“Medical marijuana has been documented as a treatment for epilepsy going back to 3,800 years ago,” said Orrin Devinsky, director of the Comprehensive Epilepsy Center at NYU Langone Medical Center, who led the research. But the randomized, placebo-controlled study represents the first research to demonstrate the product's value in a scientific way, he said.
Unlike THC, the compound in marijuana that produces a high when consumed, cannabidiol is not psychoactive. The main side effects suffered by the children in the study, whose average age was just under 10, were vomiting, diarrhea and loss of appetite.
But the product is not available anywhere in the world, except to about 1,500 children who are receiving it from the manufacturer, GW Pharmaceuticals, under compassionate-use rules for the condition. Dravet syndrome causes ongoing seizures, cognitive problems and risk of early death.
There is no FDA-approved treatment for Dravet, so many parents have been traveling to Colorado and other states that offer medical marijuana in search of cannabidiol. Other medicines have proven largely ineffective; the children in the study had tried an average of four anti-epilepsy drugs each.
GW Pharmaceuticals, which funded Devinsky's research, plans to submit the drug for approval by the Food and Drug Administration in the middle of this year with the hope that it could be available by prescription in 2018 for children with Dravet and another severe form of epilepsy, Lennox-Gastaut syndrome. The company announced in March 2016 that an earlier stage of the study had shown the drug to be effective.
“The important thing for us is that patients like this deserve a pharmaceutical solution,” said the company's chief executive, Justin Glover. “They should not be moving across the country. They deserve the right to have access” to the drug, which GW calls Epidiolex. The company conducted 18 years of basic research before beginning the development of the drug three years ago, he said.
Marijuana remains a Schedule 1 drug in the United States, which means it has no legitimate medical use. If the FDA were to approve Epidiolex, the Drug Enforcement Administration would change that listing.
The substance was difficult to obtain even for research purposes, Devinsky said. The lab had to install a safe that was so heavy that it consulted with structural engineers to make sure the floors would support it, he said.
Devinsky said cannabidiol appears to bind to a receptor in the brain that dampens the excitability of nerve cells that lead to seizures. It isn't addictive and causes no high. It may someday prove to be valuable as a treatment for many kinds of epilepsy, which afflicts 3 million people in the United States, he said.
In an editorial that accompanied the study, the New England Journal of Medicine praised the study for finally bringing academic rigor to the debate about medicine derived from cannabis. “The trial represents the beginning of solid evidence for the use of cannabinoids in epilepsy,” the journal wrote. “It requires replication.”
Correction: This story originally stated that Devinsky has a child with epilepsy.