Encouraging results in progeria trial

 Progeria Research Foundation

Encouraging results in progeria trial

In a paper published Sept. 24 in the Proceedings of the National Academy of Sciences a team of physicians led by Dr. Leslie Gordon, associate professor of pediatrics (research), reported significant benefits from a candidate drug for children with progeria, a rare and fatal disease in which children appear to age very rapidly. The prognosis for the disease is never good, with children dying of athlerosclerosis typically by age 13. In the new study, Gordon, medical director of the Progeria Research Foundation and mother of a boy with the disease, worked with Boston Children’s Hospital to try a cancer drug as a therapy in a clinical trial. Results in 28 children from 16 countries over the last two and a half years showed improvements in weight gain and bone structure and a decrease in arterial stiffness in a significant number of the kids. “To discover that some aspects of damage to the blood vessels in progeria can not only be slowed by the [drug] called Ionafarnib, but even partially reversed within just 2.5 years of treatment is a tremendous breakthrough,” Gordon said in a press release announcing the study.