On Monday, the FDA approved its first marijuana-derived drug: a purified form of CBD (cannabidiol, a largely non-psychoactive chemical present in the Cannabis sativa plant) to treat patients with Dravet and Lennox-Gastaut syndromes, rare forms of childhood-onset epilepsy. Called Epidiolex, the drug represents not only a landmark advancement in the treatment of severe epilepsy, but a milestone for medical marijuana research.
“This is huge,” says Dr. Shaun Hussain, assistant professor of pediatrics at UCLA and director of the UCLA Infantile Spasms Program, who has been treating patients with Dravet and Lennox-Gastaut syndromes since 2005. “People who were randomized to receive cannabidiol saw substantial benefit that was above and beyond. It’s very solid data.” The decision follows three high-quality clinical trials in which about 40 percent of patients experienced half or fewer as many seizures, and a unanimous vote from an FDA expert advisory panel that determined CBD has a “negligible” abuse potential.
For perspective, patients with Dravet and Lennox-Gastaut are diagnosed early in life and can experience seizures every day (sometimes in the hundreds), which often cause intellectual development issues. “These are kids who don’t go to regular school, graduate from high school, live or work independently, get married,” says Hussain, who adds that the handful of medications currently available to treat these forms of epilepsy often come with “side effect lists a mile long.” In other words, Epidiolex could not merely increase their quality of life, it will afford them one.
And its significance goes beyond the medication itself. Calling the development “an important medical advance” in a statement, FDA commissioner Dr. Scott Gottlieb cited the well-controlled clinical studies and manufacturing methods surrounding the drug in providing a reliable, reproducible dosage and delivery method that ensures consistent benefits to patients—issues that have at times plagued the developing medical marijuana industry and now offer the potential to catapult the drug into the realm of prescription medicine.
As the first pharmaceutical-grade formulation of rigorously purified cannabis, Epidiolex, an orally-consumed oil, is extracted from a plant groomed by GW Pharmaceuticals, a U.K.–based company, to feature high concentrations of CBD and low concentrations of THC (marijuana’s most famous component, which is known for inducing the “high” of euphoria, nausea suppression, and appetite stimulation). GW Pharmaceuticals then further refines the extraction to a 98–99 percent concentration of CBD and almost no THC. The resulting formula is purer and more easily regulated than over-the-counter CBD available in the U.S.
Though Gottlieb makes the careful distinction—“this is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use”—there is reason for the medical marijuana research community to celebrate. According to GW Pharmaceuticals, in 90 days, the Drug Enforcement Administration (DEA) will be obliged to change the classification of CBD from a Schedule 1 drug, because a government agency has approved it for medical use. And while some, including Hussain, remain doubtful that the DEA will put such a change into motion for marijuana as well, the mere fact of FDA approval may do wonders to remove the stigma from such research.
There’s also the fact that, as Hussain explains it, Schedule 1 drugs are difficult for research facilities to procure and even harder to get approval to bring to trial, requiring a formal sign off by the FDA and obtaining a DEA “researcher” registration that’s good for a single year. The validation of Epidiolex will likely spark a ripple effect, encouraging research for broader uses of medical marijuana.
“We want to learn which products are most effective, which have the most potential, which are truly safe, what are the risks, who are they truly safe for, and are there any people at especially high risk,” says Hussain. Due to the social and bureaucratic barricades that have surrounded marijuana, “that research is just not happening.” Promising fields where cannabis could have potential include benefits for people suffering from Alzheimer’s, autism, and diabetes. And the next focus of research will likely take the form of chronic pain—exploring marijuana derivatives that not only treat it but act as opioid-sparing alternatives. Whether or not cannabis is a medical miracle plant remains to be seen, but now, finally, the research is starting to happen.