Speeches & Testimony

Unfulfilled Promises and Stem Cell Research

Abbey S. Meyers
President
National Organization for Rare Disorders (NORD)


MIT
Cambridge, Massachusetts
November 20, 2001

I am Abbey Meyers, President of the National Organization for Rare Disorders (NORD). NORD is a non-profit voluntary health agency dedicated to the identification, treatment and cure of rare diseases. Under the Orphan Drug Act of 1983, a rare disease is defined as a health condition that affects fewer than 200,000 Americans. There are more than 6,000 known rare diseases, cumulatively affecting an estimated 25 million Americans. The majority of orphan diseases are genetic, but they span the entire gamut of illnesses with known and unknown causes.

With respect to stem cell therapy, there is little doubt that many rare diseases may someday benefit from this new technology. However, the arguments that may be inhibiting, and some say preventing, the advancement of stem cell research are rooted in profoundly personal religious, political, and moral principles. In America, where so many of our ancestors settled for the purpose of religious freedom, the arguments against the use of fetal stem cells either reinforce or violate our deepest moral and religious convictions. Some stem cell advocates suggest that religious zealots have hijacked their most precious freedom to decide when life begins, while stem cell opponents feel it is their moral duty to protect unborn fetuses at any cost. Both groups have every right to believe as they do. Nevertheless, many parents of frozen embryos don't want to pay the annual costs of keeping their fertilized eggs frozen indefinitely, so they are often thrown in the trash, which violates everyone's principles.

According to a May 2001 poll by the Coalition for the Advancement of Medical Research, 34 percent of Americans strongly support stem cell research, and 35 percent somewhat support it. Only 23 percent strongly or somewhat oppose this research, and the remainder are either undecided or don't care. But even those who disagree with President Bush's decision to confine fetal stem cell research to existing cell lines, do not have the political fortitude to overturn that decision. Why? I suggest that stem cell research is paying a heavy price for the medical technology hype of the past decade, which promised investors and the public more than the technology could deliver. Additionally we are seeing a rebound effect to highly publicized violations of human subject protection rules, which has seriously damaged public trust in the biomedical research enterprise.

Hardly a day goes by when the media isn't reporting on a new drug, device, or medical technology that holds the promise of curing a serious health condition. One need only read newspaper articles announcing the discovery of a new gene, which inevitably predicts that the discovery will lead to new treatments or a cure for a specific disease. The public is only beginning to realize that we still don't have any cures for diseases with genetic defects that have been known for decades such as Tay-Sachs disease and sickle cell anemia. Therefore, people have grown understandably skeptical about news of major medical breakthroughs and new scientific technologies, because so few of them have lived up to their promise.

There has been no scientific technology that has been more over-promised in the past decade than gene therapy Since the very first gene therapy experiment in 1990, propaganda about this small field of clinical experiments has been astounding. Fueled by small companies trying to attract investment capitol, and large research institutions that felt it necessary to capture the prestige of cutting edge research, they promised marketable products without any scientific basis for their predictions.

In fact, when I served as a member of the NIH Gene Therapy Subcommittee and the Recombinant DNA Advisory Committee (RAC) between 1989 to 1996, we would vote on a phase I protocol, and by the time I reached my hotel room I would hear news about our vote on CNN. In one case, the investigator announced right after our vote, that he had just called his hospital on his cell phone and told his colleagues to inject the vectors immediately because he didn't want to deny patients the treatment any longer than absolutely necessary. That night I watched the injection of the vectors into the first patient on CNN (so much for patient confidentiality), and the following morning as I ate my breakfast I read about the experiment on the front page of USA Today. Of course the patient still is not cured, which is an important fact that has not been reported by CNN or the research community.

The companies and hospitals that enjoyed spinning these stories didn't recognize the effect of their hype on the patient community. Companies aimed their announcements toward Wall Street, but it was heard on Main Street. The public received a loud and clear message that gene therapy was curing people, and if they or their loved one had an incurable disease they demanded access to gene therapy protocols. Meanwhile, at every meeting of the RAC we tried to get the message out that no one had been cured, and that phase I trials have no expectation of being therapeutic. Nevertheless corporate propaganda and the public's unrealistic expectations, lasted until the death of Jesse Gelsinger. It was only then that investigative reporters revealed that gene therapy, while a very promising technology, had not yet delivered on its promise. The public heard that message, and today gene therapy researchers are having problems soliciting volunteers for clinical trials.

Now I don't mean to fault the media because they only report on stories provided to them by research facilities, scientists and companies, but I do have a great deal of concern that the lofty promises of stem cell research may not live up to the headlines. Scientists, and particularly companies, have over-promised too many miracle cures, thus bringing into serious doubt the validity of medical predictions. It would have been more ethical and humane for stem cell advocates to underestimate the therapeutic value of this technology, and pleasantly surprise us later by surpassing humble goals.

A far more responsible approach must be taken if the research community is to regain the trust of patients and their families because the public is now just realizing that cures for genetic diseases are not imminent, despite gene therapy and despite the Human Genome Project. And yet we all want to believe that scientific promises will come true because we cannot lose hope that suffering will be diminished, and the lives of our loved ones will be saved.

Now we're embroiled in the political, religious, and scientific arguments surrounding fetal stem cell research, and we are being told by scientists that they must have unencumbered access to as many stem cell lines as possible. Why, then, is the public not enraged at the federal barriers constructed by the Bush administration? Where is the political resolve needed to force the President to expand access to fetal stem cell lines?

I would suggest that the public is not enraged because they have become skeptical about so many false promises of scientific breakthroughs. They prefer to sit back and wait until there is proof that stem cells actually work as scientists predict. If they do effectively treat or cure a serious disease in humans (not mice), embroiled in the political, religious, and scientific arguments surrounding fetal stem cell research, and we are being told by scientists that they must have unencumbered access to as many stem cell lines as possible. Why, then, is the public not enraged at the federal barriers constructed by the Bush administration? Where is the political resolve needed to force the President to expand access to fetal stem cell lines?

I would suggest that the public is not enraged because they have become skeptical about so many false promises of scientific breakthroughs. They prefer to sit back and wait until there is proof that stem cells actually work as scientists predict. If they do effectively treat or cure a serious disease in humans (not mice), I predict there will surely be sufficient political resolve to change federal policy and expand the cell lines. The public has been hurt too many times by Wall Street hype about gene therapy, xenotransplantation, brain tissue transplants for Parkinson's disease, etc., and stem cell research is paying a very heavy price for those errors.

Additionally, human subject protections have suffered repeated body blows in the past few years, and this has seriously eroded public trust in the entire research enterprise. Let me explain that I am a member of the National Human Research Protection Advisory Committee (NHRPAC), but I am speaking now not as a member of NHRPAC, and simply on my own behalf as a patient advocate.

The Gelsinger case raised serious questions about the adequacy of our human subject protection system, not only for gene therapy, but for all clinical research. It raised questions about conflict-of-interest, adverse event reporting, the process and substance of informed consent, as well as many other ethical questions. The problems found at the University of Pennsylvania were only symptomatic of problems found at numerous other research institutions, most recently at Johns Hopkins. As the Office of Human Research Protections has suspended federal funding and temporarily halted clinical research at numerous institutions for violations of human protection rules, the public has learned about these problems and they are appalled. As a consequence, fewer people are volunteering for research protocols, and the horrors of Tuskegee and Willowbrook are once again in the public's conscience.

The protestations of the research community, and its adversarial relationship with federal regulators, have only fed the flames of public mistrust. In most of these cases, it was not a matter of inadequate human protection rules, but rather a matter of scientists and Institutional Review Boards ignoring the rules. Too many researchers resent the red tape associated with human protection regulations, and they grudgingly ask for informed consent that is hardly understandable to the average patient, and often omits facts about embarrassing conflicts-of-interest. I certainly would want to know whether my doctor was paid $3,000 for referring me to a trial that may not be in my best interest!

If there is one message I can send to the research community it is this: Go back to your laboratories. Go back to your patients, and using the limited cell lines made available to you by the federal government, make the technology work. Restore the health of a few patients, extend their lives or relieve their suffering, and rebuild public trust by proving your theories are applicable to human health. Moreover, do not use threats to shake the public out of its complacency; despite claims to the contrary, fetal stem cell research will not stop because of the government's limitations. We know the private sector will underwrite the research, as will foreign governments. Remember, the patient community doesn't care where our medical advances come from. We know if the French or British or Russians can prove that fetal stem cells are therapeutic to humans, our nation's political pendulum will quickly swing back and we will have access to those treatments.

So prove to us that it works. Show us that the technology should not be denied to us, and we will do the rest. Unfortunately, public trust in the research enterprise has eroded profoundly since the deaths of Jesse Gelsinger and Ellen Roche, and it must be reconstructed. Avoid hype and spin, tell us the truth, give us realistic expectations, and protect human subjects not because you have to, but because you want to. Then we in the patient community will be your strongest advocates.

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