A Merck drug first developed for cancer has shown promise as a treatment for Progeria, a rare and fatal rapid-aging disease in children. The results of the first-ever clinical drug trial for children with Progeria were released and demonstrate the efficacy of a farnesyltransferase inhibitor (FII) to treat the rare aging condition. 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, according to The Progeria Research Foundation (PRF) and Boston Children’s Hospital. The study results were published in the Proceedings of the National Academy of Sciences.
Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare, fatal genetic disease characterized by an appearance of acerbated 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 5 years years old, with the average age of death at 13 years. The condition affects approximately one in every 4 million to 8 million infants. There are only about 200 children living with the disease worldwide.
The PRF raised $2 million dollars that funded this ground-breaking clinical trial at Boston Children’s Hospital. The quick pace of research is in large part the result of Leslie and Scott Gordon — both physicians. Their son Sam suffers from Progeria — amazing what “parent entrepreneurs” can do in the lifetime of their children!
Read the official press release from the Progeria Research Foundation.
You can also read an insightful story about the Progeria drug news written by Amy Marcus of the Wall Street Journal.