Rare Disease Report

AAN 2015 Update-Cannabidiol Shows Promise in Dravet and Lennox-Gastaut Syndromes

APRIL 22, 2015
Christina T. Loguidice
At the American Academy of Neurology (AAN) 67th Annual Meeting, a study was presented suggesting that cannabidiol, a medical marijuana extract that does not produce the psychoactive effects associated with marijuana, may offer promise in treating children and young adults who have severe epilepsy that is refractory to other treatments.1 In the study, cannabidiol reduced the number of seizures by more than half.
The open-label, 12-week study, which sought to determine whether the drug was safe and well tolerated, included a total of 213 people (median age, 11 years) with Dravet syndrome, Lennox-Gastaut syndrome, or 1 of 10 other severe epilepsies known to cause intellectual disability and lifelong seizures. The researchers also sought to assess the number of seizures, which were recorded as convulsive, nonconvulsive, or atonic. All patients received cannabidiol as an oral liquid, which was taken daily and dose-titrated up to 25 mg/kg.


Of the patients enrolled in the study, 137 (64%) completed treatment. Among these patients, the number of seizures decreased by approximately 54% from the beginning of the study to the end.
Dravet syndrome. Among the 23 individuals with Dravet syndrome who finished the study, there was a 53% reduction in the number of convulsive seizures by the end of the study.
Lennox-Gastaut syndrome. Among the 11 people with Lennox-Gastaut syndrome who completed the study, there was a 55% reduction in the number of atonic seizures.
Cannabidiol was well tolerated. Only 12 people (6%) discontinued treatment because of adverse effects. Common adverse effects were considered those that occurred in more than 10% of participants and included drowsiness (21%), diarrhea (17%), fatigue (17%), and decreased appetite (16%).
“So far there have been few formal studies on this marijuana extract,” said Orrin Devinsky, MD, New York University Langone Comprehensive Epilepsy Center, New York, New York, and a Fellow of the AAN in an AAN press release. “These results are of great interest, especially for the children and their parents who have been searching for an answer for these debilitating seizures,” he added.
Dr. Devinsky said in the media release that these are early findings and that larger, placebo-controlled, double-blinded trials are needed to more accurately measure the safety and effectiveness of the drug.
GW Pharmaceuticals, which sponsored the study, is working towards advancing its formal clinical development program as a means to seek US Food and Drug Administration approval for cannabidiol (Epidiolex) in the treatment of Dravet syndrome and Lennox-Gastaut syndrome.2 The company recently commenced the first of 2 phase 3 trials in Dravet syndrome and expects to commence phase 3 trials in Lennox-Gastaut syndrome in the second quarter of 2015.2


1. Devinsky O, Sullivan J, Friedman D, et al. Epidiolex (cannabidiol) in treatment- resistant epilepsy. Poster presented at: 67th Annual Meeting of the American Academy of Neurology; April 18-25, 2015; Washington, DC.
2. GW Pharmaceuticals notes new Epidiolex® data released by the American Academy of Neurology [news release]. London, UK: GW Pharmaceuticals; April 13, 2015. http://globenewswire.com/news-release/2015/04/13/723979/10128639/en/GW-Pharmaceuticals-Notes-New-Epidiolex-R-Data-Released-by-the-American-Academy-of-Neurology.html. Accessed April 21, 2015.

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