Anti-Cancer Drug Mitigates Autism’s Social Deficits

By Marcia-Ruth Ndege ‘21

Figure 1. Autism is thought to affect one out of every 45 children under the age of 17.  

Autism Spectrum Disorder (ASD), better known as autism, is a developmental disorder characterized by a tendency to engage in repetitive behaviors and to have trouble communicating both verbally and nonverbally. While there is no known cure for autism, current treatments include a variety of therapies as well as antipsychotic drugs such as quetiapine and risperidone, which reduce psychiatric effects such as schizophrenia, irritability, and depression. None of these drugs, however, treat the social-interaction difficulties that are common among those who suffer from autism. Researchers at the University of Buffalo are hoping to use anticancer drugs to treat such social deficits. Zhen Yan, PhD, professor in the Department of Physiology and Biophysics in the Jacobs School of Medicine and Biomedical Sciences at UB, took on this study as an extension of her 2015 research, which linked the absence of the Shank 3 gene on chromosome 22 to social deficits in ASD.

Researchers used the anticancer agent romidepsin on mice that had similar genetic defects as humans with autism. Romidepsin is an FDA-approved anti-cancer drug that works by inhibiting histone deacetylases HDAC2, enzymes that cause DNA to wrap more tightly around histone proteins and thereby prevents crucial genes from being expressed, leading to social and behavioral changes in individuals with autism. Through the inhibition of HDAC2, the formerly suppressed genes could be expressed. For three days, researchers dosed mice lacking the Shank 3 gene with small amounts of romidepsin. Their data shows that the effects of the drug lasted for three weeks, which is equivalent to a few years in humans. Upon further research into the effects of the drug, Yan and her team concluded that over 200 genes that had not been expressed prior to treatment were now expressed. These promising results have led Yan to strive to investigate further therapeutic agents to treat the social difficulties that accompany ASD.



  1. L. Qin, Social deficits in Shank3-deficient mouse models of autism are rescued by histone deacetylase (HDAC) inhibition. Nature Neuroscience 21, 564-575 (2018). doi: 10.1038/s41593-018-0110-8.
  2. Image retrieved from:

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