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)

Based at the UC San Diego School of Medicine's Center for Medicinal Cannabis Research (CMCR), Scientists 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 behaviours, repetitive or self-injurious behaviours, hyperactivity and social and communicative deficits.

ASD affects an estimated one 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. 

"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.

CBD is a primary chemical compound found in cannabis. It does not produce the effects of feeling "high," which are caused by tetrahydrocannabinol or THC, but as one of more than 100 cannabinoids in cannabis, CBD interacts with the body's endocannabinoid system, a network of neurotransmitters that regulate diverse physiological and cognitive processes and response to stress. 

The goals of the study are three-fold: to determine if CBD is safe and tolerable and whether it alleviates adverse symptoms of ASD; to find out if and how CBD alters brain wave activity, neurotransmitters and brain network connectivity; and to uncover whether biomarkers of neuroinflammation are altered by CBD.

The clinical trial will consist of 30 children, ages eight to 12 years, with autism and severe symptoms. All will receive behavioral testing, MRI scans and electroencephalograms.

In the first phase of the study, half the children will receive CBD and half placebo. In the second phase, the groups will be switched and the half who originally received CBD will receive placebo, while the initial placebo group will receive CBD. Investigators will be blinded to which children are receiving which treatment until after all of the testing is completed at the end of the study.

To understand the mechanisms of any potential clinical benefit from CBD, the Noorda gift will support two closely linked basic science studies.

First, using skin cells taken from trial participants, a research team headed by Alysson Muotri, PhD, will use induced pluripotent stem cells to create in vitro models of participants' neurons and organoids -; simple but organised, functional, three-dimensional cell assemblies sometimes called "mini-brains." Muotri and colleagues created the first human cellular model of autism, which can be used as a tool for testing, diagnosis and drug screening, in 2010.

"The findings have the potential to provide treatment options for countless kids with neuro disabilities and can be foundational for the emerging field of cannabinoid therapy."