Funding Research for Treatments and a Cure

Pre-clinical assessment of cannabidiol as a treatment for Angelman syndrome
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Paul Carney, M.D. UNC-Chapel Hill

Pre-clinical assessment of cannabidiol as a treatment for Angelman syndrome

Summary of Dr. Carney Study

A national figure in cannabidiol (CBD) research, Dr. Paul Carney has been working with the National Academy of Medicine, the National Institute of Drug Abuse and the Florida Department of Health for several years to evaluate existing research on CBD and its potential therapeutic effects. Existing research is anecdotal and there is little, if any, scientific research proving how CBD works; however, some clinicians have already begun using it in a clinical setting. It is known that CBD could be a powerful drug to address symptoms of Angelman syndrome such as seizures, behavioral and motor challenges, and others—but no scientific research exists to support it. Dr. Carney, in collaboration with Dr. Ben Philpot, is pioneering this research to evaluate its effectiveness in treating symptoms of Angelman syndrome.

Dr. Carney will be testing CBD in an AS mouse model for its effectiveness in specific areas: behavioral and motor deficits, seizures and brain activity. This is the first pre-clinical evaluation of CBD in Angelman syndrome, a necessary step to justify a clinical trial per the NIH’s standards.

“Dr. Carney’s research will be valuable to the Angelman syndrome community. Currently, many families are obtaining CBD through hemp oil or from medical marijuana. These formulations differ from each other and can vary significantly among manufacturers. Many families have reported that it has helped with seizures, non-epileptic myoclonus, sleep and even anxiety, but no formal studies have been done in individuals with Angelman syndrome, and in the general population this has only been studied for its effects on seizures. With so many families trying these compounds or interested in trying them, this study will be useful in developing parameters for future clinical trials.”

-Dr. Ron Thibert, M.D. and AS Clinician at the AS Clinic at Massachusetts General Hospital in Boston