Cannabidivarin (CBDV) vs Placebo in Children with PWS

Funding Summary

This grant will support a small clinical trial evaluating the impact of cannibidiol cannabidivarin (CBDV) on hunger and behavior.  CBDV is a compound similar to CBD, but with potential advantages over CBD for PWS. In this study, Dr. Hollander’s group will investigate a form of CBDV, a non-psychoactive drug that has anti-inflammatory, antioxidative, neuroprotective, anti-anxiety and anticonvulsant properties. They will perform a clinical trial comparing CBDV to placebo in children and young adults with PWS, evaluating irritability, restricted/repetitive behaviors, hyperphagia, and caregiver quality of life.

Theresa Strong, Director of Research Programs, shares details on this project in this short video clip. 

Watch the full webinar describing the 11 research projects funded in this grant cycle here. 

Lay Abstract

Prader-Willi Syndrome (PWS) is a neurodevelopmental disorder characterized by irritability,repetitive/disruptive behaviors, hyperphagia, and sleep disturbances. This combination of symptoms in PWS often contributes to high levels of caregiver strain. We propose a 12-week treatment study of cannabidivarin (CBDV) vs. placebo 24 in children and adolescents ages 5-18 with PWS. (CBDV) is a non-psychoactive phytocannabinoid and a homolog of cannabidiol (CBD).This proposal is the first test of this novel therapeutic agent in pediatric PWS. While there is no pediatric data of CBDV in PWS, there are ongoing studies of CBDV in pediatric Rett Syndrome and Type II Diabetes (ages 5-18), and preliminary studies show no safety concerns for pediatric populations. Additionally, we were recently funded by the Department of Defense to study CBDV in childhood ASD (ages 5-18). CBDV has the potential to be a valuable therapeutic agent for PWS individuals by virtue of its anti-inflammatory, antioxidative, neuroprotective, anti-anxiety and anticonvulsant properties. CBDV, similarly to CBD, may modulate sleep that is often disturbed in PWS and may play a role in food regulation. With its effects on multiple mechanisms known to be dysfunctional in PWS and low side effect profile, CBDV is a promising treatment that needs further exploration in this population. Additionally, CBDV has a number of distinct advantages over CBD for PWS, including better outcomes in animal models of related disorders and no appreciable tetrahydrocannabinol (THC) levels [less than 0.2%]. We hypothesize that CBDV will be superior to placebo and will have a positive effect on the core symptoms of PWS including irritability, restricted/repetitive behaviors, hyperphagia, and caregiver quality of life. If proven superior, this data will contribute to the knowledge of CBDV as a beneficial treatment with minimal side effects for PWS symptoms. This knowledge will lay the foundation for future studies of CBDV in PWS, and is a crucial step towards the approval and marketing of CBDV as a treatment for PWS. GW Pharmaceuticals will provide the CBDV drug and matching placebo, but does not have the funds to support this project. Our group has developed close ties with the PWS community, having successfully studied oxytocin vs. placebo in childhood PWS in a prior grant with the same budget and sample size.

Research Outcomes: Public Summary

We have completed plots for the 6 subjects randomized into the study for key behavioral measures. Despite the small sample size, preliminary findings suggest improvements in select symptoms associated with PWS in the CBDV group compared to placebo. Reductions in repetitive behaviors and rigidity were observed on the Montefiore-Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS), Repetitive Behavior Scale-Revised (RBS-R), and the Children’s Yale-Brown Obsessive Compulsive Scale (CY-BOCS) in the CBDV group relative to placebo.