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About Sickkids
About SickKids

May 6, 2014

SickKids and Holland Bloorview receive $2.5 million to test new drugs for autism

With over 250 genes implicated in autism, research so far has shown that no single gene is responsible for this neurodevelopmental disorder that affects one in 68 children in North America. While there have been huge advances in scientists’ understanding of the biology of autism, because of the heterogeneity in the disorder, it is difficult to find a standard treatment that would be effective for everyone.

On May 1, researchers from The Hospital for Sick Children (SickKids) and Holland Bloorview Kids Rehabilitation Hospital were awarded $2.5 million by the Government of Canada, in partnership with Brain Canada and the Azrieli Neurodevelopmental Research Program, to begin testing new drugs for the treatment of autism.

This research, led by Drs. Jason Lerch and Evdokia Anagnostou, will take innovative new drugs to clinical trial in mice, and simultaneously, in children and youth with autism. “Our hope is that these co-clinical trials in youth and mice will help us determine which treatments will work for which children, ultimately helping us create individual treatment plans for each patient,” says Lerch, Scientist in Neurosciences & Mental Health at SickKids.

The co-clinical trial approach will address the need to test promising new drugs, while at the same time determining which patients will respond to each therapy. This model of co-clinical trials has been successful in cancer therapeutics, an area with similar challenges.