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Medical News & Perspectives
September 25, 2018

The Path to the First FDA-Approved Cannabis-Derived Treatment and What Comes Next

JAMA. 2018;320(12):1227-1229. doi:10.1001/jama.2018.11914

Harvard neurologist Elizabeth Thiele, MD, PhD, is only half-kidding about the surprising direction her research has taken.

“If someone would have said to me 5 years ago that I would be spending most of my time thinking about cannabis, I would tell them they were high,” said Thiele, director of the Pediatric Epilepsy Service at Massachusetts General Hospital, Boston.

Thiele was a lead investigator in clinical trials that led to the June 25 US Food and Drug Administration approval of Epidiolex—“the first drug comprised of an active treatment derived from marijuana,” as the agency describes it—for patients aged 2 years or older who have Lennox-Gastaut or Dravet syndromes. Each is a rare, severe form of epilepsy with unrelenting seizures that can lead to cognitive impairment and, in the case of Dravet syndrome, a high rate of epilepsy-related death at a young age. Currently, she is leading a placebo-controlled clinical trial of Epidiolex to treat seizures in patients with tuberous sclerosis complex, a rare genetic disorder.