Clinical Trial: FDA-Approved CBD Formulation Associated With Clinically Evident Improvements in Autistic Boys

San Diego, CA: The adjunctive use of CBD in the form of the plant-derived FDA-approved drug Epidiolex is associated with symptomatic improvements in autistic boys with severe behavioral problems, according to clinical trial data published in the Journal of Autism and Developmental Disorders.

Investigators affiliated with the University of California at San Diego assessed CBD treatment versus placebo in a cohort of 39 autistic boys ages 7 to 14. Clinicians observed participants on either a weekly or biweekly basis throughout the study period but were unaware of which participants were consuming CBD or placebo until after the study’s conclusion.

“Blinded clinical impressions showed almost two-third of the participants had behavioral improvements with CBD,” researchers reported. “Reductions in aggressive behaviors and hyperactivity were observed most frequently, with improvements in communication being seen in almost 30 percent. Since many of the participants were non-speaking at the onset of the study, this was a notable change, with several of the children beginning to use words for the first time.”

Investigators did not identify significant differences between CBD treatment and placebo on other outcome measures – a result that they attributed to the study’s small sample size and a greater than expected placebo effect. They acknowledged that Epidiolex possesses an acceptable safety profile and is well-tolerated in this patient population. (Epidiolex was approved by the FDA in 2018 specifically for the treatment of Dravet Syndrome and Lennox-Gastaut Syndrome, two rare forms of childhood epilepsy.)

“Despite the limitations, this study provides valuable new information about a potential role of CBD in improving behavior in a subset of children with autism, as well as the strong role the placebo effect plays in this condition,” the study’s authors concluded.

Several other placebo-controlled trials and observational studies have shown that the use of cannabinoids can improve symptoms and provide quality-of-life benefits to patients with autism.

Full text of the study, “Cannabidiol treatment for severe problem behaviors in autistic boys: A randomized clinical trial,” appears in the Journal of Autism and Developmental Disorders. Additional information on cannabis and autism is available from NORML’s publication, Clinical Applications for Cannabis and Cannabinoids.