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These inquiries, whether they come via telephone, e-mail, or handwritten letter, express a desire for information filled with hope and sometimes desperation: Do you offer clinical trials for my disease? What new therapies are available? Can you help me?
HHMI is not a medical center with a full roster of doctors and nurses. We don't see patients or manage clinical trials here at our headquarters in Chevy Chase, Maryland. Therefore, our responses may discourage those who turn to us for immediate, direct help. At our host institutions, our scientists focus primarily on the basic biological processes that underlie human diseases. They rely on worms, flies, mice, and other model systems to probe the functions of specific genes or to deconstruct complex biochemical pathways that are important to understanding human biology.
Yet the medical part of our name represents a critical part of our mission and our work. HHMI's mission is not only “the promotion of human knowledge within the field of the basic sciences (principally the field of medical research and education)” but also “the effective application thereof for the benefit of mankind.” Finding the effective application for our knowledge can take time. Indeed, it may take decades to make the definitive connection between the lab and our ultimate aim: benefiting humanity.
Some HHMI physician-scientists are actively engaged in translating the discoveries in their laboratories into new therapies or diagnostic tools. I was particularly struck by the medical impact of HHMI science during a recent gathering of investigators at HHMI headquarters. These periodic meetings—each of our 350-plus scientists attends one a year—are always exciting because we hear about new work and share ideas. This particular meeting focused on translational medicine and the talks covered the gamut of human disease—from coronary artery disease and cancer to Alzheimer's disease, tuberculosis, and HIV/AIDS.
For example, Helen Hobbs at the University of Texas Southwestern Medical Center at Dallas focuses her research on the genetic factors that influence an individual's blood cholesterol levels and risk for heart disease. As leader of the Dallas Heart Study, she and her colleagues are combining population-based research with the powerful tools of reverse genetics to tease out the genetic basis for complex traits. Already, Hobbs' research has generated an important hypothesis—that early intervention to lower “bad” LDL cholesterol can substantially reduce the risk of heart disease, even among individuals who have other significant risk factors.
Photo: Bruce Weller
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