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Clinical Trial Tests Possible Treatment for Autism

September 02, 2013

Sunday's Boston Globe reports on a clinical trial at Boston Children’s Hospital which is testing the drug rapamycin in children with the genetic condition tuberous sclerosis complex (TSC). Nearly half all people with TSC develop autism. If the trial shows that rapamycin relieves symptoms of autism in people with TSC, it is possible that it could be effective for others on the autism spectrum as well.  

"Researchers frequently use diseases that involve simple, clear gene mutations, like the ones in tuberous sclerosis, to better understand more complex conditions such as autism that involve hundreds of genes. Because so many genes underlie the disorder, 'one pessimistic point of view would be that you’d need 500 different drugs to help individuals with autism,' said Dr. Mustafa Sahin, who is leading the trial at Boston Children’s. 'But maybe, these 500 genes converge on certain common pathways and they can be rescued by common drugs.”

The study is partially funded by Autism Speaks and the Globe story quotes Daniel Smith, senior director of discovery neuroscience for Autism Speaks.