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information transfer and health care activities.
Expansion of the centers program will stimulate the overall effort against diabetes. Expansion of all mechanisms of diabetes research will allow faster progress in diabetes research.
Question: What additional areas could the various centers profitably investigate?
Answer: The diabetes centers environment provides a particularly good opportunity to investigate many aspects of diabetes from a multidisciplinary approach. Thus, questions concerning the etiology of diabetes can be addressed in a single setting by virologists, geneticists, immunologists, epidemiologists, pediatricians and internists. Investigations regarding the mechanisms by which hormones act, particularly insulin, can be conducted in an integrated fashion by biochemists, physiologists, molecular biologists and internists. These are but a few of the many areas of research on diabetes that could be profitably investigated within the diabetes centers. The diabetes centers also provide an excellent environment in which to conduct clinical investigation, for example, to study in man the relationship between the metabolic abnormalities of diabetes and the development of the chronic complications of this disease.
LOW BACK PAIN
Question: What did you learn from this workshop and how will the outcome affect your research activities for the 1982 budget year?
Answer: The workshop on low back pain was divided into 5 sections: Epidemiology and Natural Course; Anatomy and Ultrastructure; Biomechanics; Neural Mechanisms; and Biochemistry of Supporting Structure. Each section prepared a summary paper of the major findings to date and a list of promising questions for future research. In many such areas, investigators from more than one discipline would collaborate to produce a more clinically and scientifically significant study.
The forthcoming book based on the proceedings of the workshop and a summary to be published in the journal Spine should serve to stimulate interest in the area by the general academic community. New grant applications for projects in research in this area may therefore be anticipated during the 1982 budget year.
Senator SCHMITT. The subcommittee will stand in recess until 2 p.m., when we will hear further budget requests.
[Whereupon, at 1:30 p.m., Wednesday, February 18, the subcommittee was recessed, to reconvene at 2 p.m. this same day.]
(AFTERNOON SESSION, 2:05 P.M., WEDNESDAY, FEBRUARY 18, 1981)
NATIONAL INSTITUTE OF NEUROLOGICAL AND COMMUNICATIVE DISORDERS AND STROKE
STATEMENT OF DR. MURRAY GOLDSTEIN, ACTING DIRECTOR
DR. DONALD S. FREDRICKSON, DIRECTOR, NATIONAL INSTITUTES OF HEALTH
DR. KATHERINE BICK, ACTING DEPUTY DIRECTOR
WILLIAM B. MATTHEWS, JR., BUDGET OFFICER
NORMAN MANSFIELD, DIRECTOR, DIVISION OF FINANCIAL MAN-
ANTHONY ITTEILAG, ACTING DEPUTY ASSISTANT SECRETARY,
Senator ANDREWS [presiding]. The subcommittee will come to order. This afternoon we're going to resume our hearings on the National Institutes of Health. Our first witness is Dr. Murray Goldstein, Acting Director, National Institute of Neurological and Communicative Disorders and Stroke. As I understand it, the Institute's mission is to support and conduct research into disorders of the nervous system, including the brain, spinal cord, nerves, and muscles, as well as research into problems of hearing and communication.
Doctor, we welcome you, and ask that in the interest of time-and I'm sure your colleagues have told you about the extensive hearings we had this morning-that you very briefly highlight your opening statement so that we may quickly get to questions. And I would appreciate it if you would introduce your colleagues. And then you may proceed. [The statement follows:]
STATEMENT OF DR. MURRAY GOLDSTEIN
Fiscal year 1981 has been a year of both success and difficulty in our nation's research effort to prevent and treat the devastating effects of disorders of the brain, the spinal cord, the nerve-muscle apparatus, hearing and that uniquely human attribute of speech and language.
The brain tells us what we are, who we are, where we are and controls our relationships to the environment by means of thought, memory, speech, movement and the coordination of every organ of the body. Malfunctions of this amazingly sensitive and complex system can easily, and often do, lead to death. However, perhaps even more ominous to the individual and society, these disorders if not fatal, inevitably lead to a significant decrease in the quality of life characterized by severe disabilities such as paralysis, dementia, convulsions, deafness or intractable pain.
Despite impressive advances in the prevention and treatment of stroke, it remains the third leading cause of death and a major cause of long-term disability. Our success against the results of accidental injury to the brain and spinal cord--the nation's leading cause of death in persons under 45 years of age--remains poor and establishes head and spinal cord injury as a modern epidemic of continuingly increasing proportions rivaling even heart disease, cancer, and stroke. Dementia--the loss of intellectual function due to brain degeneration--in itself does not cause death, but the disease--and it is a disease and not a normal phenomenon of the aging brain--accounts for more institutionalized or hospitalized patients in the U.S. in any single day than all other diseases combined. When one adds to these examples the consequences of the developmental disorders of the nervous system of children (e.g., Tay-Sachs disease, spina bifida), infectious disorders of the brain or its covering (e.g., Reye's syndrome, meningitis), epilepsy, the still unknown causes and ineffective treatment of multiple sclerosis and of amyotrophic lateral sclerosis (Lou Gehrig's disease), the muscular dystrophies, and hearing loss due to childhood diseases and environmental trauma, the aggregate adds up to a total of 600 known diseases of the nervous system and of human communication for which the NINCDS has primary research responsibility.
Every Institute is pleased to be able to report on successful accomplishments. On behalf of the NINCDS, I have selected five (5) achievements which are representative of research progress made this past year.
The first is the unprecedented explosion of new knowledge about the basic functioning of the brain and nervous system. very large proportion of disorders for which the NINCDS is responsible is characterized by unknown causes and therefore the availability of only symptomatic therapy and inadequate methods of prevention; thus, the need for the priority given to basic research in the neurosciences and communicative sciences. As a result of this research, more has been learned about the way the nervous system works in the past few years than in the total of the previous fifty years. Methods are now available to probe how the nerve cell functions, how it communicates with other cells
and how it reacts to signals from the environment. We now recognize that the nervous system is not only a series of finely balanced electrical-like relay stations but is also a complex chemical factory that modulates the activity of every body tissue by means of the production of peptides, neurotransmitters and neuroendocrine agents.
Another success involves an innovative research approach which the NINCDS was able to initiate just one year ago-positron emission tomography (PETT). Last year, our recently retired Director, Dr. Donald Tower, described for you the excitement surrounding a technological breakthrough for studying in human beings the metabolism of the brain in health and disease. Seven PETT research centers and a center at the NIH were established to apply the use of this advanced space-age methodology to the study of the human brain. The early success of this investment has already provided completely new insights on the reaction of the brain cell to stroke, the metabolic activity of the brain in epileptics and the site of pathology in Huntington's disease.
The third area of success I would like to comment upon is that of replacement therapy for enzyme deficiency disorders of infancy and childhood. These are a class of disorders characterized by the pathological accumulation of metabolic materials in brain and other tissues because of the lack of a genetically controlled enzyme necessary for their disposal. This enzymatic deficiency leading to excessive storage causes the cells to degenerate, failure of the brain and body to develop, and ultimately death. To date, there has been no treatment for these genetic disorders. An example of this type of disorder is Tay-Sachs disease, an inevitably fatal genetic disease of infancy and childhood. NINCDS and other NIH scientists and grantees have untangled the complicated enzyme stories underlying this condition and other similar disorders. The NINCDS Developmental and Metabolic Neurology Branch is providing experimental enzyme replacement to a few children with a related disorder known as Gaucher's disease to see if we can overcome the deficiency. pleased to report that the treatment appears to be effective and children under study are apparently developing normally, the genetic defect having been overcome. On the basis of these preliminary findings, the NINCDS hopes to develop methods to produce larger quantities of the needed enzyme so that more patients can be helped, develop animal models to test the safety and efficacy of new enzymes, and launch carefully controlled clinical trials of treatment for this and other enzyme-deficiency diseases.
The fourth area concerns progress against the principal fatal disorder of the nervous system, stroke. As a result of the joint research effort of the NINCDS and the NHLBI, stroke as a major health problem is gradually coming under control as indicated by the significant decrease in stroke incidence and stroke mortality. Our success against stroke is not the result of a single dramatic scientific breakthrough but rather the cumulative effect of a series of contributions over time. A most important consequence is the decline of the hemorrhagic aspects of stroke for which improved methods of hypertension control and advances in neuro-surgical therapy have substantially diminished the impact of this great killer which previously was 80 percent fatal. However, we now recognize that if all