Published January 2017
Molecular Autism

A first-of-its-kind study by autism researchers at Cincinnati Children’s has found that a drug used since the 1950s to treat tuberculosis can improve and maintain the social, emotional and interaction skills of high-functioning children with autism spectrum disorder (ASD).

“Even with a dose that’s one-tenth of the dose used to treat tuberculosis, we found that d-Cycloserine (DCS) is a well-tolerated drug that has the promise to boost durability of social skills training in ASD children,” says Craig Erickson, MD. “The children who were treated with DCS did significantly better, and the continued improvement is very marked.”

The study, conducted in conjunction with experts at Indiana University, involved 60 children with ASD, ages 5-11, with IQs above 70, and significant social impairment. All underwent social skills training involving greetings, emotions, creative play and social conversations. Pre-training, half received a 50-milligram dose of DCS, the other half a placebo.

Children also engaged in an eye-tracking program featuring images of human faces. ASD children typically look less at people’s eyes and faces; those who do tend to show greater social interest. At weeks 11 and 22, social skills were assessed by parental social responsiveness scale (SRS) ratings.

Compared to the placebo group, SRS scores for the DCS treatment group decreased and remained low at week 22, indicating less social impairment. DCS-treated children also spent more time looking at faces in the eye-tracking test.

Erickson and co-researcher Logan Wink, MD, call the results “a potential paradigm shifter.” The drug offers a minimal-risk option with significant potential for helping children with ASD “where there are no approved treatments for the core social skills deficits associated with the disorder.”