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that direct a protein to fold into the three-dimensional shapes that we have been talking about so that it will be active.
In addition, of course, to this commitment to basic research, the National Institute of General Medical Sciences supports a very large share of the biomedical research training that is funded by NIX. This training prepares scientists to pursue research careers in a wide variety of areas. Hopefully, it will lead to further Dr. Elias J. Coreys.
In response to concerns about the need to train more scientists to do research in biotechnology, as you know, in 1989, we established a special biotechnology research training program. We expanded the program, with funds provided by the Senate, by about 60 percent in fiscal year 1991.
The Institute also sponsors the Minority Access to Research Careers Program and the Minority Biomedical Research Support Program, both of which aim to raise the number of under-represented minorities in science. The MARC program has recently extended its Honors Undergraduate Research Training Program to support students in the first 2 years of college, and plans are also being made to broaden the eligibility for MARC individual predoctoral fellowships to minorities
who are students at any of the colleges and universities throughout the United States.
The fiscal year 1992 budget request for the Institute is $833,180,000.
I would be very pleased to answer any questions you might have. The statement follows:
STATEMENT OF DR. RUTH KIRSCHSTEIN
I an honored once again to appear before this committee to present the
exceptional achievements of the grantees of the National Institute of General
Medical Sciences (NIGMS), which stimulates scientific progress through the
support of basic biomedical research.
As has been the case in almost every
year in which I have testified before you, in 1990 an NIGMS grantee has again
won the Nobel Prize.
The chemistry prize went to Dr. Elias J. Corey of
Harvard University for his many contributions to the field of synthetic chemistry. According to the Nobel committee, "It is probable that no other
chemist has developed such a comprehensive and varied assortment of methods
which, often showing the simplicity of genius, have become commonplace in the
NIGMS has supported Dr. Corey's work for the past 20 years, and he is
clearly at the pinnacle of his career.
But there are other scientists whose
research is supported by NIGMS - • and many more people who receive training for
research careers --who represent the Elias J. Coreys of the future.
One of these is Dr. Elizabeth Blackburn.
She has been an NIGMS grantee
since starting her independent research career 13 years ago.
In this time,
Dr. Blackburn has made major advances in understanding the structure and
function of chromosomes, the cellular components that carry each person's
Dr. Blackburn, who is a professor at the University of
California, San Francisco, studies the ends of chromosomes, called telomeres.
These structures protect the genetic information during the chromosome
duplication that precedes cell division.
She has determined how the telomeres
function, and has recently found evidence that these structures may play a
role in the development of cancer and in aging.
Dr. Stuart Schreiber of Harvard University, an NIGMS grantee since 1982,
has made several important findings in the past year related to drugs that act
on the immune system.
To study drugs that suppress the immune system, thus
helping to prevent rejection following organ transplantation, he devised a
synthetic chemical that contains the common structural elements of two
promising new drugs.
His research is expected to lead to more effective and
less toxic immunosuppressive agents, and to provide new insights into the
workings of the immune system.
Dr. Schreiber also discovered a series of small molecules that bind to
the human immunodeficiency virus (HIV), 'which causes AIDS.
prevent HIV from infecting T cells, without disrupting normal cell function.
He believes that these molecules are promising forerunners of an effective
inhibitor of AIDS infection.
The advances in structural chemistry represented
by this work should be applicable to fighting other diseases, as well.
Another "rising star" is Dr. James Wilson of the University of Michigan
He led one of two teams of scientists who, in test-tube
systems, inserted normal genes into cells taken from patients with cystic
Dr. Wilson showed that these inserted genes functioned correctly.
This finding, which came just one year after the cystic fibrosis gene was
discovered, may lead to treatment for this common, fatal genetic disease.
Dr. Wilson's research training, leading to the combined M.D.-Ph.D. degree, was
supported by the NIGMS Medical Scientist Training Program.
which currently supports 766 students in 29 different programs, has been
remarkably successful in training highly creative, dedicated, and productive
physician-scientists such as Dr. Wilson, who made this important finding just
six years after completing his research training.
At a point even earlier in her career is Dr. Lisa Matsuda, who recently
completed a postdoctoral fellowship in the intramural NIGMS Pharmacology
Research Associate Program. During her fellowship, Dr. Matsuda conducted much
of the research that recently culminated in the identification of the long
sought receptor for marijuana in brain cells.
This achievement opens many
exciting avenues for further research.
One of the first goals is to find out
why this receptor is present in the brain and what it normally does there.
The discovery of the marijuana receptor also offers hope of designing a drug, that will have the therapeutic benefits of marijuana --which is useful against
epilepsy, nausea, asthma, pain, and high blood pressure--without its
Preparing Scientists for Research Careers
In addition to its commitment to basic research, NIGMS supports a large
share of the research training that is funded by the National Institutes of
This training recognizes the interdisciplinary nature of
biomedical research today, and stresses approaches to biological problems that
cut across departmental lines.
Such experience prepares trainees to pursue
research careers in a wide variety of areas, including those within the
missions of virtually all the other NIH Institutes.
In fact, over half of the
trainees supported by NIGHS obtain research funding from other parts of NIH.
It is especially critical to train scientists who are qualified to do
research in biotechnology if..as pointed out by Congress and the National
Academy of Sciences--the United States is to maintain its position as a world
leader in this field.
In response to this concern, in 1989, NIGMS established
a special program to support biotechnology research training. The Institute
expanded this program substantially in fiscal year 1991, increasing the number
of individuals to be supported by about 60 percent.
NIGMS also sponsors two programs aimed at increasing the number of
underrepresented minorities in science.
These programs make the Institute the
primary focal point at NIH for minority bionedical research and research
In an August 1990 article in Science magazine, the Institute's
Minority Access to Research Careers (MARC) and Minority Biomedical Research
Support (MBRS) Programs were called "a major factor in stimulating the
research Interest" of minority students. Among these students are Robert
Turner, I MARC honors undergraduate research trainee at Lehman College of the
City University of New York, and Maria Elena Hernandez, a graduate student in
chemistry at the University of Texas at El Paso who is working with MBRS
support. Mr. Turner is studying hemoglobin structure, and plans to
concentrate on sickle cell research in graduate school.
Ms. Hernandez, whose
research focuses on the use of microscopic bubbles of fat, called liposones,
to dellver drugs to cancer cells, was selected as the outstanding graduate
student at her school for the 1988-89 acadeaic yoar.
In an effort to extend its impact, the MARC Program recently began
accepting supplemental applications from schools with existing honors
undergraduate research training programs.
These supplements will enable the
schools to expand their programs, which are now llaited to the support of
college juniors and seniors, to also support promising students in the first two years of college. Other plans are being made to broaden eligibility for
MARC individual predoctoral fellowships, which are currently available only to
students who graduate from the MARC honors undergraduate program.
Structural Biology and AIDS
While most NIGMS - supported basic research is not targeted to any specific
disease, the Institute does have a program in which the techniques of
structural biology are used to design drugs against AIDS.
is a flourishing field in which a molecule's physical structure is related to
An NIGMS - supported scientist working in this area, Dr. Irwin
Kuntz, Jr., of the University of California, San Francisco, recently developed
a computer program that uses the three-dimensional shape of a molecule, rather
than its chemical structure, to identify compounds that might bind to it.
This approach, a form of rational drug design, enabled Dr. Kuntz to locute a
compound that blocks the action of an enzyme essential to the survival of the
AIDS virus, HIV.
The compound, which is the common antipsychotic drug
haloperidol (sold under the trade name Haldol), is only effective against HIV
when used in doses that greatly exceed its lethal limit.
For this reason,
discovering the potential therapeutic advantages of haloperidol can only be
considered a starting point. Dr. Kuntz and his colleagues are now making
changes in the drug's structure that, hopefully, will preserve the desired
antiviral activity while lessening its toxicity.
In late 1990, two groups of NIGMS - supported scientists at Harvard and
Columbia Universities made another important advance in AIDS research.
Investigators determined the three-dimensional structure of the part of the
receptor protein of human cells through which HIV enters the body.
should greatly increase our understanding of how the virus works, as well as,
help to design new drugs to block AIDS infection.
Basic Research Advances Biotechnology
Among the direct beneficiaries of the basic biomedical research supported
by NIGMS is the biotechnology industry.
Such research has yielded genetic
techniques that could lead to new ways to inactivate viruses; insert modified
genes into cells; and even produce tomatoes that ripen slowly and can
withstand mechanical handling, while still retaining their flavor and color!