Research conducted by the University at Buffalo suggests that anti-cancer drug romidepsin could help improve social impairments associated with autism spectrum disorder (ASD).

Romidepsin restores gene expression and function using an epigenetic mechanism, thought to play a major role in ASD. Researchers believe that targeting certain genes could improve behavioural symptoms of ASD, for which there is currently no treatmet.

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In a study, mice deficient in the Shank  gene, an important risk factor for ASD, were given a low dose of romidepsin for three days. Researchers observed that the drug had ‘restored gene expression and alleviated social deficits’ in the animals.

This was sustained for three weeks, the juvenile to late adolescent period in mice. This is equivalent to several years in humans, suggesting the effects of a similar treatment could potentially be long-lasting.

“We have discovered a small molecule compound that shows a profound and prolonged effect on autism-like social deficits without obvious side-effects.”

University at Buffalo Jacobs School of Medicine and Biomedical Sciences, Physiology and Biophysics department professor Zhen Yan said: “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.”

The current study is based on Yan’s previous research that showed the loss of Shank 3 affects the functioning of the n-methyl-D-aspartate (NMDA) receptor, which hinders neuronal communications and leads to social preference deficits in those with ASD.

Many of the mutations of ASD are caused by chromatin remodelling factors. This is also the case for some cancer genes, meaning epigenetic drugs used in cancer treatment can be repurposed as targeted treatments for ASD.

Romidepsin is a histone deacetylase (HDAC) modifier that alters the histones that help to organise the DNA in the nucleus. It turned down the effects of HDAC2 to enable the normal expression of genes involved in neuronal signalling. The drug is approved by the US Food and Drug Administration for the treatment of lymphoma.