In the News: Mriganka Sur on the research origins of the first approved drug to treat Rett syndrome
On March 10 the FDA approved Trofinetide, a drug based on the protein IGF-1.
The MIT professor’s original research showing that IGF-1 could treat Rett was published in 2009. Rett syndrome is a devastating developmental disorder, principally occurring in girls, caused by mutations in the gene MECP2 that leads to severe cognitive, motor, and other symptoms..
The approval is also a dream come true for Mriganka Sur, Newton Professor of Neuroscience in The Picower Institute for Learning and Memory and the Department of Brain and Cognitive Sciences at MIT.
His lab’s preclinical discoveries in mice, particularly a highly influential paper published in 2009, provided the first demonstration that injecting IGF-1 or its peptide fragment could reverse the effects of reduced or altered MECP2. This provided a mechanism-based rationale for IGF-1 as a potential therapeutic intervention.
And Sur’s lab has never stopped studying Rett syndrome since.
..