Washington, D.C. — A landmark approval by the FDA will put the first marijuana-derived prescription drug behind pharmacy counters this fall. Made from cannabidiol (CBD) and containing no mind-altering tetrahydrocannabinol (THC), Epidiolex is approved to treat two rare types of epilepsy, Lennox-Gastaut Syndrome and Dravet Syndrome, in which seizures begin in early childhood and can produce devastating effects down the line.
The medicinal potential of CBD has intrigued scientists for a long time, but it’s been difficult to probe on account of strict marijuana regulations, even in academia. Epidiolex’s road to approval is an uncommon one, highlighting the extraordinary journey of one patient—and his mother.
Berkeley, Calif. native Sam Vogelstein was four years old when he experienced his first seizure, and it wasn’t long before he was having as many as 100 seizures a day. His particular condition, called Epilepsy with Myoclonic-Absences, is classified as a refractory epilepsy, meaning that it resists conventional treatment and tends to persist into adulthood. The procession of prescription drugs that Vogelstein got from his doctor gave him severe hallucinations, anger-management issues, and full-body rashes. His mom, Evelyn Nussenbaum, grew increasingly frightened, and he fell behind in school.
Seven years ago, Nussenbaum happened upon a journal article about CBD effectively treating seizures in rats. But the substance was still difficult to obtain—and illegal. Later, after hearing that GW Pharmaceuticals, the British company that now manufactures Epidiolex, was studying the effects of CBD on Multiple Sclerosis patients, she spent months working to persuade them to let her son sample the treatment, which he was ultimately permitted to do: under strict doctor supervision, in London. After just three days, the results were staggering: his daily seizure tally was down to one, with zero side effects.
The London trial was just two weeks long. In collaboration with doctors back home in California, Nussenbaum spent four months petitioning the FDA to permit her son to have more CBD. Ultimately, the administration approved a one-person clinical trial. Vogelstein, now 17, has been seizure-free for over two years.
Why This Matters
While CBD is legal now, available online and all over, quality and dosage tend to vary substantially—because the substance is not regulated like prescription medications—and it’s thus an unviable course of treatment for a serious condition. But thousands of epileptic patients will be able to count on a prescription CBD-based drug for relief. Additionally, the approval of a marijuana-derived medication may also herald more vigorous research into the plant’s broader healing potential.