PHILADELPHIA — In the largest study of its kind, researchers report that Epidiolex, a pharmaceutical-grade, purified form of cannabidiol, is generally well-tolerated and is a promising treatment for treatment-resistant epilepsies.
The results, from the open-label Expanded Access treatment programs from 16 sites, were presented at the 2015 American Epilepsy Society Annual Meeting in Philadelphia.
Epidiolex, manufactured by GW Pharma, is a highly-standardized, plant-derived form of cannabidiol (CBD), the most abundant non-psychoactive cannabinoid derived from the cannabis plant. Previous animal studies and anecdotal reports in children have shown anticonvulsant efficacy, especially in those with treatment-resistant epilepsies including Dravet syndrome and Lennox-Gastaut syndrome.
In the current study, led by Orrin Devinsky, MD, of NYU Langone Medical Center, children and young adults with treatment-resistant epilepsy were enrolled in a 12-week prospective observational study. During a 4-week baseline period, parents and/or caregivers of patients kept prospective seizure diaries of all countable motor seizure types. During the 12 weeks of CBD therapy, patients received a gradually increasing dose from 2-5 mg/kg/day until intolerance occurred or a maximum dose of 25 mg/kg/day was reached. Patients were seen every 2 to 4 weeks during the therapy program, and testing for hematologic, liver, kidney function, and antiepileptic drug levels was performed at baseline and at 4, 8, and 12 weeks of CBD therapy.
Ultimately, 261 patients (52% male; average age 11.8 years (range = 4 mo.-41 years old; average weight = 38 kg; average concomitant AEDs = 3) received 12 weeks of CBD therapy with data available at the last group collection. Seventeen percent had a diagnosis of Dravet syndrome and 15% had Lennox-Gastaut syndrome.