Article by Andrew Joseph, Stat News
For the first time, scientists have demonstrated that a component of cannabis reduces seizures in children with a rare form of epilepsy, marking a significant step in efforts to use marijuana and its derivatives to treat serious medical conditions.
The company that sponsored the Phase 3 trial, GW Pharmaceuticals, had already announced some of the results, but researchers said the full peer-reviewed study, published Wednesday in the New England Journal of Medicine, validated the importance of the research. They also pointed out that the drug, cannabidiol, helped some patients more than others and was associated with a range of sometimes severe side effects, a significant finding because some families have been treating their children on their own in states where recreational marijuana use is legal.
“We now have solid, rigorous scientific evidence that in this specific syndrome, cannabidiol is effective at reducing seizures,” said Dr. Orrin Devinsky, a neurologist at New York University Langone Medical Center and an author of the new study. But, he added, “This is not a panacea.”
Cannabidiol, which GW has branded as Epidiolex, is a non-hallucinogenic component of marijuana that can be purified and administered in oil.
For the trial, researchers enrolled 120 children from 2 to 18 years old with Dravet syndrome, a rare genetic form of epilepsy that kills up to 20 percent of patients by the time they are 20. There are no drugs approved specifically for Dravet.
During the study, the patients stayed on their normal treatment regimen, and half of them also received cannabidiol while the remainder were given a placebo. Over a 14-week treatment period, the median number of convulsive seizures in the cannabidiol group decreased from 12.4 to 5.9 per month; for the placebo group, the number went from 14.9 to 14.1.
In the cannabidiol group, 43 percent of patients had their number of seizures cut in half or more, compared with 27 percent in the placebo group. And 5 percent of patients taking cannabidiol saw their seizures disappear, compared with none in the placebo group.