorders. But in terms of the 
nervous system, some adult stem cells can be used as vehicles 
to deliver some therapeutic agents into the brain because they 
can still generate nerve cells or some glial cells, the 
supporting cells of the brain. Therefore, I think they can 
still be used.
    Also, maybe some time in the future when we figure out how 
to get embryonic stem cells to specialized nerve cells, we may 
actually learn these tricks and apply these tricks to adult 
stem cells and then teach these adult stem cells to become 
specialized. They may still have the potential. That is why in 
the scientific community we support both types of stem cells 
instead of just one or the other.
    The Chairman. Cord blood as well.
    Dr. Zhang. Sure.
    The Chairman. Senator Kohl.
    Senator Kohl. Thank you. To put that in another way and 
elicit an opinion from you, you are saying, as I understand it, 
that embryonic stem cell research is essential, that adult stem 
cell research all by itself is really not a viable option.
    Dr. Zhang. I didn't say that. [Laughter.]
    If I was understood in this way, then--no, I am saying that 
different types of stem cells have different kinds of uses.
    Senator Kohl. Right.
    Dr. Zhang. I think Senator Smith already asked this 
question. I think the answer would be the same. Adult stem 
cells have their use in specific areas, and embryonic stem 
cells also potentially have their usage, although we are just 
beginning to understand how they might work. They actually will 
fuse to each other and to promote the understanding how stem 
cells, whether they are in embryo or in adult, work.
    Senator Kohl. Yes. Is it fair to say it in another way that 
to maximize what you are doing, you need to have opportunity to 
do both?
    Dr. Zhang. Sure.
    Mr. Gearhart. Correct.
    Senator Kohl. That is categorical in your opinion.
    Dr. Zhang. Absolutely.
    Senator Kohl. It is not subject to opinion anymore.
    Mr. Gearhart. No.
    Dr. Zhang. No.
    Senator Kohl. I thank you.
    I thank you, Mr. Chairman.
    The Chairman. Do you have any closing comments?
    Senator Kohl. I am done.
    The Chairman. OK. Gentlemen, this has been very, very 
helpful, and your contribution will be reflected ultimately 
when this debate goes to the Senate floor. So we thank you very 
much for your time. We respect your work, and we hope to help 
you advance it.
    Chris Dudley, again, our many thanks for coming this long 
way.
    With that, the committee is adjourned.
    [Whereupon, at 4:13 p.m., the committee was adjourned.]
                            A P P E N D I X

                              ----------                              


          Statement submitted by The Johns Hopkins University

    One of the greatest discoveries in Medicine is the 
potential to use a single undifferentiated cell to help address 
the severe pain and suffering that numerous diseases, such as 
heart disease, diabetes, and cancer inflict every day. However, 
The Johns Hopkins University recognizes that stem cell research 
raises significant ethical concerns and that public policy on 
stem cell research must carefully balance the ethical and 
medical considerations, yet enable researchers to fulfill the 
promise of stem cell research for providing medical therapies.
    Johns Hopkins strongly supports the use of stem cells for 
legitimate research and therapeutic purposes. Stem cell 
research promises to have an enormous impact on human health 
and quality of life, and also on fundamental biomedical 
understanding. Stem cells can be obtained from embryonic, 
fetal, and adult tissues. It is essential that all these 
sources be investigated to determine which is most likely to 
fulfill the goals of basic research and lead to the development 
of new medical therapies.
    John Hopkins supports the use of the somatic cell nuclear 
transfer technique (popularly known as ``therapeutic cloning'' 
or ``research cloning'') for the purpose of producing stem cell 
lines that are geneticaly idjentical to the person from whom 
the nucleus was obtained. These stem cell lines are critical to 
help researchers better understand the pathogenesis of disease 
and provide information useful in developing therapies for 
people with a wide variety of diseases and injuries. In 
addition, stem cell lines produced using somatic cell nuclear 
transfer could overcome the rejection of tissues following 
transplantation.
    However, Johns Hopkins strongly opposes the use of stem 
cell technology and somatic cell nuclear transfer for the 
purposes of creating a cloned human being (popularly known as 
``reproductive cloning'').
    Stem cell research at John Hopkins is conducted under 
strict scientific and ethical guidelines that meet all 
federally mandated requirements. Johns Hopkins has long been a 
leader in the development of new therapies for patients, and 
stem cells represents a unique and promising approach in the 
development of new, critically needed treatments. Research at 
Johns Hopkins on stem cells is supported by the National 
Institutes of Health, patient-based organizations, partnerships 
with corporations, and private philanthropy.

                                 
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