New research from the University at Buffalo has shown that an approved anticancer drug can significantly restore the social deficits associated with autism spectrum disorder (ASD). At this stage the effect has only been demonstrated in animal models, but this promising research could pave the way for a therapeutic drug that helps restore social and communication skills in people with ASD.

The new study focuses on a gene called Shank3. Previous research has revealed strong connections between deficiencies in Shank3 and the irregular behavioral patterns associated with ASD. Shank3 has been found to be vital in neuronal communications and mice engineered with inactive Shank3 genes have displayed autism-like behaviors including compulsive repetitive behavior and anxiety.

The University at Buffalo study treated Shank3-deficient mice with a low-dose of romidepsin for just three days. Romidepsin is an FDA-approved anti-cancer drug that has been used to treat lymphomas for several years. After a short treatment the mice displayed significant social and behavioral improvements lasting up to three weeks. This duration of effect suggests a similar treatment in humans could be long-lasting, with three weeks in a mouse model equivalent to several years in humans.

"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," says Zhen Yan, a senior author on the study.

This is not the first piece of research demonstrating a cancer drug reactivating Shank3. In 2016 a team at MIT used a breast cancer drug called tamoxifen to similar effect in mice engineered with inactive Shank3 genes. This prior study also demonstrated positive alterations in the animal's behavior from a reduction in anxiety to improved motor skills.

A growing body of research is finding more and more curious genetic connections between autism and cancer. "The extensive overlap in risk genes for autism and cancer, many of which are chromatin remodeling factors, supports the idea of repurposing epigenetic drugs used in cancer treatment as targeted treatments for autism," says Yan.

The new study was published in the journal Nature Neuroscience.