First-Ever Treatment for Hutchinson-Gilford Progeria

[TECH NEWS]

Hutchinson-Gilford Progeria Syndrome (HGPS, Progeria), is a rare, fatal, “rapid-aging” childhood disease that is linked to the normal aging process. Results of the first-ever clinical drug trial for children with the disease demonstrate the efficacy of a treatment using a farnesyltransferase inhibitor (FTI), a drug originally developed to treat cancer. The clinical trial results, completed only six years after scientists identified the (genetic) cause of Progeria, included significant improvements in weight gain, bone structure and, most importantly, the cardiovascular system, according to The Progeria Research Foundation (PRF) and Boston Children’s Hospital. The study results were published Sep. 24 in Proceedings of the National Academy of Sciences (Epub ahead of print). Researchers plan to continue studying the effect of FTIs, which may help scientists learn more about cardiovascular disease that affects millions, as well as the normal aging process.

September 24, 2012, Spectrum / Eurekalert (Progeria Research Foundation)