June 23 2014
Rett syndrome drug shows promise in clinical trial
Rett syndrome, a rare genetic disorder that causes intellectual disabilities, autism, and physical deformities, has no cure. However, a small clinical trial has found that a growth factor known as IGF1 can help treat some symptoms of the disease.
Children who received the drug for four weeks showed improvements in mood and anxiety, as well as easier breathing, in a trial led by researchers at Boston Children's Hospital. MIT scientists first identified IGF1 as a possible treatment for Rett syndrome in 2009.
"This trial shows that IGF1 is safe in the cohort of 12 kids, and at least on certain measures, it provides some effectiveness," says Mriganka Sur, the Paul E. and Lilah Newton Professor of Neuroscience at MIT and an author of the paper, which appeared recently in the Proceedings of the National Academy of Sciences.