Open Label Clinical Trial of Sulforaphane in School-Aged Children with Autism with Metabolomic Biomarkers

Friday, May 12, 2017: 5:00 PM-6:30 PM
Golden Gate Ballroom (Marriott Marquis Hotel)
S. Bent1, T. Warren2, F. Widjaja3, K. Dang1, J. W. Fahey4, J. Kinchen5, J. Buckthal6, B. S. Cornblatt7 and R. Hendren8, (1)University of California, San Francisco, San Francisco, CA, (2)Psychiatry, University of California, San Francisco, San Francisco, CA, (3)UCSF, San Francisco, CA, (4)Johns Hopkins University, Baltimore, MD, (5)Metabolon, Inc., Durham, NC, (6)Metabolon, Inc., Laguna Hills, CA, (7)Nutramax Laboratories Consumer Care, Inc., Edgewood, MD, (8)University of California San Francisco, San Francisco, CA

Children with ASD have been found to have elevated markers of oxidative stress and impaired antioxidant function compared to children without autism. A previous study has shown a significant improvement in ASD symptoms after supplementing children with autism with sulforaphane; known to cause transcriptional up regulation of genes that control oxidative stress. This study reports changes in behavior of children with autism following sulforaphane supplementation.


The objective of this study was to explore parent- and teacher-reported changes in behavior of children with ASD following sulforaphane supplementation. A second objective was to examine the metabolomic profiles of children with ASD who supplemented with sulforaphane, comparing responders and non-responders to their siblings who did not supplement, in order to look for biomarkers that might better indicate the mechanism by which sulforaphane acts in the body.


This was an open-label, 12-week study examining the effects of sulforaphane in children ages 5 to 22 years with clinically diagnosed autism. All subjects were enrolled at the Oak Hill School in San Anselmo, California, and had a diagnosis of ASD, or were siblings of enrolled students. The clinical diagnosis of autism was established by meeting DSM-IV criteria. The ASD group was given a dose of sulforaphane determined by weight, while siblings did not take the supplement. Behavioral assessments of children with ASD were collected using the Aberrant Behavior Checklist (ABC) and the Social Responsiveness Scale (SRS) at screening and close-out (week 12) from parents and teachers. Urine samples were collected from the ASD group and from their siblings, and metabolites were analyzed and compared.


Of 20 ASD subjects enrolled, five dropped out from the study because of the inability to ingest the sulforaphane tablets. Subjects with a decrease of four or more points on the ABC (n=8) were identified as responders to sulforaphane. Responders exhibited a 26.7-point decrease in total ABC (p<0.001) and an 18.6-point decrease in SRS (p=0.001) at close-out (Week 12), compared to non-significant increases of 2.9 points in ABC and 0.9 points in SRS for non-responders. Metabolomics of all subjects and siblings showed that responders had higher levels of sulforaphane-cysteine, sulforaphane, and sulforaphane N-acetyl-cysteine in their urine after supplementing than did non-responders. There were no adverse effects reported during the study and at the one-month follow-up after completion.


Sulforaphane was well tolerated and seems to improve ASD symptoms as measured by ABC and SRS. There may be a subgroup of children with ASD who show a strong response to sulforaphane, while others may not show a significant response based on metabolic profile. Metabolomic analysis confirms different metabolites found in urine of responders versus non-responders in this study, and would be a future direction to pursue in research on sulforaphane in children with ASD.