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E--in combination with deprenyl to treat people with Parkinson's disease are
being evaluated in an ongoing clinical trial. Study of tissues from people
with amyotrophic lateral sclerosis, or Lou Gehrig's disease, has provided a
lead to evaluate the use of branched-chain amino acids for this disease.
One third of the people who will have a stroke this year will become
permanently disabled: stroke is the most common cause of disability requiring
The NINDS research effort on stroke will be enriched by a
newly established intramural program.
We also have ongoing clinical trials
to evaluate clot-dissolving agent g--CPA and heparinoids--for their
effectiveness in limiting brain damage from ischemic stroke.
We are working
with a time-frame of only 90 minutes to three hours to determine whether tPA
must be given as an emergency measure in order to be effective.
also underway to elucidate risk factors and prevent stroke in those people at
risk for an initial insult or recurrence.
Within the past year, the NINDS
found that aspirin and warfarin were so effective in preventing stroke in
people with atrial fibrillation that as many as 30,000 people could be spared
from a stroke this year, at a potential health care savings of $200 million.
Early results from another, ongoing study have related ethnic differences in
risk factors to differences in stroke type and outcome.
NINDS has revised
and reissued a program announcement to encourage new studies in Blacks, other
minorities, and women to improve our understanding of different risk factors
and types of stroke in various populations.
Intramural scientists using magnetic resonance imaging (MRI) have
evidence that multiple sclerosis (MS) progressively attacks the central
nervous system even when patients may be relatively symptom-free. This finding will change the way many patients are treated, especially in the
early stages of the disease.
In other work, intramural and extramural
investigators are deciphering the immunologic processes related to MS and
exploring new forms of treatment such as the possibility of a T-cell vaccine.
At a minimum, 25 percent of all genetic disorders affect the brain and
rervous system, some estimate that there are neurological consequences in
neurofibromatosis I (NF-1) gene has been located on chromosome 17; ongoing
research will clarify the gene's normal function and its role in the symptoms
of NP-1 and other di
rders of cell growth such as cancer.
closing in on the genetic basis of some forms of epilepsy. Intramural investigators are refining the optimal dose for enzyme replacement therapy in
Type 1 Gaucher's disease while continuing the development of methods to
repair or replace the defective gene. Dystrophin research has unexpectedly revealed that other genes may be producing proteins that can "pinch hit" for
dystrophin and mitigate the symptoms of muscular dystrophy. Both genetic
linkage studies and studies of cell biochemistry are probing independently the underlying causes of the various types of Batten', disease. In other
studies, researchers are working to isolate and sequence the gene for the
animal model of narcolepsy. While investigators continue work to localize the genetic defect in Huntington's disease (HD), research to determine the physiologic defect and possible therapies has progressed; preliminary efforts
are underway to test the efficacy of a drug called idebenone to protect at
risk brain cells. Transgenic nice are also being developed as a genetic
model for HD--an invaluable tool for the expected wave of new studies once
the gene is found.
An estimated 10 to 15 percent of all children have mild cognitive
deficits or learning disabilities. This year, an estimated 9,000 babies will
develop cerebral palsy and several thousand more babies and children will
have to live with--or die from--the neurological consequences of their
mother's drug abuse.
An ongoing multidisciplinary study to develop,
standardize, and validate a uniform classification system for the diagnosis
of children with higher cortical dysfunctions will facilitate research.
Improved treatments for epilepsy in the pediatric age group are a priority
for the Institute.
As evidenced by the advances and initiatives described here, we cannot separate progress in prevention, treatment, and diagnosis from the importance "memory centers" to release more neurotransmitter. Studies of single nerve cell activity are feeding into better understanding of brain circuits
of basic neuroscience research. Neurochemistry and neurobiology are
providing important leads to understand cognition and behavior.
grantees recently found that one possible mechanism for strengthening long
term neural interactions appears to be the ability of the sending cell in
activated by normal cognitive functions or affected by disease.
Brain imaging technology continues to push back the limits of our
ability to study the living brain. Experiments with positron-emission
tomography (PET) have linked discrete areas of the brain to word-processing
tasks and have confirmed the unique role PET has for understanding the higher
New PET tracer chemicals are opening the door to view
the working circuitry of the brain. MRI has been established as a
a critical tool for research on neuro-AIDS and multiple sclerosis (M$); new
ew studies will further elaborate understanding of how MS attacks the nervous system and will be very important in evaluating the effectiveness of treatments. The
application of several techniques to the same research problem is proving
very fruitful; PET, MRI, and magnetoencephalography (MEG) provide complementary information necessary to precisely diagnose and localize the
epileptic focug--especially critical when considering surgery. Ongoing research seeks to overcome technological barriers to better integrate data from PET and MRI. These exciting opportunities underscore the importance of
brain imaging centers in which these technologies could be integrated and
applied to brain research.
Exciting work with neural prostheses points the way to restoration of
function in the damaged nervous system.
A neural prosthesis that will
utilize extension movement in the wrist to control opening and closing of the
hand is under development.
A brain probe containing both electrodes and
cultured neurons is being developed to make very specific connections with the central nervous system. Scientists are also studying genes called into
action after traumatic injury to the nervous system for their potential to
promote nerve cell growth and regeneration.
The declaration of the 1990s as the Decade of the Brain has generated
new enthusiasm among neuroscientists, stimulated international collaborative
research efforts, and provided a focus for efforts to highlight and maintain
the U.S. lead in research, medicine, and biotechnology.
The promise of the
"Decade" has provided in its first year new treatments benefiting nany
Americans--treatments which in many cases have the added plus of being
inexpensive or cost-saving. The potential for further progress, as outlined
in the "NINDS Implementation Plan for the Decade of the Brain," impacts
particularly vulnerable segments of our population--elderly, children, and
minorities--and encompasses many of the major health care problems facing the
nation today, such as AIDS and drug abuse. Significant progress has been
achieved in the neurosciences in just the past few years.
We are optimistic
that in the year 2000, when we look back to see what has been accomplished in
the "Decade of the Brain", we will be pleased with the progress made in
understanding the human brain and significantly improving the quality of
Mr. Chairman, the FY 1992 budget request for this Institute is
I am pleased to answer any questions you might have.
BIOGRAPHICAL SKETCH OF DR. MURRAY GOLDSTEIN
October 13, 1925, New York, New York
EDUCATION: B.A., New York University, 1947; D.O., Des Moines Still College of Osteopathic Medicine, 1950; M.P.a., University of California, School of Public Health, 1959; Mayo Clinic (Neurology), 1968.
PROFESSIONAL HISTORY: Director, NINDS, 1982-present; Acting Director,
PROFESSIONAL ORGANIZATIONS: Aner ican Acadesy of Neurology: American
HONORS, AWARDS: Beta Alpha Epsilon, Biology National Bonor Society, NYU; Psi Chi, Psychology National Bonor Society, NYU; Sigma Alpha, Osteopathic Scholarship National Honor Society, College of Osteopathic Medicine; Delta
Omega, Public Health National Honor Society, University of California; Doctor
ACUTE SPINAL CORD INJURY
Senator HARKIN. Thank you very much, Dr. Goldstein.
I understand there are nerve cells that are damaged by trauma and repair themselves if you get to them with 8 hours, you say.
Dr. GOLDSTEIN. Yes, sir; in the spinal cord.
What do you do? What kind of a treatment is it? Let's say that someone has broken their neck or broken their back or something like that. And the spinal cord, obviously, has not been severed. It has been bruised.
Dr. GOLDSTEIN. That's right, sir.
Senator HARKIN. What do you do? What is the treatment? What do they do?
Dr. GOLDSTEIN. There is a drug that is commonly available on shelves in emergency rooms. It is not a new drug. It has been there for years. That drug in one large amount, an overwhelming amount-and that was the amazing thing about it is given to the patient intravenously immediately and then for 24 hours. That drug prevents the damaged nerve cell from dying. It gives it the opportunity to be protected from the deadly process killing it and then begin to repair itself.
The treatment, by the way, costs about $100, and it prevents a lifetime of severe disability, often being confined to a wheelchair. It is as simple as that.
The drug is so innocuous that if the physician thinks the patient may have a spinal cord injury and is not sure, it is well worth giving the drug. It costs about $100 and it does not have a deleterious side effect. It can have a tremendous positive effect.
Let me volunteer the next question, and that is will it work in the brain, a head injury. We don't believe it will.
CHRONIC DEGENERATIVE BRAIN DISEASES
Senator HARKIN. How about Parkinson's disease?
Senator HARKIN. I mean early intervention. Not that drug, but could there be early intervention for things like Parkinson's or Alzheimer's, things like this?
Dr. GOLDSTEIN. I think I understand your question
The scientific issue is shared with my colleague in the Aging Institute-and we work closely together on it. The focus of NINDS attention on Alzheimer's disease and Parkinson's diseases and other degenerative diseases of the brain, which we have singled out for priority attention, is not the patient who has advanced Parkinson's