also generating knowledge about the mechanisms whereby the female hormone estrogen protects against bone resorption in osteoporosis. Research on inherited metabolic diseases advanced this year with the approval by the FDA of a treatment for children whose immune systems are destroyed by the deficiency of a key enzyme, called ADA. The new enzyme replacement treatment involves chemically protecting the critical enzyme to keep it from breaking down rapidly after administration. This novel enzyme delivery system was used to treat the first child to receive experimental gene therapy, and NIH investigators now wait to determine whether the newly inserted gene will make a normal product. Clearly, this new formulation of an enzyme treatment will be a valuable adjunct to gene therapy research. The NIDDK research mission is broad and complex, encompassing many costly chronic diseases; health problems of the young and the elderly; and diseases that particularly affect women and minorities. In highlighting some of our research advances, I hope I have conveyed the scientific momentum we feel as we pursue our mission into the 1990s. Mr. Chairman, the budget request for the NIDDK is $658,557,000. I will be pleased to answer any questions. BIOGRAPHICAL SKETCH OF DR. PHILLIP GORDEN December 22, 1934. Baldwyn, Mississippi. Married, two children. Education: B.A., Vanderbilt University, Nashville, Tennessee, 1957; M.D., Vanderbilt University School of Medicine, Nashville, Tennessee, 1961. Professional History: 1961-62, Intern, Yale University, New Haven, Professional Organizations: American College of Physicians, Honors, Awards: Alpha Omega Alpha Medical Fraternity, 1961. Doctor of Medicine (Honoris Causa) - University of Geneva, Geneva, Switzerland, 1986. Public Health Service Distinguished Service Medal, 1986. Public Health Service Commendation Medal, 1988. Distinguished Alumnus Award and Medal, Vanderbilt Medical School, 1990. INTERSTITIAL CYSTITIS Senator HARKIN. Thank you, Dr. Gorden. I know that Senator Reid has some questions for you. Senator REID. You are very kind, Mr. Chairman, in allowing me to appear out of order after you have been here all morning. Dr. Gorden, last year the committee provided the urology program an additional $2.5 million to be directed toward research on interstitial cystitis. Can you tell me how you intend to direct these funds? Dr. GORDEN. Yes, sir; at the present time we have a request for applications for new research project grants in this area, and in addition we are soliciting applications for the establishment of a data base or registry. Both of those programs, that is, this increased research project grant program and the registry or data base program, are well on their way, and will be inaugurated in the very near future. Senator REID. Will these moneys be used this year? Dr. GORDEN. They will be used this year with the possible exception of the data base/registry, which may be difficult to fund this fiscal year for technical reasons relating to timing of the review process. But if it is not this fiscal year, it will be done very early in the next fiscal year. Senator REID. Doctor, how much of the money is designated for the interstitial cystitis register? Dr. GORDEN. We do not have the proposals in, so I cannot be precise. But we estimate a figure of something in the range of about $650,000, which would include a data coordinating center and three proposed clinical centers that will actually support this data coordinating center with clinical studies. And that is our present estimation at this point. Senator REID. And is this a sufficient amount, as far as you know? Dr. GORDEN. We believe that it will be sufficient. It always has the possibility of flexibility in the future once we get this program started. Senator REID. I have some other questions that I will submit to the panel in writing, Mr. Chairman. I would acknowledge that I am going to visit your facility sometime early next month, I think on the eighth. Dr. GORDEN. We would be very pleased, Senator Reid. NUTRITION Senator HARKIN. Thank you, Senator Reid. Dr. Gorden, as you know, the American public is bombarded by endless diet claims that may or may not work and may or may not be healthy for those that follow them. It just seems to me that there is more now than I can ever remember. I have followed some of these, and I am concerned that a lot of people are investing money in doing things that may not be entirely healthy for them. This committee last year asked that NIH and give the giving a study of nutrition greater organizational status at NIH, perhaps even creating a division for the study of nutrition or to give some research focus to evaluating the many diets that we hear about. Your Institute is charged with studying obesity and has the lead on nutritional questions as well. Can you give us a rundown on your evaluation of giving a study of nutrition greater organizational status that we asked about last year? Dr. GORDEN. There is sort of two parts. One is the issue related to obesity, which we feel is a terribly important issue. The easiest thing in the world to do is to lose weight, and the hardest thing is to sustain that weight loss. And this is a major concern for us. We have a task force at present at work attempting to make recommendations in this arena to the point where our knowledge base ends, and where we would very much like to inaugurate clinical studies to address this very important issue of how to sustain weight loss. This is a critical issue as obesity is a major risk factor for a whole variety of other problems, including diabetes and cardiovascular disease and perhaps other neoplastic diseases. That is one aspect. We believe that we have an organizational structure that is capable of dealing with this issue. Nutrition research is vested in several institutes at the NIH. You have discussed some aspects of nutrition research already with Dr. Lenfant and Dr. Broder, and we are very pleased to cooperate with those institutes in furthering nutrition research and being sure that the resources are available for the kind of research that we need to be doing. So, we believe we have an organizational structure. We believe there are some terribly important issues. We have focused on the issue of centers, which we refer to as Clinical Nutrition Research Units. We support five at the present time, and feel that this is a very important mechanism by which we can address these particular problems of nutrition. We would prefer to utilize this more direct mechanism of trying to deal with the issues that you refer to rather than sort of organizational changes within the NIH per se. Senator HARKIN. So, you feel that this is getting adequate attention. Dr. GORDEN. I believe it is getting adequate organizational attention. Senator HARKIN. But your Institute is sort of the lead on this. Right? Dr. GORDEN. Yes; we are. Dr. RAUB. Mr. Chairman. Senator HARKIN. Yes, Dr. Raub. Dr. RAUB. May I add that we have a particularly strong central effort. There is not only a nutrition coordinating committee, but it has a full-time staff based in the NIH Director's office. That staff works very closely with Dr. Gorden, with colleagues in the Heart, Cancer, Child Health, and Aging Institutes, and the Nursing Center, and others to ensure a good flow of information, but in some cases it promotes certain initiatives. For example, going back several years, the concept of the Clinical Nutrition Research Units actually was launched under the umbrella of the central coordinating group. In addition to the five funded by Dr. Gorden's Institute, three others are funded by the National Cancer Institute. We believe we have a strong handle on the trans-NIH aspects of nutrition. DIABETES Senator HARKIN. My time is running out, but I do want to ask you one other question about juvenile diabetes. I understand that type I is the result of the immune system destroying cells in the pancreas. We have been told in the past that of the two types of diabetes, this type is most likely to result in a cure. Last year this committee provided an additional $2 million to pursue promising research to find better treatments and a cure for type I diabetes. Can you just give me a brief update on this on how we are proceeding? Dr. GORDEN. Yes; there have been several important specific discoveries in the past year. One is the recognition of markers that precede the onset of the clinical disease and actually refinement of the chemical nature of these markers. We now have the opportunity to actually detect this disease at least 3 and perhaps 5 years before its clinical onset. It used to be that we believed that a child was perfectly well today, and tomorrow that child had juvenile diabetes. That is almost certainly not the case. The process is going on for months and years, and we now have the ability to detect that process by a chemical marker. That provides a very important opportunity now to try to develop prevention strategies, and we are at the present time looking at several proposals that might be used to interdict the process at this very early stage. Our ability to detect it and our ability to use drugs or other pharmacologic agents to actually prevent it-agents that we can be certain are safe is something that we have under discussion. It is a difficult issue because we do not have what we would like to think of as a "magic bullet," so to speak, that is perfectly safe and perfectly effective. If we had that, it wouldn't be a matter of discussion at all. But this research certainly is an opportunity. We have developed other ways of achieving immunologic privilege. Other studies have demonstrated that transplants of islet cells that produce insulin in particular areas of the body seem to be immunologically privileged. This raises further hope for transplantation types of therapies either from direct islet cells themselves or from whole organ transplants. So, I think that we clearly have a focus on this disease that we have never had before, and we have now, I think, a real opportunity for prevention and, we hope, for understanding the cause and the cure. QUESTIONS SUBMITTED BY THE SUBCOMMITTEE Senator HARKIN. Thank you very much. There will be some additional questions which will be submitted for your response in the record. [The following questions were not asked at the hearing, but were submitted to the Institute for response subsequent to the hearing:] |