There are currently no approved medications for improving core symptoms of individuals with autism spectrum disorder (ASD), including difficulty socializing and communicating. In a study published online on July 6 in JAMA Psychiatry, Eric Hollander, M.D., and colleagues report the results of a placebo-controlled clinical trial evaluating the drug balovaptan for treating social communication in children and adolescents with ASD. Balovaptan blocks the hormone vasopressin and was designed to modulate the oxytocin-vasopressin system. Both hormones compete for the same receptors in the brain and help to strengthen social bonds, but oxytocin is considered more helpful; balovaptan’s developers hoped that blocking vasopressin would increase oxytocin’s ability to help people with ASD become more socially engaged.
In the randomized, double-blind phase 2 study—the largest ever conducted in pediatric autism to date—339 children with ASD were assigned to three treatment groups: 4 mg of balovaptan, 10 mg of balovaptan, and placebo. After 24 weeks, a standardized measure of social communication indicated there were no statistically significant differences among the groups. The researchers note that the high placebo response in clinical trials of ASD treatments is a challenge to drug development and that normalizing nonexistent skills such as social communication, is more difficult than reducing other problematic ASD behaviors such as irritability.
Dr. Hollander is professor of psychiatry and behavioral sciences at Einstein and director of the Autism and Obsessive Compulsive Spectrum Program at Montefiore Health System and Einstein.
Posted on: Thursday, July 07, 2022