Autism-Like Social Deficits Reversed by Epigenetic Drug

A chromatin-loosening drug already approved as a cancer therapeutic may have another application—reversing the social deficits associated with autism spectrum disorder (ASD). If chromatin is packed too tightly, it can entomb genes, preventing their expression by closing them off from the cell's transcriptional machinery.

Effectively disinterring genes, and thereby allowing them to be expressed again, is a strategy that has been pursued in the development of anticancer drugs such as romidepsin, which potently inhibits histone deacetylases (HDACs), histone modifiers that remodel chromatin and regulate the transcription of targeted genes.

It happens that the genes affected by romidepsin have roles in both cancer and autism, say scientists based at the University of Buffalo. According to these scientists, brief treatment with a very low dose of romidepsin restored social deficits in animal models of autism in a sustained fashion.

This effect lasted for three weeks, spanning the juvenile ...