Dravet syndrome, a severe form of childhood epilepsy that affects approximately one in 20,000 people, is notoriously difficult to treat. Most patients don't reach adulthood (typically due to sudden death during a seizure), and those who do typically can't live independently. A promising new treatment option has come from an unexpected place: a chemical from marijuana plants. According to new research published in The New England Journal of Medicine, cannabidiol decreases the number of convulsive seizures in patients.
120 children with Dravet syndrome participated in the study, which was a randomized, double-blind, placebo-controlled human trial. The patients were assigned to receive either an oral solution of cannabidiol or a placebo for a 14-week period. According to the research published this week, participants who took cannabidiol reported a seizure decrease that was 23 percentage points greater than the decrease among children assigned to take a placebo. Furthermore, five percent of the children became entirely seizure-free during the course of the study.
There was a major downside — a whopping 93% of cannabidiol patients reported side effects, and eight of them dropped out of the study entirely for this reason.
"Tiredness (somnolence or fatigue) was most common; others were decreased appetite, diarrhea and vomiting," said Dr. Orrin Devinsky, lead author of the study and director of NYU Langone's Comprehensive Epilepsy Center. However, he believes the study's results indicate cannabidiol could be beneficial to many Dravet syndrome patients and people who suffer from other forms of epilepsy.
The marijuana plant is classified as a Schedule I controlled substance, and scientists report that strict federal guidelines have slowed down their research regarding the plant's medicinal benefits.
"Hopefully, the FDA will see this as a sign it should be moved off of Schedule I. If it stays on Schedule I, we will have trouble getting it to our patients when it does become available," says Dr. Brenda Porter, an associate professor of neurology at Stanford School of Medicine.
"Before publication of this trial, much of the clinical evidence about CBD's effects on people's seizures was uncontrolled and anecdotal," says Brandy Fureman, vice president of research and new therapies at the Epilepsy Foundation. She describes the new study as "critically important" for the epilepsy community.
Although the findings are enlightening and encouraging, experts agree that additional scientific research is needed. "Natural substances are not necessarily safe and effective. They need to be evaluated rigorously," Devinsky says.