Results of the first-ever clinical drug trial for children with progeria, a rare, fatal "rapid-aging" disease, demonstrate the efficacy of a farnesyltransferase inhibitor (FTI), a drug originally developed to treat cancer. The clinical trial results, achieved 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 online in an open article on Septermber 24, 2012 in PNAS. Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare, fatal genetic disease characterized by an appearance of accelerated aging in children. All children with progeria die of the same heart disease that affects millions of normal aging adults (atherosclerosis), but instead of occurring at 60 or 70 years of age, these children may suffer heart attacks and strokes as early as age 5 years, with the average age of death at 13 years. "To discover that some aspects of damage to the blood vessels in progeria can not only be slowed by the FTI called lonafarnib, but even partially reversed within just 2.5 years of treatment is a tremendous breakthrough, because cardiovascular disease is the ultimate cause of death in children with progeria," said Leslie Gordon, M.D., Ph.D., lead author of the study, medical director for the PRF, and mother of a child with progeria. In addition, Dr. Gordon is a staff scientist at Boston Children's Hospital and Harvard Medical School, and an associate professor at Hasbro Children's Hospital and Alpert Medical School of Brown University.
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