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Anti-Cancer Drug Used To Successfully Treat Social Difficulties Associated With Autism In Mice

The study is limited in that there are many more genes associated with ASD other than Shank 3. PhotoUG/Shutterstock

Researchers have found that a drug already approved for use to treat cancer can reduce some of the social difficulties associated with autism spectrum disorder (ASD) in mice.

Published in Nature Neuroscience, the work hints at the possibility of using drugs to alleviate some of the main social symptoms displayed by those with ASD. Others, though, are warning that these are incredibly early days and that the applications to the majority of autistic people are very limited.   


“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, who coauthored the research.

Using mice missing a particular gene known as Shank 3, which is associated with changes in social preference often seen in those with ASD, the researchers tested a particular cancer drug known as romidepsin. The drug basically loosened the DNA wound around proteins in the nuclei of cells, allowing genes involved in neuronal signaling that were previously inaccessible to be transcribed.

This, in turn, influenced the social behavior of the mice. In fact, the researchers found that over 200 genes that were suppressed by the DNA being wound too tightly around the proteins suddenly started being expressed as normal.

Other researchers have urged caution about reading too much into this study, however. “Autism is a very diverse condition with many different genes playing a role so developing a mouse model to represent it is challenging,” explained Dr Georgina Warner, research manager at the autism charity Autistica, who was not involved in the study. “While mouse studies like this one can play an important role in research, findings can’t easily be applied to humans.”


In the case of this particular study, for example, the use of mice with the Shank 3 gene knocked out has very limited applications for humans. The authors themselves admit that those without this gene only represent between 0.5 and 2 percent of ASD cases, and for the vast majority who don’t, this study might not be particularly relevant at all.

“The study reports interesting findings about the drug romidepsin, but it’s far too early to say whether it would have any effect on autistic people’s social skills,” Dr Warner continued. “It’s also not clear that all autistic people would welcome a drug which aimed to improve their social ability, especially if it had other side effects.”


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