Speaker: Randall L. Carpenter, M.D.
Affiliation: Chief Scientific Officer, Rett Syndrome Research Trust; Research Affiliate, Picower Institute for Learning and Memory
Date: March 21, 2018
Talk title: Development of Targeted Therapeutics for Autism Spectrum Disorders
Abstract: Twelve years ago, I co-founded Seaside Therapeutics with an MIT professor based upon a strong sense of optimism that substantial progress would soon be realized in treatment of neurodevelopmental disorders. A new era of molecular genetics and neurobiology suggested that genetic diagnoses need not equate to permanent mental disability, but can actually provide insights toward disease-altering treatments. A number of highly penetrant genetic mutations had been discovered and reproduced in animal models. novel therapeutics targeting the underlying pathophysiology were being validated in these animal models and preliminary human trials were demonstrating promising efficacy. However, subsequent larger definitive trials failed to demonstrate convincing therapeutic benefit. This lecture will address how these failures are informing therapeutic discovery and development with a particular focus on the Rett Syndrome Research Trust’s Roadmap to a Cure for Rett Syndrome.