NATIONAL INSTITUTE OF ARTHRITIS, DIABETES, AND DIGESTIVE AND KIDNEY DISEASES STATEMENT OF DR. G. DONALD WHEDON, DIRECTOR ACCOMPANIED BY: DR. DONALD S. FREDRICKSON, DIRECTOR, NATIONAL INSTITUTES OF HEALTH L. EARL LAURENCE, EXECUTIVE OFFICER DONALD F. CYPHERS, FINANCIAL MANAGEMENT OFFICER NORMAN D. MANSFIELD, DIRECTOR, DIVISION OF FINANCIAL MAN BUDGET DEPUTY ASSISTANT PREPARED STATEMENT SECRETARY, Senator SCHMITT. We move to the next witness, who is Dr. G. Donald Whedon, Director of the National Institute of Arthritis, Diabetes, and Digestive and Kidney Diseases. This Institute is one of the most complex of the Institutes, as we heard earlier. It is responsible for 10 major research areas. Its research encompasses most of the serious chronic diseases being studied at present. Doctor, we welcome you. We ask you to introduce your colleagues and summarize your statement. Your prepared statement will be included in the record in its entirety. [The statement follows:] STATEMENT OF DR. G. DONALD WHEDON I appreciate the opportunity to report on the research programs of the National Institute of Arthritis, Diabetes, and Digestive and Kidney Diseases (NIADDK). During 1980 the Institute began its fourth decade of serving the American people through the conduct and support of biomedical research. The programs of NIADDK deal with a wide range of chronic diseases, as exemplified by the recent passage of legislation (P.L. 96-538), which expanded the name of the Institute to the National Institute of Arthritis, Diabetes, and Digestive and Kidney Diseases. I would like to describe some of the ways in which this Institute is meeting its responsibilities to the American people and to give you a brief overview of research planned for the coming year. Arthritis and Related Diseases Research advances are developing rapidly in the study of arthritis and rheumatic diseases. Recent research has shown that systemic lupus erythematosus, a potentially fatal connective tissue disease, tends to run in families, involves abnormalities of the immune system, and that certain blood cells are defective in lupus patients. New findings also have established the influence of sex hormones in lupus. Penicillin therapy has been shown to alleviate the initial skin rash characteristic of Lyme disease (an infectious form of arthritis) and may prevent or lessen the severity of the arthritis that develops later. In patients with scleroderma, life saving drug treatment has been successful in improving kidney function, lowering blood pressure, reducing ulcers on the fingers and even softening of hardened skin, problems characteristic of the disease. New hope for an effective treatment for the debilitating and incurable skin disease, epidermolysis bullosa, has been reported. Young patients with this uncommon inherited disorder experience severe scarring, deformities, and malnutrition. Phenytoin, an anticonvulsant drug, was found to reduce significantly the blistering of skin and mucous membranes that characterizes this rare skin disease, a major advance for its victims. Diabetes and Other Metabolic Diseases NIADDK will continue to emphasize diabetes research. During the past year, Institute-supported investigators successfully transplanted the insulin-producing cells of the pancreas across animal species without immune rejection, a major advance in the continuing search for better methods of treating diabetics whose lives depend on insulin injections. We will capitalize on the progress that has been made in the development of insulin infusion systems ("artificial pancreas") to normalize elevated blood sugar levels in humans and animals. Other promising research is taking advantage of the significant advances that have been made in recombinant DNA biology. Basic research by NIADDK grantees using recombinant DNA techniques has laid the foundation for the bacterial production of human insulin. Research on the bacterial production of other peptide hormones is receiving special emphasis, following the completion of the Congressionallymandated evaluation of research needs in endocrinology and metabolic diseases, the "Ingbar Report." The Institute plans to undertake clinical trials of human growth hormone produced in bacteria by recombinant DNA techniques. This research represents a major advance in overcoming hypopituitary dwarfism, since previously the only source of human growth hormone has been that extracted from human pituitaries collected at autopsy. Research on cystic fibrosis (CF) represents a special focus for the Institute. It is the most common lethal inborn error of metabolism among Caucasian children. Institute grantees have recently reported the development of tests that may make it possible to identify people who carry the gene for CF and to predict the condition before birth. Digestive Diseases NIADDK-supported research has demonstrated successfully that "monooctanoin"--an emulsifying agent derived from digested fat--is an excellent solvent for cholesterol gallstones. This effective and inexpensive agent appears to be a satisfactory treatment for removing gallstones that remain in patients after biliary tract operations. Over the past 20 years, the detection of disease responsible for life threatening gastrointestinal bleeding has been improved by the introduction of endoscopy--the use of flexible, fiberoptic instruments to view the inside of the upper portion of the GI tract. NIADDK grantees are studying the control of GI bleeding by endoscopic systems involving laser coagulation, electrocoagulation, suture clips, and tissue glues. They have found that these techniques offer new hope for the safe and effective control of massive bleeding. Kidney and Blood Disease NIADDK research efforts in kidney disease are focusing on the development of new methods of preventive therapy, earlier diagnosis, and more effective treatment through understanding of the basic mechanisms and causes. Such efforts provide hope that the numbers of patients dependent on artificial substitutes for normal kidney function will be reduced in the future. NIADDK research has recently shown that the success of cadaver kidney transplants increases in proportion to the number of blood transfusions given before transplantation. Patients receiving many transfusions, surprisingly, had much better graft survival than nontransfused recipients. Accordingly, investigators recommend more liberal transfusions and frequent antibody screening for transplant candidates in order to ensure the most effective cadaver kidney transplants. The Institute will continue its support of research into basic mechanisms of normal blood cell function and blood diseases. For example, NIADDK-supported research has led to the development of a highly safe and sensitive method for prenatal diagnosis of sickle cell disease, proven useful in 70 percent of pregnancies at risk for this potentially fatal blood disorder. A major advantage of this method is that it does not require a fetal blood sample, but rather tests amniotic fluid surrounding the fetus. This advance has stimulated activity in the research community in the effort to duplicate this type of testing in a variety of genetic diseases. Intramural Research The Institute's intramural scientists continue outstanding research in basic and clinical sciences. Our staff scientists have made major contributions in arthritis, diabetes and many other areas of our research responsibility and have attained international renown. Institute scientists in the Diabetes Branch have found evidence that nearly every cell in the human body makes insulin--not just the islet cells of the pancreas, as was previously thought. New studies now suggest that cells may regularly produce all the proteins encoded in their genetic material--but at a low rate--throughout their lives. In the area of genetics, NIADDK scientists have provided the foundation for understanding inborn errors of metabolism, which can cause physical deformities, mental retardation, and a shortened life span. Knowledge gained from these studies is now routinely used to provide diagnosis and prenatal and genetic counseling to families afflicted with these disorders. A study of glucose tolerance and its relationship to pregnancy in the Pima Indians, in whom diabetes is 10 times more prevalent than the national average, has resulted in description of the risks associated with various degrees of glucose intolerance before, during and after pregnancy. Mr. Chairman and Members of the Committee, this concludes my statement. I shall be glad to answer questions. |