Friday, July 25, 2003

Session 7: Public Comments

CHAIRMAN KASS: Look.  We have come to - only five minutes late - we've come to the end of the time allotted for this session and the time for the public comment, and we have four people who have signed up for public comment, and if Council is willing, why don't we simply stay at the table rather than take a break, and then we can leave promptly by 12?

For people who are with us for the first time, the house rules are public comments, by all means welcome, but there is a five-minute time limit on the comment to be made, and the first name on my list is Allison Hoffman from the National Partnership for Women and Families.

Ms. Hoffman, welcome and please come to the microphone.  It should be on.

MS. HOFFMAN:  The National Partnership for Women and Families is pleased to submit public comments to the President's Council on Bioethics for this meeting of July 24th and July 25th, 2003.

The National Partnership is a nonprofit, nonpartisan organization that is working to promote better health care for women and their families.  The National Partnership for Women and Families strongly supports allowing research using somatic cell nuclear transfer, commonly known as therapeutic cloning.

The purpose of this technology is to develop treatments for diseases and give millions of people access to life saving therapies using their own DNA.  Medical research involving therapeutic cloning holds tremendous promise for patients with countless diseases.  This research could produce treatments for diseases particularly affecting women, such as breast and ovarian cancer, arthritis, osteoporosis, and a range of genetically based diseases.

In addition to their own health concerns, women are the primary health care decision makers and care givers for their families as well.  Women continue to be the primary care givers for elderly parents, children, and family members with chronic or degenerative diseases.

SCNT offers hope to women struggling to care for parents with Alzheimer's disease or suffering after a stroke, children with juvenile diabetes and husbands with heart disease.  Any restriction on biomedical research, such as scent, jeopardizes this hope by threatening to delay or prevent important medical advances and sets a dangerous precedent for regulating medical research.

Instead the National Partnership supports measures that will maximize the benefits of this research and minimize the risk to women and their families, as well as society as a whole.

In establishing federal or state guidelines, we support policy proposals that ensure that all research, including therapeutic cloning, perceived with strong safeguards in place to protect individuals from unscrupulous practices.  We believe that therapeutic cloning research should be done only under the highest ethical standards with strong informed consent requirements, measures to protect women from exploitation, and a prohibition of undue financial inducements to donate eggs.

The National Partnership, along with countless other women's health advocates have worked for years to make up for researchers' past neglect of women's health.  In our pursuit of better information, treatment and cures for women and their families, we must insure that the newest and most promising techniques are available to those same researchers.

Thank you for your consideration of our concerns on this critical issue for women.

CHAIRMAN KASS:  Thank you very much.

Next, Sean Tipton from the American Society for Reproductive Medicine.

Welcome.

MR. TIPTON:  Thanks.

I would point out to everyone that tomorrow morning and Sunday morning you have an interesting opportunity to observe a convergence of the two topics you dealt with this morning, and that is in the person of Lance Armstrong as he pursue a fifth victory in the Tour de France.

You may recall Mr. Armstrong had testicular cancer, advanced testicular cancer, and some of the controversies upon his return were some question of enhancement versus therapy, and if he wins on Sunday, I'm sure you will see him with his children on the platform.  Those children were conceived with the assistance of ICSI.

So at least that's the spin I'm going to give my wife tomorrow and Sunday as I try to watch that on television rather than take the kids to swim lessons or Sunday school.

(Laughter.)

MR. TIPTON:  So we'll see how that goes.

Among the topics you all discussed at some length yesterday and today was the question of outcome studies and what the federal government can and cannot fund and those kind of questions.  I would point out that the NIH and the NICHD do, in fact, fund some limited outcome studies.  They have a trial underway now where the main site is at Baylor down in Houston looking at outcomes for ICSI children.

So I think there's certainly more that could be done there, and I think in terms of other directions to go, I think there is a huge, huge difference between an NIH funded multi-center outcomes trial and a mandatory reporting or tracking of all embryos created and, indeed, all children conceived and born.

I think those are very, very different animals, and those differences need to be considered with some care.  You have some significant privacy concerns if nothing else that I think you need to deal with in a little bit more explicit manner.  The outcomes work that we do with the CDC in terms of tracking the number of cycles of ART performed and how many of those lead to live births or multiple births of children is very difficult to do, in part, because infertility patients like to put their infertility experience behind them as soon as possible.

So I think doing other long-term studies has some significant obstacles.  Having said that, we are very supportive of more outcome studies, and I would comment to the committee looking at the recent data from the European Society of Human Reproduction, which I think only now was an abstract at a meeting and not yet fully published, but should be out in a few months, and we think that outcome was very important and very promising.

Finally, I would like to wish a happy birthday to Louise Brown, the first IVF baby who turns 25 today.  I think that's a very significant milestone, and as we reflect upon that milestone, I think it's worth noting and remembering the huge hue and cry and, indeed what in some ways now looks to be a little bit of hysteria that accompanied that birth around a technique which we are now nearly a million children into.

And I think maybe with a few notable exceptions most of those families are very grateful that the technique was invented or perfected by Professor Edwards and Steptoe, and I think we're a million happy children into it, and that should not be forgotten in your deliberations.

Thank you.

CHAIRMAN KASS:  Thank you very much.

Charles Queenan, Juvenile Diabetes Research Foundation.

MR. QUEENAN:  Good morning, Chairman Kass, and members of the President's Council on Bioethics.  Thank you for the opportunity to testify to you this morning.

My name is Charles J. Queenan, III.  I'm here today on behalf of my daughter Jenna, who is age 14 and suffers with juvenile diabetes, also on behalf of the million other Americans who suffer with juvenile or Type 1 diabetes, the Juvenile Diabetes Research Foundation International, and the Coalition for the Advancement of Medical Research.

Jenna was diagnosed with diabetes 11 years ago when she was only three years old.  Following her diagnosis, I very quickly became actively involved with JDRF.  I currently serve on the international board of JDRF and for the last two years I was the chair of the Research Committee for JDRF, and in that capacity was responsible for grants that during the last two years totaled over $200 million for diabetes research.

In addition, I have overseen the development of JDRF's embryonic stem cell research program.  JDRF has entered into stem cell research partnerships with three international governments.  We are also in discussions with another six international governments for such partnerships.

This year JDRF expects to commit over $10 million to stem cell research, ten million out of a total of 100 million research grant commitments, and JDRF is the only non-governmental organization represented on the United Kingdom sponsored international stem cell forum, which also includes representatives of 12 governments.

Much has changed over the last two years since the administration announced its policy for federal funding of embryonic stem cell research.  Stem cells continue to hold significant potential for a wide range of diseases and disorders that impact some 100 million Americans.  But it is clear that this area of science has not advanced as rapidly as it was envisioned when the federal policy was determined.

In our view it is time to consider expanding the availability of embryonic stem cell lines so that  millions of Americans can hopefully begin to see benefits from this research.

Let me provide you with an update on how embryonic stem cell research should impact juvenile diabetes.  JDRF is actively supporting embryonic stem cell research because we feel it holds enormous promise to play a critical role in understanding and curing juvenile diabetes.

Actually I should say it has promise to extend the cure for juvenile diabetes.  We are currently curing juvenile diabetes through transplantation of insulin producing cells, but we are currently helping only a very tiny percentage of those who have the disease.

Of the more than 250 individuals who have received these transplants, approximately 80 percent no longer require insulin injections to control their blood sugar.  To these men and women who were previously unable to control their blood sugar levels, even with multiple daily injections, this is, indeed, a cure.

Unfortunately, despite this unparalleled scientific progress that has been hailed as the biggest advance in diabetes research since the discovery of insulin, there's a severe shortage of insulin producing cells suitable for transplantation.  Only 2,000 cadaver pancreases are available annually from organ donors.  This means that the vast majority of the one to two million Americans with juvenile diabetes will never benefit from this therapy unless we can develop additional lines of insulin producing cells.

Embryonic stem cell research could play a key role in developing an unlimited supply of cells so that we might extend the cure for juvenile diabetes to all Americans who currently suffer from this devastating disease.

The current federal policy will never be sufficient to adequately  advance this promising field.  Just a fraction of the stem cell lines that were thought to have been available based on this policy can actually be used by a scientist, and even though stem cell lines might not safely be used for clinical trials because of the way they were derived.

We have worked closely with the White House, the Department of Health and Human Services, and the National Institutes of Health to try to implement the administration's stem cell policy.  However, research progress in this area to date has been frustratingly slow primarily because of the limited number of stem cell lines scientists have to work with.

The importance of having access to additional stem cell lines from excess in vitro fertilized eggs that would otherwise be discarded is crystal clear.  The time has come for a fresh look at the policy so that it can be revised in light of what we now know to more successfully move the research forward.

Take the following four examples.  First, the number of embryonic stem cell lines determined to be available under the President's plan was 78.  However, at present information about only 11 are on the NIH registry, and even fewer are fully available to researchers.

Second, most, if not all, of the embryonic stem cell lines on the NIH registry were developed using mouse feeder material as a nutrient.  We are very concerned that this could make these lines ineligible for use in human therapeutics because of the potential for cross-species contamination.

Third, while much concern has been expressed about the lack of genetic diversity in the limited number of embryonic stem cell lines that are currently on the registry, less attention has been paid to a related issue.  Scientists are finding that the various embryonic stem cell lines are proving to be much more heterogeneous than was previously thought, and specific lines are tending to differentiate to specific types of tissues.

This means that researcher access to additional stem cell lines will be even more important as we try to grow genetically diverse tissue specific therapeutics for a number of different diseases.

Finally, fewer than expected young scientists are entering this area of research.  Anecdotal evidence indicates that the reason for this, beyond the laundry list of practical considerations I have just discussed, is concern among young investigators entering a new field that there is a lack of long-term commitment by the federal government as evidenced by the present limited policy to provide a stable, creative, and nurturing environment for stem cell research.

Chairman Kass, I appreciate the Council's continued interest in embryonic stem cell policy.  JDRF shares the President's interest in advancing this field, and we look forward to working with you to develop new ideas and approaches to the current federal embryonic stem cell policy and to make the promise within this field of research a reality.

In conclusion, I just want to add that I understand that there is concern that some patient organizations may look upon this research with unrealistic and perhaps overly optimistic assessments of the difficulty in finding a cure, and I want to say for the record that in my experience both with other members of the JDRF board, thousands of volunteers both in JDRF and in other foundations, that really is not the case.

I think you would find that they are very well informed both about the scientific potential and the policy issues surrounding human research.  What we do want, however, is to avoid artificial barriers to moving forward the scientific research that must go forward.

Thank you very much.

CHAIRMAN KASS:  Thank you.

Kirsten Moore of the Reproductive Health Technologies Project.

Welcome.

MS. MOORE:  Thank you.  Thank you for this opportunity to present our comments.

My name is Kirsten Moore.  I'm president of the Reproductive Health Technologies Project.  We're a national, nonprofit advocacy organization, and our mission is to advance the ability of every woman of any age to achieve full reproductive freedom without access to the safest, most effective, appropriate and acceptable technologies for insurance her health and controlling her fertility.

We view technology not as an end in itself, but as an essential component for all women and men, including those who are underserved and historically been excluded to control their own health and fertility.  We believe each technology requires careful analysis of its safety, effectiveness, acceptability, appropriateness and ethical aspects, recognizing that these vary from person to person and community to community.

Almost two years ago we began a process of dialogue and education within the women's health community on the issue of stem cell nuclear transfer.  Over the past 18 months, we've invited experts, scientists, legal scholars, bioethicists, practitioners, and patient advocates to share their perspectives on the science policy and politics of SCNT to help inform the position of a number of organizations within the women's health and reproductive rights communities.

You will not be surprised to hear there is no uniform position on the use of SCNT for research or reproductive purposes among the many organizations and constituencies which make up this community.  At the same time there is a strong consensus among many groups that SCNT holds the potential to expand our knowledge of human physiology, and with that, to develop safe, effective treatments for a broad array of diseases and injuries.

The promise of such benefit from this research is significant, and to many organizations, including my own, the Society for Women's Health Research, the National Partnership for Women and Families, and the National Women's Law Center, justifies use of preimplantation embryos in such research.

At their request and with your permission I'd like to leave statements from the society and from the National Women's Law Center in this record.

Even with that consensus, there are varying opinions as to how such research should move forward.  Speaking from my own organization, we believe SCNT research holds potential for unintended consequences, as well as intended uses that may have adverse outcomes.

To maximize the benefits of this research and minimize the risk both to individuals and society at large, the Reproductive Health Technologies Project calls for policy proposals that will enhance and strengthen existing policy and regulatory guidelines which protect human subjects in research and insure that consumers can rely on the safety and efficacy of FDA approved products.

In establishing such federal or state oversight, we would like the following guiding principles to be observed, and I would like to point out that we ducked the question that you were dealing with earlier about exactly how to do this.  So these are just our thoughts, no agencies assigned.

The scientific rationale as well as the risks and benefits of proposed SCNT research are evaluated with public oversight and accountability.  The short and long-term safety and efficacy data on any therapies or procedures that use SCNT are made available to the general public on a timely basis and in an appropriate format.

Informed consent and confidentiality of all participants, including donors of genetic material who will not benefit directly from the research is ensured.

Adequate protections are established to minimize the potential that embryos created for use in SCNT research are used for the purposes of reproduction.  Payment for gametes and other genetic materials used in SCNT research which exceed the cost of participation are prohibited.

The same regulatory guidelines and enforcement mechanisms should apply to all SCNT research, whether or publicly or privately funded, and inappropriate activities are discontinued and violators subject to civil penalties.

Thank you.

CHAIRMAN KASS:  Thank you very much.

Before we break, let me make clear, as clear as I can, what the next steps are with the two things that we've talked about this morning.

At the break Carter Snead had to leave, but he and I spoke about the need to tap a number of you for some paragraphs on the topics on which you either eloquently spoke or we suspect you have some knowledge that would help.  You probably know who you are, but expect to hear some requests from us to help fill out some of those things on the monitoring and things talked about earlier.

With respect to the Beyond Therapy project, we'd like your written comments by the date indicated, and you should expect - we didn't quite finish additional materials to give you today, but you'll have them some time next week, the next two installments for your reading pleasure and critical comments having to do with the pursuit of less aging bodies and more happy souls.

Cheerful thoughts, good health.  Thank you for your attention and wonderful contributions.  Safe trip, and we'll see you in September.

(Whereupon, at 11:57 a.m., the meeting was concluded.)