Autism’s Social Deficits Are Reversed by Epigenetic Anti-Cancer Drug In Animal Models

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. New research at the University at Buffalo (UB) in New York reveals the first evidence that it may be possible to use a single compound to alleviate the behavioral symptoms by targeting sets of genes involved in the disease. The research, published online on March 12, 2018 in Nature Neuroscience, 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 deficient in a gene called Shank 3, an important risk factor for ASD. This effect lasted for three weeks, spanning the juvenile to late adolescent period, 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. The Nature Neuroscience article is titled “Social Deficits in Shank3-Deficient Mouse Models of Autism Are Rescued by Histone Deacetylase (HDAC) Inhibition.” "We have discovered a small molecule compound that shows a profound and prolonged effect on autism-like social deficits without obvious side effects, while many currently used compounds for treating a variety of psychiatric diseases have failed to exhibit the therapeutic efficacy for this core symptom of autism," said Zhen Yan (photo), PhD, Professor in the Department of Physiology and Biophysics in the Jacobs School of Medicine and Biomedical Sciences at UB, and senior author on the paper.
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