Project Detail
Interagency Autism Coordinating Committee (IACC) logo
Office of Autism Research Coordination (OARC) logo

Development of mGluR5 antagonists to treat fragile X syndrome and autism  

Genetically engineered animal models have facilitated understanding of the effects of the mutations in the FMR1 gene that causes fragile X syndrome (FXS) on brain development and function. Studies with these animal models suggest that the symptoms of FXS reflect excessive protein synthesis downstream of mGluR5, a metabotropic glutamate receptor. Blocking this receptor with an antagonist could yield an effective drug treatment. This project will develop an existing lead compound, an mGluR5 antagonist, and advance it through the preclinical studies necessary to fulfill FDA requirements to open an Investigational New Drug application and perform initial testing in humans. These studies will test the hypothesis that mGluR5 antagonists can be effective treatments of FXS and other disorders of brain development, including autism. Project Status


Funder National Institutes of Health
Fiscal Year Funding $1,048,100.00
Project Number 5U01MH078270-03
Principal Investigator Carpenter, Randall
Received ARRA Funding? No
Strategic Plan Question Question 4: Which Treatments And Interventions Will Help? (Interventions)
Strategic Plan Objective 4O. Not specific to Question 4 objectives
Federal or Private? Federal
Institution Seaside Therapeutics, LLC
State/Country Massachusetts
Web Link 1 Development of mGluR5 antagonists to treat fragile X syndrome and autism (External web link)
Web Link 2 No URL available.
Web Link 3 No URL available.
History/Related Projects Development of MGLUR5 antagonists to treat fragile X syndrome and autism | $1,068,100.00 | 2008 | U01MH078270-02