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Randomized controlled trial of sulforaphane and metabolite discovery in children with Autism Spectrum Disorder

Andrew W Zimmerman, Kanwaljit Singh, Susan L Connors, Hua Liu, Anita A Panjwani, Li-Ching Lee, Eileen Diggins, Ann Foley, Stepan Melnyk, Indrapal N Singh, S Jill James, Richard E Frye, Jed W Fahey
Molecular Autism 2021 12(1):38

Bibliography

PubMed
PMID 34034808
PubMed Central
PMC8146218
DOI
10.1186/s13229-021-00447-5
Trial registry
View on ClinicalTrials.gov Registered trial — results not yet published in a peer-reviewed journal.
Funding
Funded by the U.S. Department of Defense, U.S. Army Medical Research and Materiel Command, AR140087 (W81XWH-15-1-0156). Avmacol tablets were provided by Nutramax Laboratories; the published correction clarified the commercial product used.
Competing interests
Andrew W. Zimmerman reported paid legal testimony related to pediatric neurology/autism and was named on a Johns Hopkins patent for sulforaphane in autism. Jed W. Fahey reported serving as a scientific advisor to Brassica Protection Products LLC, which supplies glucoraphanin-rich broccoli seed extract to the supplement industry.

Study snapshot

DesignRandomized parallel double-blind placebo-controlled trial with open-label extension
ModelChildren aged 3-12 years with autism spectrum disorder
Sample57 randomized; 45 analyzed in the initial double-blind phase
InterventionOral sulforaphane versus placebo for 15 weeks, followed by 15 weeks open-label sulforaphane and a 6-week no-treatment extension.
Duration36 weeks total; 15-week randomized double-blind phase
EndpointsOhio Autism Clinical Impressions Scale; Aberrant Behavior Checklist; Social Responsiveness Scale-2; Glutathione redox biomarkers; Mitochondrial respiration; Inflammatory markers; Heat shock proteins; Safety

What the study showed, in plain terms

The randomized phase did not significantly improve the primary overall clinical outcome, although one caregiver-rated secondary scale and several biological markers showed signals. The study illustrates why autism evidence should be described as mixed rather than uniformly positive.

Key findings

Primary OACIS effects were not statistically significant in the randomized phase. Caregiver ABC scores improved at 15 weeks, SRS-2 did not, and several biomarker changes were reported.

What this study can and cannot tell us

Small analyzed sample, attrition, multiple secondary endpoints, and an open-label extension vulnerable to placebo and expectation effects.

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Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology