Sulforaphane treatment of children with autism spectrum disorder

PL1‐6. Sulforaphane Treatment of Children with Autism Spectrum Disorder (ASD) – A Progress Report  Singh K (Worcester, MA), Diggins E, Connors S, Zimmerman A.  Child Neurology Society Meeting, 2018

Objective: Direct treatment of underlying mechanisms in ASD is limited. The “fever effect” in ASD, where febrile illness temporarily ameliorates disordered behavior, may offer a clinical clue. Fever stimulates heat shock proteins (HSP), Nrf2 transcription and cell‐stress responses (CSR), leading to improved synaptic function and long‐range connectivity. Sulforaphane (SF), an isothiocyanate from broccoli sprouts, induces HSP, Nrf2 and CSR that may benefit ASD through common cellular mechanisms underlying heterogeneous phenotypes.

Methods: This is a phase‐2 clinical trial to test safety and efficacy of oral SF in 50 children (age 3‐12 years) with ASD. Treatment period is 30 weeks, with study‐visits at screening, 7, 15, 22, 30 and 36 weeks. The first 15 weeks are randomized, double‐blind, 1:1 placebo‐controlled, the second 15 weeks open‐label (all participants receiving SF), and last 6 weeks wash‐out. Ohio Autism Clinical Impressions Scale (OACIS), Aberrant Behavior Checklist and Social Responsiveness Scale are administered and samples are collected for safety and research studies at each visit.

Results: We have completed enrollment; 32 participants have completed the trial, 16 are actively enrolled, with 2 drop‐outs. A preliminary analysis of the OACIS showed: 23% participants were much/very much improved at 7 weeks, 31% at 15 weeks, 59% at 22 weeks, and 53% at 30 weeks (see Figure). Most adverse events so far are mild and transient [insomnia (28%), vomiting (19%), flatulence (17%), diarrhea (15%), and constipation (13%)]. Full results, including biomarker data, are expected after 07/2018.

Conclusions: Our preliminary results show that sulforaphane appears to be safe and effective in children with ASD.