Translation in psychopharmacology research: from animal model to clinical trials; the FMR1 story

To date, translational drug discovery for autism has been unproductive largely due to the lack of mechanistic understanding as well as the absence of predictive animal models. Translational research focused on single gene disorders, such as Fragile X Syndrome, is changing this paradigm through scientific exploration focused on identifying the fundamental pathophysiology and application of this knowledge to develop targeted therapeutics. This talk will demonstrate this point by using Fragile X as an example of the potential for translational research in ASD. Recent discoveries have revealed that a molecular pathway, the mGluR5 signaling cascade, is dysregulated in a specific disorder of brain development – Fragile X syndrome. With this knowledge, further research has provided insights for translating these discoveries into novel medications designed to normalize the function of this pathway.  This approach holds promise for developing disease modifying therapeutics that target the fundamental pathophysiology of autism