10 January 1999 Testimony on Stem Cell Research Presented by Douglas Melton, Ph.D. Good morning Chairman Specter, Senator Harkin and other members of the subcommittee. My name is Doug Melton and I appear before you today on behalf of the Juvenile Diabetes Foundation International and the millions of families in this country touched by diabetes. I am Chairman of the Department of Molecular and Cellular Biology at Harvard University, but more importantly for today’s discussion, I am here today because my 7 year old son, Sam, has had Type 1 (or insulin-dependent) diabetes since he was 6 months old. I am here this morning as a father and a researcher, as someone who can speak to you about the human burden of diabetes and the scientific potential of stem cell research. Mr. Chairman, before I begin my remarks on the topic of this morning’s hearing, I want to take this opportunity to thank you and the other members of this subcommittee for your leading role in last year’s historic increase in NIH funding. The strong bipartisan support for NIH exhibited last year provides renewed hope for those of us struggling with chronic disease on a daily basis. Founded nearly 30 years ago by parents of children with diabetes, JDF is a voluntary health agency dedicated to finding a cure for diabetes through the support of research. JDF gives more money to diabetes research than any other nonprofit, non-governmental organization in the world. This year, the Foundation expects to commit nearly $65 million directly to diabetes research. Diabetes is an insidious disease, and remains widely misunderstood by the general public. Insulin is not a cure for diabetes, it is merely life support. Diabetes is the leading cause of new adult blindness, kidney disease, and non-traumatic amputations, costing this country nearly $100 billion annually. Sixteen million Americans suffer from diabetes. On average, a person with diabetes can expect to live 15 fewer years than someone who does not have the disease. In addition, it is one of the most common chronic diseases affecting children, a group upon which it has an especially devastating impact. Before discussing the exciting potential of stem cell research, allow me to speak briefly as a parent. The daily regimen of Sam’s blood checks and insulin injections (up to 5/day) are coupled with our need to balancing his diet and exercise: a serious challenge in dealing with a 7 year old soccer player. The medical troubles for Sam are compounded by the vigilance and worry that extract a heavy toll on the rest of the family. For example, my wife is regularly up in the late hours of the night doing blood checks while Sam sleeps: we wonder is his blood sugar too low? Will he find the middle ground between a "low" or coma and being too "high" in the morning? I can’t recall a night since Sam was diagnosed when we slept peacefully, free of the worry that the balance between his food, insulin and exercise was not good enough. I’m unwilling to accept the enormity of this medical and psychological burden and I am personally devoted to bringing it to an end for Sam and all type I diabetics. I implore you to continue to make it possible to cure diabetes, for diabetics and their families. Human Stem Cell Research: A Potential Cure for Diabetes I would like to commend the Subcommittee for holding these important hearings on stem cell research. The recent discoveries in the field hold the potential to end all of this and finally find a cure for diabetes. Based on these discoveries, it is now foreseeable that human stem cells could be stimulated to develop into pancreatic islet cells to replace those that have been destroyed in individuals with Type 1 diabetes. Stem cells have the potential to develop into any tissue or organ in the body and yet cannot develop into a full human being. Moreover, these cells could be engineered in such a way that people who receive them might not need highly toxic immunosuppressive drugs, which prevent the body from rejecting "foreign" tissue–currently a major obstacle to successful islet transplantation. One of the most promising ways of curing diabetes is to restore biologically the function of islet cells. This could occur either through islet cell transplantation or through engineering of cells to restore the insulin-secreting function. In both instances, the availability of stem cells would significantly expedite research progress. Islet cell transplantation has been largely unsuccessful for two important reasons:
The problem of insufficient supply of islet cells could potentially be solved through additional stem cell research. Because the cells being studied are so early in their developmental stage, we are hopeful that we will be able to one day direct their development into any human tissue or organ. If and when scientists can specialize these cells to become insulin-producing islet cells, cell lines could be developed to produce an unlimited number of islet cells. This would effectively solve the islet cell supply problem. In addition, in most cases, the immune system of a person with Type 1 diabetes will not tolerate an islet cell transplantation, even when an individual is given anti-rejection medications (which themselves can cause serious problems). Because stem cells are primordial all-purpose cells from which all tissues of the body develop, it may be possible to alter them genetically so that they will not be susceptible to an immune attack. This would negate the need for immunosuppression. Ethical Considerations JDF understands that stem cell research raises important ethical considerations that need to be addressed. However, we feel that appropriate safeguards can and should be established to ensure that this important research can be conducted using federal funding. The 1994 Human Embryo Research Panel, which included scientists and ethicists, studied the ethical issues raised by this type of research and concluded that stem cell research involving "preimplantation" human embryos is acceptable for federal funding. We believe that the Panel’s report provides a scientific and ethical basis to justify federal funding for human stem cell research. This report could also serve as the basis for the establishment of a set of safeguards to ensure such research is conducted ethically, in both public and private settings, while still allowing for significant advances in the fight to cure diabetes. Summary Mr. Chairman, the opportunities presented by human stem cell research offer us the promise of significant advances–and perhaps a cure–for diabetes. A world without diabetes for all of the children and adults who currently suffer from its devastating impact continues to be our goal, and we urge you to ensure that federal policies allow this research to continue to speed our path to cure this disease.
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