Autism spectrum disorder (ASD) affects over 1% of children, can have many causes, and is an enormous societal and economic problem for which there is no established treatment based on an understanding of the underlying body chemistry. Sulforaphane is a natural chemical in broccoli that has potent activity in regulating genes that control self-protective mechanisms in the body's cells and act against damaging effects from inflammation and oxidative stress. Based on frequent observations that fever can lead to improved behavior in children with ASD, we hypothesized that sulforaphane might lead to improvements in ASD because it can stimulate cell-protective responses similar to those that occur during fever. These responses may improve communication among nerve cells and improve long-range connections in the cerebral cortex, which are depressed in ASD.
We recently completed a placebo-controlled, randomized, double-blind clinical trial of daily sulforaphane (derived from broccoli sprouts) for 18 weeks in 44 young men with ASD; 29 received the drug and 15 received placebo ("dummy drug"). We measured behavior on three scales; two were recorded by parents, the other by the doctors. Two-thirds of those receiving sulforaphane showed substantial improvements in behavior, whereas those taking placebo experienced minimal change. The differences were statistically significant for all three tests.
Our three objectives in this study are: (1) To determine if there are measurable effects on social responsiveness and problem behaviors in children with ASD during treatment with sulforaphane. (2) To determine if treatment with sulforaphane in children with ASD is safe and well tolerated. (3) To determine which biomarkers respond to treatment with sulforaphane.
This is a clinical trial of oral sulforaphane (as broccoli seed powder) in 50 boys and girls (3-12 years) with ASD. The study will take place over 36 weeks. In Phase 1 (1-15 weeks), 25 children will receive sulforaphane and 25 will receive placebo. In Phase 2 (15-30 weeks), all children will receive sulforaphane. In Phase 3 (30-36 weeks), children will receive no medication. Behavioral assessments will be carried out by the families and doctors. We will follow children closely and measure lab tests for safety and to determine the effects of sulforaphane on cells to learn which tests are useful as biomarkers and to show us how sulforaphane affects the body's metabolism. Sulforaphane is safe, although there may be a risk of seizures in children who have had seizures previously. Children may also have discomfort from blood tests.
From our experience with the positive effects of sulforaphane in young men with ASD, we expect that it will be safe and will show equal or greater clinical benefits in children because the brain is undergoing rapid development and has greater possibilities for improvement than in adults. In addition to its clinical benefits, this study will help us to understand how sulforaphane works in ASD and may give us new basic information about the biochemistry of ASD.