Experimental drug alleviated autism symptoms in mice

By Deborah Kotz, Globe Staff

While many potential drugs to treat autism are being tested in experimental settings, a new one appears fairly promising -- at least in mice. Researchers from the National Institutes of Mental Health and Pfizer reported Wednesday that an experimental compound, called GRN-529, increased social interactions and lessened repetitive self-grooming behavior in a strain of mice bred to display autism-like behaviors.

The mouse in the video above was interested only in repeatedly cleaning its coat of fur before it was given the drug; after getting the drug, it went to an attached cage to try to engage with a new mouse it had never encountered before.

Our findings suggest a strategy for developing a single treatment that could target multiple diagnostic symptoms, Jacqueline Crawley, a researcher at the mental health institute, said in a statement. Many cases of autism are caused by mutations in genes that control an ongoing process -- the formation and maturation of synapses, the connections between neurons. If defects in these connections are not hard-wired, the core symptoms of autism may be treatable with medications.

The study, published in the journal Science Translational Medicine, is one of several to test a class of drugs that inhibit a cell receptor called mGluR5, thats known to play a role in Fragile X, an autistic-like syndrome. Clinical trials to test these drugs on Fragile X patients are already underway.

Together, the new and previous findings suggest that clinical trials should be initiated to test the effects of mGluR5 inhibitors in autism patients, wrote Baltazar Gomez-Mancilla, a researcher for the drug company Novartis, in a paper that accompanied the study. But whether the agent would work in older children and adults with autism remains questionable at best. It could be, added Gomez-Mancilla, that the limited plasticity of the adult brain mandates intervention at earlier ages, ideally at the time of diagnosis.

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Experimental drug alleviated autism symptoms in mice

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