An experimental drug that muffles the activity of neurons in the skin moderates heightened reactions to touch in six mouse models of autism, according to a study published today in Cell1. The drug also tempers anxiety and a few social difficulties in some of the mice.
The findings hint at a new strategy for treating autistic people who are highly reactive to touch. Unusual sensory responses are one of the core features of autism.
“It’s a hopeful, possible therapeutic avenue for treating specific features of autism,” says Lauren Orefice, assistant professor in molecular biology at Massachusetts General Hospital in Boston. Orefice worked on the project as a postdoctoral fellow in David Ginty’s lab at Harvard.
The results also suggest that sensory problems alter brain development and contribute to social difficulties. “This study is a technical tour de force,” says Mark Wallace, dean of the graduate school at Vanderbilt University in Nashville, Tennessee, who was not involved in the study. “Sensory symptoms are likely playing a bigger role in autism than we would have thought.”
The drug, isoguvacine, is an experimental compound yet to be tested in people. It works in a similar way to some drugs used to alleviate anxiety, and to other experimental compounds being tested for autism. Unlike those drugs, isoguvacine cannot enter the brain and be sedative — and therefore may be safer to use in children.
However, although about 70 percent of autistic people have atypical responses to sensory stimuli, not all are unusually responsive to touch. That means drugs such as isoguvacine are unlikely to work for everyone.
“The key next step is to define which individuals with autism may most exhibit this [reactivity], and then focus on these individuals first in human trials,” says Craig Erickson, associate professor of psychiatry and behavioral neuroscience at Cincinnati Children’s Hospital Medical Center in Ohio, who was not involved in the work.