First approved drug for Rett Syndrome orignates from MIT Professor’s Lab

Findings from Professor Mriganka Sur’s lab lead to first FDA approved drug to treat Rett Syndrome

On March 10, Acadia Pharmaceuticals’ drug for treating Rett Syndrome was approved, the first ever for the condition. The basic scientific research for the drug originated in Professor Mriganka Sur’s lab in the MIT’s Brain and Cognitive Sciences Department.

Rett syndrome is a neurodevelopmental disorder that deprives one of the ability to communicate verbally and nonverbally. It is caused by mutations in the gene MECP2 and manifests itself in loss of cognitive and motor abilities, including speech and communicative hand movement. 

According to an interview with MIT News, Sur’s lab was able to “understand the molecules underlying Rett syndrome’s prolonged development and plasticity,” by using a “large-scale, unbiased screen.” They found that injecting the protein IGF-1 in mice models led to improved symptoms and longer life expectancy. 

Sur’s lab paved the way for a therapeutic to be developed by establishing the IGF-1 mechanism of action in Rett syndrome. After multiple rounds of clinical trials, Acadia Pharmaceuticals’ drug, trofinetide, was approved, which is based on IGF-1. 

Sur emphasized that “there is certainly still much left to learn” and that his lab has “never stopped working on Rett Syndrome.” Regardless, Sur said that his lab’s work culminating in clinical impact was a “dream come true.”