A class of anticancer drugs currently being evaluated in phase 3 clinical trials may also be an effective treatment for Hutchinson-Gilford progeria syndrome (HGPS), a fatal genetic disorder that causes premature aging. If upcoming studies in a HGPS mouse model validate the results of experiments in cultured cells in the laboratory, a clinical trial of these drugs in HGPS children may begin as early as next spring.
Brian Capell, a New York University medical student participating in the Howard Hughes Medical Institute/National Institutes of Health Research Scholars Program and the first author of the study, reported the findings in the August 29, 2005, issue of the Proceedings of the National Academy of Sciences. The HHMI-NIH Research Scholars Program gives outstanding medical and dental students the opportunity to conduct biomedical research under the direct mentorship of senior NIH research scientists.
Although HGPS is a rare disease that affects only one child in 4 million, the disease has received wide publicity due to its striking nature. Children born with HGPS appear normal, but they experience growth retardation and show symptoms of accelerated aging -- namely hair loss, skin wrinkling, and fat loss -- around the first year of age. Accelerated cardiovascular disease also ensues, which typically causes death around age 12.
Jennifer Donovan | EurekAlert!
One step closer to reality
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