Glutathione, Vitamin C and Cysteine Use in Children with Autism and Severe Behavior Concerns: A Double Blind, Placebo-Controlled Crossover Study

Background:  Autism is a complex neurodevelopmental disorder characterized by severe social communication deficits and limited range of interests and activities.  While the specific etiologies of autism are unknown, one of the proposed mechanisms leading to autism has been oxidative stress.  Glutathione is a tripeptide which protects against oxidative stress and which has been found to be decreased in children with autism spectrum disorder (ASD).   Anecdotal reports have suggested that glutathione supplements may result in improved behavior in children with ASD and severe behavior disorder.

Objectives:  To determine the efficacy of 1) glutathione, 2) glutathione, vitamin C and N-acetylcysteine, or 3) placebo in remediating severe behavior concerns in children with ASD.

Methods:  Twenty-four children with ASD between the ages of 5 and 16 were randomized to receive intrvenous 1) glutathione, 2) glutathione, vitamin C and N-acetylcysteine or 3) placebo over an 8 week period in this double-blind, placebo-controlled study.  After a one week washout period, they received the alternate regimen for the subsequent 8 weeks.  Pre and post behavioral measures and glutathione blood levels were obtained.

Results:  Clinically significant improvement in overall Aberrant Behavior Checklist (ABC) scores and Repetitive Behaviora Scales (RBS) were noted for the Trio (2) group as compared to the placebo and glutathione groups.  No group effects was observed for the other four behavioral instuments.  No significant adverse side effects were reported during the study. 

Conclusions: This pilot study suggests that the combination of glutathione, vitamin C and N-acetylcysteine may result in behavioral benefits and decreased repetitive behaviors in children with ASD and severe behavior disorder.  Additional large scale randomized controlled studies are needed to verify these results.