Researchers at University of California San Diego School of Medicine are preparing a first-of-its-kind, multidisciplinary investigation to determine if and how cannabidiol (CBD), a non-psychoactive compound found in the cannabis plant, provides therapeutic benefit to children with severe symptoms of autism spectrum disorder (ASD).

The study, scheduled to launch in approximately 1 year, is funded by a $4.7 million gift from the Ray and Tye Noorda Foundation, in partnership with the Wholistic Research and Education Foundation. It represents the largest known private gift to date for medicinal cannabis research in the United States.

Based at the UC San Diego School of Medicine’s Center for Medicinal Cannabis Research (CMCR), the study will involve a team of physicians and scientists employing clinical assessments, coupled with basic and translational research, across a cohort of patients, to better understand how CBD interacts with or alters neural activity and if it effectively and safely alleviates some of the more problematic symptoms of ASD, such as aggressive behaviors, repetitive or self-injurious behaviors, hyperactivity, and social and communicative deficits.

“We clearly need more treatment options for children with moderate-to-severe autism—those who suffer the greatest impact and who need the most help,” said Igor Grant, MD, CMCR director and chair of the Department of Psychiatry at UC San Diego School of Medicine. “It is becoming more apparent that CBD has a number of effects on the central nervous system that may be relevant to autism and there are anecdotal reports suggesting CBD treatments may improve functioning in an ASD child.”

“Investigating the effects of CBD on autism has been a long-term goal for CMCR. We are excited to begin this clinical trial and unlock additional, evidence-based knowledge of how cannabis and cannabinoids can be used as medicines, not only for autism, but in the future for other neurodevelopmental and neuropsychiatric disorders.”

ASD affects an estimated 1 in 68 children in the United States, primarily boys. The neurodevelopmental disorder is complex, with multiple known or suspected causative factors, from inherited genetic mutations to environmental conditions to metabolic dysfunction. A major consequence is abnormal development and functioning of connectivity and communications between brain cells and among neural networks, resulting in many of the observed social and cognitive impairments in persons with ASD.

“The most exciting part of this novel study is that the findings could have tremendous implications for not just children with autism, but for children with other types of neurodevelopmental disabilities,” said Doris Trauner, MD, Distinguished Professor of Neurosciences and Pediatrics at UC San Diego School of Medicine and an attending pediatric neurologist at Rady Children’s Hospital-San Diego with special expertise in neurodevelopmental disabilities. As principal investigator, Trauner will oversee the design and execution of the clinical trial.