Hope for Autism

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Can the behavioral symptoms be reversed by a drug approved for cancer?

Of all the challenges that come with a diagnosis of autism spectrum disorder (ASD), the social difficulties are among the most devastating. Currently, there is no treatment for this primary symptom of ASD. But new research at the University at Buffalo reveals the first evidence that such a treatment may be possible.

The study demonstrated that brief treatment with a very low dose of romidepsin, an FDA-approved anti-cancer drug, restored social deficits in animal models of autism in a sustained fashion.

The three-day treatment reversed social deficits in mice lacking a gene called Shank 3, an important risk factor for ASD. This effect lasted for three weeks, spanning the juvenile to late adolescent period in the animal, a critical developmental stage for social and communication skills. That is equivalent to several years in humans, suggesting the effects of a similar treatment could potentially be long-lasting, the researchers say.

Therapeutic effect

“We have discovered a small molecule compound that shows a profound and prolonged effect on autism-like social deficits without obvious side effects,” says lead author Zhen Yan, SUNY Distinguished Professor in the Department of Physiology and Biophysics in the Jacobs School of Medicine and Biomedical Sciences at UB. She adds that many currently used compounds for treating psychiatric diseases have not displayed similar therapeutic efficacy for this core symptom of autism.

Previous research by Yan’s team showed how the loss of Shank 3 disrupts communication between nerve cells. The current study reveals how a very low dose of romidepsin, which works by switching certain genes on, restores the expression of many important genes in the autism model.

“The extensive overlap in risk genes for autism and cancer,” Yan says, “supports the idea of repurposing certain drugs used in cancer treatment as targeted treatments for autism.”