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Question. How many research project grants which focus on HIVrelated research specifically focused on mothers and children have been funded by each Institute? What is the total dollar amount for these grants by Institute?

Answer. Within the AIDS Clinical Trials Group (ACTG), 15 research grants and twelve supplementals, at a total cost of $16.2 million, were issued in FY 1990 on HIV-related research specifically targeted to children and/or mothers.

NIAID epidemiology studies in FY 1990 related to mothers and children included seven research project grants for $3,433,819; four contracts, totaling $7,059,487, for the Women and Infants Transmission Study; and an interagency agreement, the Maternal Factors Influencing Perinatal Transmission of HIV Study, also referred to as the Newark Perinatal Study, for $915,628.

The NICHD has a Branch--the Pediatric, Adolescent and Maternal AIDS Branch--which focuses all of its activities on research related to HIV infection in infants, children, pregnant women, mothers, adolescents, and families. The NICHD supports 26 research grants for a total of approximately $6.0 million which focus exclusively on biomedical and biobehavioral research issues in pediatric and maternal AIDS. The Institute also supports six research contracts and interagency agreements for approximately $16 million which focus on various aspects of HIV infection and disease in women and children. Lastly, the NICHD co-sponsors with the NIAID the Women and Infants Transmission Study (WITS), a contract-supported research study to which the NICHD contributes $945,000 annually.





Senator HARKIN. We will call Dr. Gorden from the Diabetes and Digestive Kidney Diseases Institute.

Senator REID. I only have 4 minutes' worth of questions.

Senator HARKIN. Dr. Alexander with Child Health and Human Development; Dr. Hoel, Acting Director for the Institute on Environmental Health Sciences; Dr. Schambra of the Fogarty International Center.

Dr. Gorden, we have your request for $658.5 million, which is about 6.6 percent more than last year. About $34 million, or almost 80 percent, of your increase is focused in the area of research project grants with funding for centers and training again almost flat, as I have been repeating here in every instance. In fact, the portion of your budget not devoted to research grants increases from-well, it's a 4.2-percent increase, and funding for research grants increases about 7.5 percent.

So, again, Dr. Gorden, welcome back to the subcommittee. We would be pleased to hear your statement. Please proceed.

Dr. GORDEN. Thank you, Mr. Chairman.

Last year on the occasion of our 40th anniversary, we reviewed major clinical accomplishments of the National Institute of Diabetes and Digestive and Kidney Diseases, including the treatment of end-stage renal disease with dialysis and transplantation, major accomplishments in the treatment of chronic hepatitis, and in transplantation for end-stage liver disease.

We reported on treatment strategies in diabetes and the discovery of the abnormal gene that causes cystic fibrosis.

In fiscal years 1991 and 1992, we plan a strong basic science program to build on these major achievements. Our scientists have demonstrated, in the laboratory, that the abnormal cystic fibrosis gene can be replaced in tissue culture cells and correct the disease.

The abnormal gene in a rare form of kidney disease has been discovered. This is the first time a specific genetic defect in a kidney disease that can lead to end-stage renal disease has been elucidated.

We continue to make progress in understanding the cause of insulin dependent diabetes and in developing strategies that may be applicable to its prevention. In noninsulin dependent diabetes, which has such a devastating effect on our minority populations, we continue to make progress in understanding the complex genetic causes of this disease. Incidentally, over one-half of individuals with noninsulin dependent diabetes are over 65 years of age.

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Our endocrine program continues to make important contributions to diseases such as osteoporosis and cancers of the thyroid and prostate gland.

Our hematology program has demonstrated the effectiveness of the biotechnology-engineered erythropoietin for the treatment of the anemia of end-stage renal disease and of AIDS.

Hydroxyurea has been shown to be important in the treatment of sickle cell disease.

Our digestive diseases program continues to pursue clues to the causes and cures of inflammatory bowel disease.


Our nutrition program has demonstrated the differences in ways individuals burn calories that they eat, a process fundamental to understanding how to prevent and treat obesity.

For this multifaceted program, Mr. Chairman, our budget request for fiscal year 1992 is $658,557,000.

I would be happy to answer any questions that I can. [The statement follows:]


I am very pleased to testify about the programs of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Last year, on the occasion of our 40th anniversary, we reviewed several major, long-term clinical achievements of the NIDDK. These included progress in kidney dialysis, liver and kidney transplantation, and recombinant DNA commercial production of insulin, human growth hormone, bovine growth hormone, and


These advances represent dramatic treatments for diabetes and liver and kidney diseases, as well as major innovations in American industry and food production. As we enter the last decade of this century, our mission continues to be the advancement of science to improve the health of the American people and to prevent disease. Our major programs encompass diabetes and other endocrine and metabolic diseases; digestive and nutritional disorders; and diseases of the kidney, urinary tract and blood. Underpinning targeted research on these serious health problems are crosscutting areas of science such as molecular understanding of the human genome.

One of the most important NIDDK crosscutting areas is genetics, as exemplified by our cystic fibrosis (CF) research program. Last year, reported a significant achievement in human genetics research: the discovery by NIDDK grantees of the gene and defective protein product that cause CF. Building upon these discoveries, NIDDK grantees have now taken other giant steps forward in CF research. Recently, they successfully established longterm cell culture systems, which can be manipulated to understand and interrelate more fully the structural and functional characteristics of CF. This past year, NIDDK grantees were able to correct CF in experimental cells in the laboratory--a significant milestone in CF research. Using a virus, they inserted a normal copy of the CF gene into pancreatic cells taken from CF patients. Remarkably, the gene-altered cells produced a normal protein product and attained normal chloride ion transport. These findings suggest that CF is an excellent candidate for emerging gene therapy techniques.

As we now move into the fifth decade of NIDDK research, I am pleased to report yet another landmark in molecular genetics. NIDDK grantees have identified the gene and mutations responsible for Alport's syndrome, an inherited disease of the kidney's filtering system. This discovery is

particularly significant because it is the first primary disease gene ever identified for a disease of the kidneys.

For the many inherited diseases within our mission, the NIDDK is in the forefront of research related to gene therapy. Recently, NIDDK grantees developed methods for targeting gene therapy to specific sites in animals--the liver, muscle, and arterial wall. This research is extremely important because many inborn metabolic diseases will require organ-specific gene therapy. In this regard, the Institute is encouraging studies of genetic expression and recombination; gene targeting; stem cells and growth factors; and gene transfer techniques.

The NIDDK is also committed to research aimed at preventing chronic diseases, and at halting or slowing their progression. For example, we are supporting a major, multicenter clinical trial to determine whether strict control of blood glucose levels can prevent or ameliorate the vascular complications of insulin-dependent diabetes. Another multicenter trial is assessing whether a dietary regimen, combined with blood pressure control, can prevent or slow the progression of chronic renal disease to end-stage renal disease.


Such efforts offer the hope of reducing the enormous suffering and costs of chronic diseases, which will escalate as our population ages.

This past year has been a particularly impressive one for research on diabetes, which affects over 11 million Americans at an estimated annual cost of $20 billion. The NIDDK program in diabetes is a comprehensive one, aimed at understanding the genetic and other causes of both the juvenile and adult forms of the disease; finding ways to combat the devastating complications; and alleviating the disproportionate impact diabetes has on our minority populations, including Blacks, Hispanics, and Native Americans and Hawaiians.

An exciting discovery in research on insulin-dependent diabetes is that a pancreatic protein previously known to be an early predictor of the disease is identical to an enzyme normally produced in large quantities in the brain. This knowledge should facilitate the development of a simple blood test to identify--years in advance of clinical disease--those children who are destined for autoimmune destruction of insulin-producing cells. Such knowledge will be valuable as more effective drugs are developed that might arrest the disease in its earliest stages. Research on restoring insulin

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