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Autism treatment could be found through cancer drug

| | April 10, 2018

A low dose of a drug used to treat lymphoma may ease social problems in some forms of autism, a mouse study suggests.

Specifically, the drug might be beneficial for children with autism who carry mutations that affect chromatin, the coiled complex of DNA and protein.

The mice in the study lack part of SHANK3, a gene mutated in up to 2 percent of people with autism. The mice have several features reminiscent of autism, including anxiety, repetitive behaviors and social problems.

A low dose of the drug, romidepsin, alters chromatin and normalizes social behavior in young SHANK3 mutants for at least three weeks, says lead researcher Zhen Yan, professor of physiology and biophysics at the State University of New York in Buffalo.

Related article:  Understanding autism's broad spectrum can 'save' family relationships

Untreated SHANK3 mice show less of a preference for another mouse over an object, compared with controls; romidepsin normalizes this preference.

Romidepsin does not improve motor skills or ease anxiety in the mice, however. It also has a smaller and shorter-lived effect on social behavior in adult mice than in the juveniles. This finding suggests the drug might work only in children, Yan says.

The researchers are screening other compounds that alter histones in mouse models of autism. They hope to find a drug that works as well in adult animals as it does in juvenile.

Read full, original post: Cancer drug shows promise for treating some forms of autism

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