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Autism medication trials have targeted noncore symptoms (e.g., irritability; NEJM JW Psychiatry Sep 1 1998). To investigate whether the phytochemical sulforaphane (derived from broccoli-sprout extract) would affect core autism symptoms, investigators conducted a double-blind, placebo-controlled study. In animal models, sulforaphane normalizes metabolic processes that are aberrant in autism (e.g., mitochondrial dysfunction and neuroinflammation). Sulforaphane also increases the heat-shock protein thought to account for parent-reported improvement in autistic behaviors during febrile illnesses.
Researchers randomized 43 boys (aged 13–27 years) who met diagnostic criteria for moderate-to-severe autism spectrum disorder (ASD) to daily dietary sulforaphane capsules (patent pending) or matched placebo for 18 weeks. Most parents (80%) had observed improvement in autistic behaviors during fever, which obviated analyzing febrile effects. On parents' ratings using validated behavioral measures, the core features of verbal communication and social and stereotypic behaviors improved significantly at 4, 10, and 18 weeks in treated boys compared with controls. However, clinician ratings of global autism severity did not differ between groups. Behavioral scores reverted to baseline levels 4 weeks after treatment ended. Sulforaphane was associated with weight gain (mean, 4 lbs).
Singh K et al. Sulforaphane treatment of autism spectrum disorder (ASD). Proc Natl Acad Sci U S A 2014 Oct 28; 111:15550. (http://dx.doi.org/10.1073/pnas.1416940111)
Comment
Although global ratings did not change, even modest increments in core symptoms are important in moderate-to-severe ASD. Clinicians will need to help parents with realistic expectations. Other drugs, such as bumetanide and suramin, had promising results but serious adverse effects (NEJM JW Psychiatry Dec 21 2012 and Jun 26 2014). In this short-term study, the only significant adverse effect was weight gain, which would require management to avoid development of metabolic syndrome.
Encouraging adequate broccoli intake by pregnant women could be important because autism likely begins prenatally (NEJM JW Psychiatry Jun 12 2014) and sulforaphane might reverse some metabolic effects of environmental pollutants, known to increase autism risks (Cancer Prev Res [Phila] 2014; 7:813).