<DOC>
[109 Senate Hearings]
[From the U.S. Government Printing Office via GPO Access]
[DOCID: f:23759.wais]
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
U.S. GOVERNMENT PRINTING OFFICE
23-759 WASHINGTON : 2005
_________________________________________________________________
For sale by the Superintendent of Documents, U.S. Government
Printing Office Internet: bookstore.gpo.gov Phone: toll free
(866) 512-1800; DC area (202) 512-1800 Fax: (202) 512-2250 Mail:
Stop SSOP, Washington, DC 20402-0001
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:]
[GRAPHIC] [TIFF OMITTED] 23759.003
[GRAPHIC] [TIFF OMITTED] 23759.004
[GRAPHIC] [TIFF OMITTED] 23759.005
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:]
[GRAPHIC] [TIFF OMITTED] 23759.006
[GRAPHIC] [TIFF OMITTED] 23759.007
[GRAPHIC] [TIFF OMITTED] 23759.008
[GRAPHIC] [TIFF OMITTED] 23759.009
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:]
[GRAPHIC] [TIFF OMITTED] 23759.010
[GRAPHIC] [TIFF OMITTED] 23759.011
[GRAPHIC] [TIFF OMITTED] 23759.012
[GRAPHIC] [TIFF OMITTED] 23759.013
[GRAPHIC] [TIFF OMITTED] 23759.014
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:]
[GRAPHIC] [TIFF OMITTED] 23759.015
[GRAPHIC] [TIFF OMITTED] 23759.016
[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.
<all>