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Here is the testimony from the Doctor who is in charge of Adult Stem Cell Research, as you can see, he's not ready yet for a "Moon Shot". just me, Marjorie United States SenateTestimony of Darwin J. Prockop, M.D., Ph.D. Director, Center for Gene Therapy Tulane University Medical Center Before the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education United States Senate September 14, 2000 Good Morning. My name is Darwin Prockop. I am currently the Director of the Center for Gene Therapy of Tulane University Health Sciences Center in New Orleans, Louisiana. I have an M.D. degree, a Ph.D. degree and two honorary doctoral degrees. In addition, I am a member of the National Academy of Sciences and the Institute of Medicine. I have published over 400 scientific articles. My work has been continuously supported by the National Institutes of Health. In addition, the Tulane Center for Gene Therapy that I now head is supported by funds from Tulane University, the Columbia Healthcare Association and the State of Louisiana Consortium for Gene Therapy. Our Tulane Center for Gene Therapy is carrying out research on a special class of adult stem cells that we think can potentially be used for the treatment of a number of devastating diseases. The adult stem cells we are using were first discovered 20 years ago, and we now know they have a number of truly remarkable features. One remarkable feature is that they can easily be obtained from a small sample of bone marrow from the same patient who is to be treated. Therefore if our therapies work, we will not need fetal tissues nor will we need to introduce foreign cells into a patient. Another important feature of the cells is that we can grow them rapidly in the laboratory. A technical consequence of this fact is that we can introduce new genes without the use of a virus. But the cells are of special interest primarily because they can change into a large number of the many different types of cells found in the human body. For example, we discovered several years ago that if the cells are isolated from the bone marrow of an animal, and then injected back into the blood stream of the same experimental animal from which the cells were obtained, they travel to a number of different organs and tissues. Moreover, when they reach a given organ or tissue they become new cells of the same kind that are normally found in that organ or tissue. In effect, the cells can repair and rejuvenate the organ or tissue. These features of the cells raise some exciting possibilities for using them in the therapy of diseases. For example, after infusion into a vein, some of the cells go to bone and become new bone cells. Therefore, the cells can potentially be used to treat diseases of bone such as osteoporosis. In fact, Drs. Edwin Horowitz, Malcolm Brenner and others at St. Jude's Children's Hospital In Memphis have recently reported promising results with the cells in treating children with severe brittle bone disease. Some of the cells go to lung and become lung cells. Therefore, they can potentially be useful in treating diseases of the lung such as cystic fibrosis. Some of the cells go to joints and cartilage and become cells of joints and cartilage. Therefore, they can potentially be used to treat diseases of the joint such as arthritis. Some preliminary data suggest that in the future the cells may also be useful in treating additional diseases such as diabetes, heart disease and kidney disease. One of the most dramatic observations about such cells is that they can become new cells of the brain. The first observations here were made one and two years ago in my laboratory primarily by Dr. Asum Azizi and Dr. Donald Phinney. In the past several months these observations have been extended by Dr. Ira Black and his associates at the Robert Wood Johnson Medical School and by research groups at two other medical schools. Because the cells can become some of the cells of the central nervous system, my laboratory and others are currently trying to see if they can be used to treat disease of the central nervous system such as Parkinsonism, Alzheimer's disease, stroke, spinal cord injury and multiple sclerosis. In fact, we have recently published some promising results using the cells in a rat model of Parkinsonism. In closing I would like to make two general statements. One is, yes, it is true that if the work that my laboratory and many others are now doing continues to be successful, it will open the possibility of using stem cells from adults, or even from the patient himself or herself, to treat a large number of terrible diseases. If successful, the therapies will not use any fetal tissues and probably will not use any viruses. However, I would like to stress the second very important point: we are not there yet. We have a long way to go. In my estimation, it will be at least two years before the first adult stem cells can be tested in patients with diseases such as Parkinsonism. It will probably be much longer for testing the cells in patients with the other diseases I have mentioned. We simply cannot be certain in advance which therapies will work and which will not. We need several years of hard work and we need continuing support from sources such as the National Institutes of Health and other sources such as the Louisiana State Consortium for Gene Therapy and the Columbia Healthcare Association that are currently supporting the Tulane Center for Gene Therapy. In my opinion, it would be a serious mistake to stop all research on human embryonic cells and tissues because of the exciting discoveries my laboratory and others have recently made about adult stem cells. We are simply not ready for a moon shot-like strategy in which we place all our bets on adult stem cells. I think it would be a mistake to tell the millions and millions of people whose lives are being destroyed by these terrible diseases that they to wait three, four, five years or even longer until we see the results obtained with adult stem cells before we even begin doing research on the other kinds of tissues and cells that may cure their diseases. I myself would be extremely sorry to see this sub committee or any other group decide that because of the work we and others have done on marrow stem cells, the kinds of research called for in the new NIH guidelines should be stopped.