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News and Publications
Year In Review - 2000
Ernest Beutler, M.D., Chairman
cience is incremental. While very few major discoveries are made in any single year, programs in the Department of Molecular and Experimental Medicine continue to make exceptional progress in advancing knowledge of biomedical science. This is not a clinical department, and although a number of its faculty members have significant clinical experience, relatively few continue to see patients. Nonetheless, among the departments at TSRI, this is closest to clinical medicine, and much of the work consists of translating basic laboratory work into clinical applications. The department also has responsibility for the General Clinical Research Center. This ongoing grant from the National Institutes of Health provides a clinical study unit within the Green Hospital, defraying the expenses of maintaining a staff, a core laboratory, and the ancillary costs of doing research. Today, the faculty consists of approximately 50 full-time scientists working with a total staff of 350.
One approach to improving medical care is to better understand important diseases, their natural history, and their response to treatment, thereby trying to devise new, more effective therapies with which to treat patients. Currently, members of the department are collaborating with Kaiser Permanente, one of the nation's largest health maintenance organizations, in a large study of the epidemiology of hemochromatosis, the major hereditary form of iron storage disease. Hemochromatosis is a metabolic disorder in which excess deposits of iron occur in the liver, pancreas, and other organs. Among other manifestations that may result are cirrhosis of the liver, diabetes and cardiovascular diseases. Already, a database of health information and DNA samples from nearly 30,000 patients have been assembled. Although it was originally thought that most people with the mutation that causes hemochromatosis were symptomatic and suffered a high mortality rate if untreated, the results of these studies to date show that very few manifest the disease. Most who are homozygous for this genetic mutation seem to enjoy a normal life span. This study may lead to rethinking the cost/benefit of screening normal populations for this disease and of the importance of its early treatment.
Another major program deals with hepatitis, a serious disease that affects millions of patients around the world. Under the leadership of Frank Chisari, M.D., a biomedical scientist who joined TSRI in 1973 as a postdoctoral fellow, the department has become a world leader in the study of hepatitis, especially of the body's immune response to the disease. Why are some patients able to rid themselves of the virus, while others continue to carry it and develop serious liver damage? Chisari and his team of researchers were successful in creating transgenic mice to study the body's immune response to the virus.
BROAD INTEREST IN CANCER AND ORGAN TRANSPLANTATION
The department also includes a wide-ranging research program on cancer. The laboratory of Peter Vogt, Ph.D., has been moving into several new areas, including developing innovative screening techniques aimed at cancer treatment. There has been a great deal of interest lately in studying the existing database of chemicals and antibody libraries for drugs that will help fight tumor cells more effectively. Vogt and his colleagues are engineering cancer cells to enable the detection of a response to any of tens of thousands of chemicals and antibodies available for study.
Organ transplantation is another important clinical approach to some forms of cancer as well as to kidney and heart disease. The studies of Drs. Daniel Salomon and Bruce Torbett have received substantial attention this year. In the field of xenotransplantation, the transplantation across species, pigs have been considered the most practical donors for man. The use of pig kidneys and hearts might allow many desperately ill patients who are waiting for a suitable human donor to receive a life-saving transplant. There are many hurdles to overcome before this could become a reality. Recently, however, there has been concern that a type of pig virus, a porcine retrovirus, might be transmitted to man. Salomon and Torbett recently demonstrated in studies published in the journal, Nature, that human cells can be infected by these viruses. At this point, further study is needed to determine whether or not this can produce tissue-to-tissue or even patient-to-patient infection.
The work on cancer also operates on an institutional level. ScrippsHealth, Scripps Clinic, and TSRI have worked together to create a new Scripps Cancer Center. The Center represents a close collaboration between clinical oncology and those TSRI scientists who conduct laboratory research. A goal of this program is to allow researchers to contribute more directly to the solutions they seek by moving useful laboratory techniques in cancer treatment quickly and seamlessly into the clinic through their close collaboration with the Center's clinical staff.
One of the long-term goals in the cancer center is to create a Good Manufacturing Practices facility to test drug candidates developed in-house. This will facilitate collaboration between the basic scientist and the clinician, making it possible to test new treatment modalities, while fulfilling government-mandated safety and regulatory requirements. An initiative to seek funding -- both public and private -- for this effort is under way.
Many members of the faculty of the department are world leaders in their field and their preeminence has been recognized by numerous awards and selection for service in important advisory bodies. The election of Bernard Babior, M.D., to the National Academy of Sciences brings the total number of members at TSRI to 13, three of whom are members of the Department of Molecular and Experimental Medicine.
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