FDA Approves Landmark Marijuana-Derived Drug First Tested at UCSF

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For the first time, a drug derived from marijuana has been approved by the Food and Drug Administration, and it may soon offer relief to children with hard-to-treat seizures. In 2013, UCSF Benioff Children’s Hospital San Francisco became the first site to administer the drug in a child, and as of this week, the FDA has approved its use in two rare and devastating forms of epilepsy.

“Everybody knew that this could be a good medication for seizures, but still, nobody tried,” said Maria Roberta Cilio, MD, PhD, director of research at the UCSF Pediatric Epilepsy Center. Cilio was the senior author of a multi-center study testing the safety and efficacy of the new drug, Epidiolex, in children and young adults. The study provided evidence that Epidiolex should be considered for FDA approval.

“UCSF was very brave to support me,” Cilio said. “We pioneered with this drug.”

Eleven epilepsy centers across the nation administered Epidiolex to 162 participants over 12 weeks. The participants all had severe, childhood-onset, treatment-resistant epilepsy. The results, published in The Lancet Neurology in 2015, showed that the treatment was well-tolerated by patients and reduced the frequency of their seizures. Participants had a median 36.5 percent reduction in monthly motor seizures, which affect muscle activity, with a median monthly frequency of motor seizures falling from 30 a month to 15.8 over the course of the trial.

In the last month of the trial, six patients were free of all motor seizures and nine more were free of all seizure types. At the start of the study, some of these seizure-free patients regularly had hundreds of seizures a day and wound up in the emergency room every three weeks or so, said Cilio.

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Epidiolex is a purified form of cannabis devoid of the plant’s main psychoactive component, tetrahydrocannabinol, or THC. The liquid medication …

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