Development of Targeted Therapeutics for Autism Spectrum Disorders

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

 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 demonstrat​ing​ 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 ​R​ett S​yndrome ​R​esearch ​T​rust​’s ​R​oadmap ​to a Cure for Rett Syndrome. ​