Dusty Miller, Ph.D., of the Fred Hutchinson Cancer Research Center in Seattle, has received the National Hemophilia Foundation's highest honor for advancing gene therapy as a potential treatment for hemophilia, a bleeding disorder that affects one in 10,000 American males.
Miller and three other leading scientists accepted the 1999 Dr. Murray Thelin Researcher of the Year Award Sunday during the annual meeting of the American Society of Gene Therapy in Denver, Colo.
Also honored for their contributions to gene therapy for hemophilia were R. Jude Samulski, Ph.D., of the University of North Carolina at Chapel Hill; Inder Verma, Ph.D., of the Salk Institute in San Diego; and Savio L.C. Woo, Ph.D., of Mount Sinai Medical Center in New York City.
The researchers were credited for their pioneering work in the years before gene therapy captured widespread imagination, says National Hemophilia Foundation board member Glenn Pierce, M.D., Ph.D.
"The remarkable progress made in this decade is a direct result of the gene-therapy research agendas these scientists began in the last decade," he says, referring to several promising early stage gene-therapy trials for hemophilia that began in the past year.
In gene therapy, researchers typically use viruses as vehicles to infect genetically defective cells with therapeutic packages of corrected DNA. The goal is to get the implanted DNA to function normally and thus cure or reverse disease.
Miller and colleagues created the retroviruses used in the first human gene-therapy trial in 1990 at the National Institutes of Health. The trial involved two children with severe combined immunodeficiency, which is caused by a genetic defect in the adenosine deaminase, or ADA, gene. Today, nearly a decade later, the immune-system T cells in one of the children are still expressing a healthy copy of the ADA gene.
This technology led to the development of the first commercially available retroviral gene-transfer kit, now used by researchers at academic medical centers and biotechnology firms worldwide. The technological relevance of this advancement, for which Miller won a prestigious R&D 100 Award (dubbed "the Nobel Prize of applied research") has been compared to the fax machine and the automated teller machine.
Miller studies potential targets for gene therapy, from bone marrow to muscle cells. For example, in mice he and his colleagues have demonstrated long-term expression of factor IX, a blood-clotting protein defective in people with hemophilia, by simply injecting a vector containing the factor IX gene into the bloodstream. Following injection, the vector finds its way to the liver, where the clotting protein is made.
A member of the Hutchinson Center's Human Biology and Basic Sciences divisions, Miller also is an affiliate professor in the Department of Pathology at the University of Washington. His work is funded by the National Institutes of Health and the National Hemophilia Foundation.
The NHF Researcher of the Year award is named in honor of the late Dr. Murray Thelin, a biochemist and person with severe hemophilia who helped develop clotting agents to treat this devastating bleeding disorder. NOTE: A black and white photo of Miller is available upon request.
The Fred Hutchinson Cancer Research Center is an independent, nonprofit research institution dedicated to the development and advancement of biomedical technology to eliminate cancer and other potentially fatal diseases. Recognized internationally for its pioneering work in bone-marrow transplantation, the Center's four scientific divisions collaborate to form a unique environment for conducting basic and applied science. The Hutchinson Center is the only National Cancer Institute-designated comprehensive cancer center in the Pacific Northwest. For more information, visit the Center's Web site at http://www.fhcrc.org.