Sulforaphane treatment of autism spectrum disorder (ASD).
Level 2 - randomized trial
Individual randomized controlled trial
PubMed 25313065 · doi:10.1073/pnas.1416940111
What was done
In a double-blind, placebo-controlled randomized trial, 44 young males (aged 13–27) with moderate to severe autism spectrum disorder (ASD) received either daily oral sulforaphane (50–150 µmol; n = 29) derived from broccoli sprout extract or an indistinguishable placebo (n = 15) for 18 weeks, followed by a 4-week washout phase. Behavioral changes were evaluated using the Aberrant Behavior Checklist (ABC), Social Responsiveness Scale (SRS), and Clinical Global Impression Improvement Scale (CGI-I).
What was found
Baseline ABC and SRS scores were closely matched between groups. At 18 weeks, the placebo group experienced minimal change (<3.3%), whereas the sulforaphane group showed significant improvements: a 34% reduction in ABC scores (P < 0.001 vs. placebo) and a 17% reduction in SRS scores (P = 0.017 vs. placebo). CGI-I ratings showed significantly greater improvement in social interaction, abnormal behavior, and verbal communication with sulforaphane (P = 0.015 to 0.007). Following 4 weeks of discontinuation, total scores on all scales rose toward pretreatment levels.
Why it matters
This study provides proof-of-concept evidence that targeting pathways linked to ASD—such as oxidative stress and neuroinflammation—with a dietary phytochemical can produce measurable, reversible improvements in core autism symptoms.
Limits
The sample size was small (n = 44) and restricted entirely to adolescent and adult males with moderate to severe ASD, limiting generalizability to females, younger children, or milder phenotypes. The observed clinical improvements were transient, returning toward baseline upon treatment cessation. No biological or mechanistic markers were reported in the abstract to confirm target engagement.