The news from London-based GW Pharmaceuticals is worth celebrating:
Epidiolex — purified, pharmacy-grade and nonintoxicating cannabidiol extracted from the cannabis plant — has both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are currently no treatments approved in the U.S.
Epidiolex is being developed using best research practices in accordance with FDA requirements concerning efficacy and consumer safety and other federal laws. Its success helps underscore that non-pharmacy-grade CBD oils typically sold at marijuana dispensaries, including the so-called “Charlotte’s Web,” are not the best, safest options for using CBD for epilepsy treatment. They even may be dangerous.
As I wrote in June 2014:
I want children with intractable epilepsy, or with any other illness that qualified medical professionals believe could be treated, to receive a purified, pharmacy-grade CBD oil — meaning a substance free from THC, the intoxicating and addictive component of cannabis that is especially harmful to the developing brain. First and foremost, we need a pharmacy-grade, purified CBD oil product because children should not ingest any amount of THC — even as little as the 0.3 percent often explained as “no big deal” by many state and federal lawmakers. If a child needs 200-600 milligrams of CBD a day, as many do, this adds up to a lot of THC administered in oil products that are not pharmacy-grade! Secondly, we need a purified oil because that is what will help the world glean the most informed science. Researchers should work only with CBD oil whose content is known exactly and is produced consistently so it can be dosed precisely by a licensed, medical professional.
GW Pharmaceuticals’ full public statement about the efficacy of Epidiolex is posted here. Among the highlights:
“The results of this Epidiolex pivotal trial are important and exciting as they represent the first placebo-controlled evidence to support the safety and efficacy of pharmaceutical cannabidiol in children with Dravet syndrome, one of the most severe and difficult-to-treat types of epilepsy,” said Orrin Devinsky, M.D., of New York University Langone Medical Center’s Comprehensive Epilepsy Center. “These data demonstrate that Epidiolex delivers clinically important reductions in seizure frequency together with an acceptable safety and tolerability profile, providing the epilepsy community with the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future.”
“The primary efficacy endpoint was a comparison between Epidiolex and placebo measuring the percentage change in the monthly frequency of convulsive seizures during the 14-week treatment period compared with the 4-week baseline observation period. In this study, patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent compared with a reduction on placebo of 13 percent, which was highly statistically significant (p=0.01). A series of sensitivity analyses of the primary endpoint confirmed the robustness of this result. The difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period.”
“In addition to the Dravet syndrome trials, GW is conducting the largest global pivotal clinical trial program to date in Lennox-Gastaut syndrome, another rare and severe form of epilepsy.”
“Based on the findings of the physician-led expanded access program, GW continues to identify additional development targets for Epidiolex within the field of pediatric epilepsy. A Phase 3 trial in a third pediatric epilepsy indication, Tuberous Sclerosis Complex, is due to commence imminently and clinical development in a fourth indication is expected to commence in the second half of 2016.”
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Dr. Christian Thurstone serves as medical director of the University of Colorado’s addiction psychiatry fellowship program. He is also the co-author of the recently released book, Clearing the Haze: Helping Families Face Teen Addiction.