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Exciting Times in Translational Research

 Posted by Autism Speaks Chief Science Officer Geri Dawson, Ph.D

Robert Ring and I recently returned from three days at Roche headquarters, in Switzerland. There Roche, a global healthcare company, and the journal Nature Medicine hosted a translational neuroscience symposium: “Autism Spectrum Disorders: From Biological Understanding to Therapeutic Strategies.” Rob, as many of you know, is our vice president of translational research.

The meeting highlighted the remarkable progress that is being made. Researchers are discovering the molecular pathways involved in autism. They are identifying the biomarkers that will allow us to recognize “which” autism a person has. The latter is so important for tailoring new therapeutics to maximize benefit for particular subtypes of autism spectrum disorder (ASD). We also heard how investigators at Massachusetts Institute of Technology are developing the kind of high-throughput drug screening that will accelerate the development and delivery of safe and effective new medicines.

I had the honor to chair of one of the sessions – on the creation of clinical endpoints that will allow investigators and government regulators to assess the real-life benefits of medicines being developed for autism. Panelists in the session included Patrick Bolton, of King’s College London; Kevin Pelphrey, of Yale Child Study Center; Randall Carpenter, of Seaside Therapeutics; and John Constantino, of Washington University School of Medicine.

For me, the highlight of the meeting was an inspiring talk by Jacki Crawley, one of the lead developers of animal models for autism. She is currently at the National Institutes of Health, but is soon moving to the U.C. Davis MIND Institute, where many of our funded investigators are conducting autism research. Dr. Crawley discussed the work that she and Rob Ring conducted while Rob was still at the pharmaceutical company Pfizer. As readers of our science news column know, the exciting results of this study, based on a Pfizer compound, were published last week in Science Translational Medicine, along with a special editorial describing its importance. The study was also featured on the cover of this prestigious journal.

Here are some of the aspects of this study that I found particularly exciting: Jacki has a strain of mouse that shows the full autism profile – impaired social interaction, reduced vocalizations and highly repetitive behavior. This mouse is the closest model we have for autism of unknown cause (versus autism associated with a single-gene syndrome such as fragile X). What Rob and Jacki showed is that the Pfizer compound – a glutamate receptor antagonist – reduces both repetitive behavior and social deficits in this autism mouse model. 

Previously, this kind of “rescue” from multiple core symptoms had only been shown in mouse models of single-gene autism disorders such as fragile X. Their study shows proof-of-concept that such medicines may have wider applicability to the larger autism population. What is further inspiring is that this rescue occurs not only in young animals but also adults that already have fully developed autism-like behaviors.

Congratulations to Rob for a breakthrough study! 

Autism Speaks is currently funding other research studies involving glutamate. You can explore these and other research using our Grant Search.