Early Results from a Progeria Therapy Trial

Researchers here show data resulting from a therapy targeting the underlying cause of progeria. Accelerated aging conditions are extremely rare, but this is interesting to the rest of us because the same mechanisms that run wild in progeria sufferers apparently occur in a minor way during normal aging - so a cure for progeria might have some utility for the rest of us as well: "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, completed only six years after scientists identified the cause of Progeria, included significant improvements in weight gain, bone structure and, most importantly, the cardiovascular system ... Twenty-eight children from sixteen countries participated in the two-and-a-half year drug trial, representing 75 percent of known Progeria cases worldwide at the time the trial began. Of those, 26 are children with the classic form of Progeria. ... One in three children demonstrated a greater than 50 percent increase in annual rate of weight gain or switched from weight loss to weight gain, due to increased muscle and bone mass. ... On average, skeletal rigidity (which was highly abnormal at trial initiation) improved to normal levels after FTI treatment. ... Arterial stiffness, strongly associated with atherosclerosis in the general aging population, decreased by 35 percent. Vessel wall density also improved with treatment."

Link: http://www.eurekalert.org/pub_releases/2012-09/s-ftf092412.php


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