entists to conduct advanced research abroad and bring new techniques to the United States. SCIENTIFIC EXCHANGE FOR EASTERN EUROPE AND THE SOVIET UNION Senator HARKIN. I appreciate that, Dr. Schambra. One last thing, with what is happening in Eastern Europe and the Soviet Union, it seems like we have an opportunity here to greatly expand scientific exchange in those countries. Are you proceeding along that line? Dr. SCHAMBRA. Yes; we are doing the best we can. We certainly, as I know you and the rest of the world, have been quite fascinated by what is happening in the countries behind the former Iron Curtain. One of our greatest opportunities today is to significantly expand international cooperation with Eastern Europe and the Soviet Union on health and biomedical research problems. We have surveyed the interest of NIH scientists in expanding cooperation with their colleagues in these countries and also have heard from many biomedical scientists throughout the United States about their interests. Areas for scientific collaboration have been identified, including AIDS, environmental health, cancer, and cardiovascular diseases. Many of our scientists believe that Eastern Europe and the Soviet Union offer unusual and important collaborative opportunities. They have also noted that highly competent Soviet and Eastern European biomedical scientists are eager to work directly with United States researchers. This could produce benefits for United States science. The Soviet Union and countries of Eastern Europe have expressed their desire in participating in FIC programs. There is a great interest in, and great gains to be achieved from, biomedical research cooperation with these countries. An area where Americans and Eastern Europeans have a strong common interest is in combating the spread of AIDS. We are, as I say, doing what we can within the resources available to try to meet some of those needs, and have also developed inhouse proposals for a substantially expanded effort in Eastern Europe which I would be pleased to submit for the record. [The information follows:] PROPOSAL FOR UNITED STATES-EASTERN EUROPE FUND FOR BIOMEDICAL RESEARCH COOPERATION The political, economic, and social reforms occurring in Eastern Europe provide unprecedented opportunities for American scientists to cooperate with East European colleagues. Foreign policy priorities and the scientific benefits of joint research suggest there would be immediate value in the establishment of a program to support U.S.-East European biomedical research cooperation. If such a program is structured with flexibility, it could be rapidly responsive to new scientific opportunities and produce benefits for the East European technical community while contributing to long-term progress on U.S. biomedical research priorities. The Fogarty International Center (FIC) of the National Institutes of Health (NIH) is in a unique position to implement such a program and to provide leadership in fostering contacts between East European and American biomedical researchers. As the focal point for NIH international activities, the FIC has a long history of direct collaboration with many countries of Eastern Europe including Poland, Hungary, Bulgaria, Yugoslavia and Romania. FIC facilitates the international activities of NIH by developing direct scientist-to-scientist linkages between U.S. and foreign researchers. In relation to Eastern Europe, FIC has the flexibility and mandate required to move quickly and efficiently to take full advantage of scientific and politi cal opportunities if resources are available. FIC also has the necessary linkages to the foreign policy community. A program to support U.S.-East European biomedical research collaboration through the FIC should be funded at a level of $4 to $5 million per year for a minimum of five years. These resources would generate long-term technical linkages, support research and research training, and expand scientist-to-scientist contact. The overall goal would be the development of collaborative relationships which would extend beyond the period of initial involvement. Specifically this fund for U.S.-East European Biomedical Research Cooperation would include five major components (with estimated costs for fiscal year 1990): --Research grants for U.S. scientists to conduct collaborative research with Eastern European scientists (26 grants for $1,300,000); -Training grants in U.S. institutions for Eastern European scientists and health professionals in the field of AIDS (20 trainees for $600,000); -Research Fellowships to support Eastern European scientists in U.S. biomedical research laboratories for 1-2 years (40 fellowships for a total of $1,200,000); --Short-term exchange visits (up to 6 months) between U.S. and Eastern European scientists to develop and promote research collaborations (50 exchange visits for a total of $400,000); and -Scientific workshops to explore mutual interests, develop linkages and define areas of promising scientific collaboration (10 workshops for a total of $500,000). PARTICIPATION Countries involved in this initiative may include Bulgaria, Czechoslovakia, East Germany, Hungary, Poland, Romania, and Yugoslavia depending on the status of reform in each country and the availability of scientific opportunities. A parallel program could be established for the Soviet Union if circumstances warranted. The FIC will administer this initiative in close collaboration with the NIH Institutes, other components of the U.S. Public Health Service, the Department of State and others involved in international collaboration. FIC has extensive management experience in all areas of proposed program activity. Senator HARKIN. That concludes my questioning. I will have additional questions for each of you for the record that we will be submitting to you, and thank you for being here, and again, my personal thanks to each one of you who are doing such a fine, fine job. NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE STATEMENT OF DR. MURRAY GOLDSTEIN, DIRECTOR SUMMARY STATEMENT Senator HARKIN. The next panel, Dr. Murray Goldstein, Director of the National Institute of Neurological Disorders and Stroke; Dr. Jay Moskowitz; Dr. James Snow; Dr. Ruth Kirschstein; Dr. Carl Kupfer, and Dr. T. Franklin Williams. We will proceed just as we had with the previous panel in the order which I called them. First, the National Institute of Neurological Disorders and Stroke. Dr. Goldstein, we are delighted to have you with us again. Your budget request is $512,210,000 about a 4.45-percent increase, and again, all of your statements will be made a part of the record in their entirety. If you could summarize or highlight what you think we should know, I would appreciate that. Dr. GOLDSTEIN. Thank you very much, Mr. Chairman. This is the 40th anniversary at the Institute and the first year of the "Decade of the Brain," and I am particularly pleased to be able to share with you some very amazing results. GAUCHER'S DISEASE I have before you an exciting set of photographs. Within the last month we have had an exciting breakthrough. There is a terrible disorder known as Gaucher's disease, a genetic disorder primarily of children, which leads to disability and to death. There has been no treatment for this disease and the picture on top is that of a child with the disease. You notice the child has a very large belly. The abdomen is bloated, not because of fluid, but because the child's liver and the child's spleen have grown in size. This child would have died in the near future. Below it you have the picture of 3 months after treatment. Subsequently, the use of enzyme replacement therapy which was perfected in our own laboratories at the Neurology Institute at NIH. This was so impressive, quite frankly, sir, we did not believe it. So, we tried it on 12 other patients and each responded dramatically. It is an amazing breakthrough. It is the first time in a genetic metabolic disorder that enzyme replacement therapy has been successful. There are remaining questions to be answered. How long will this result last? Is this result something that is temporary now and 1 year from now ineffective? I do not know the answer to that, but I did want to bring it to your attention. We have had this important breakthrough and are looking forward to pursuing it with vigor. It also sets an interesting stage for the treatment of other genetic disorders of the brain and nervous system. As you know, 25 percent, one-quarter, of all genetic disorders occur in the brain and nervous system. So the nervous system is a common site of genetic abnormalities. We should not be surprised at that because of the anatomic and biochemical intricacies of the system. Unfortunately, there are many places that development and functioning can go wrong. But this breakthrough does set an important stage. As we progress and learn more about the genetic diseases of the nervous system: neurofibromatosis, Huntington's disease, dystonia; we now have one successful model that can be utilized as we learn more about possible treatments for those other diseases. PARKINSON'S DISEASE A second very important breakthrough has been the development of an exciting new drug, for the treatment of Parkinson's disease, deprenyl. What is of equal importance is that it is the first time a drug has been developed that has stopped the progression of a brain degenerative disease. This has never been possible before. For the first time, in the case of Parkinson's disease, we have such a drug. It is now on the commercial market and readily available for patients with Parkinson's disease. Senator HARKIN. And it absolutely stops the progression? Dr. GOLDSTEIN. It stops the progression of the disease. Now what I cannot tell you, as with Gaucher's disease, is for how long. That I do not know. We have continued the study in order to answer the how long questions. We decided, and it is a multimillion dollar study, to continue the study for 5 more years, to find out the longterm effects of the drug. My own guess is that it will have long-term effects, literally stop the progression of a brain degenerative disorder. STROKE AND ATRIAL FIBRILLATION The third important breakthrough is one that will be reported in 2 weeks in the scientific literature. I am going to be a little diffuse in my telling about it because scientific journal editors get very disturbed when we scoop them. There is a chronic disease of the heart of older people known as atrial fibrillation. It usually does not have any profound effect on the person who has this disease. It is sometimes a bit debilitating, but rarely life-threatening. The threat of the disease is that it does send emboli to the brain because the heart is irregular and little pieces of a blood clot break off and go to the brain. This causes stroke and it is not unusual to cause stroke in elderly people. We are just completing a study in which we now have a medication which clearly prevents this from happening. In a few weeks, this information will be available to physicians. The point I am really trying to make, sir, is that after 40 years of generous support by the Congress for the Institute's program of basic research, we are now able to convert this information into clinically useful modalities. We are now entering the arena of clinical research and the development of improved methods of prevention and cure of brain disorders. Within this year, we have had three important outcomes of successful clinical research that will have long-term consequences on the quality of life of persons with brain and other neurological dis eases. RESEARCH FAILURES Now, let me tell you about three of our failures. One is Lou Gehrig's disease, ALS. We do not know what causes it. We still do not have any treatment to either prevent it or treat it. It is one of our failures. We are working on it, but I must admit, sir, we have not been successful. A second failure is with stroke. The mortality rate from stroke has dropped 40 percent in the last 15 years. That is an amazing drop, but within the last 2 years, the incidence of new cases of stroke have begun to increase again. We are hard at work trying to figure that out. Why is it going up, when mortality continues to fall? We don't yet know. We have very good data on stroke occurrences in white populations because the base for long-term studies have been on whites. We are now trying to find a population of blacks and orientals in which there is not a great deal of movement in and out of the community. This is absolutely necessary to find out if this increase is in fact occurring also in these minority populations. We are presently working with two groups of investigators in an attempt to get those data. PREPARED STATEMENT Finally, our last failure is we still have done remarkably poorly in the treatment of head and spinal cord injury. We are working on that issue, but I have nothing of any positivity to report. Sir, those are our successes and failures at the beginning of our 40th year. I would be pleased to answer any questions you might have for me. [The statement follows:] 26-182 0-90—31 |