美国国家公共电台 NPR How One Boy's Fight With Epilepsy Led To The First Marijuana-Derived Pharmaceutical（在线收听）

NOEL KING, HOST:

The first medication derived from marijuana could be in pharmacies as early as this fall. The FDA recently approved it to treat two rare types of epilepsy. KQED's Lesley McClurg has the story of one family's quest to get this drug.

LESLEY MCCLURG, BYLINE: Evelyn Nussembaum used to watch her son Sam suffer through 100 seizures a day.

EVELYN NUSSEMBAUM: When they were bad, they were once every three minutes.

MCCLURG: Sam was diagnosed with epilepsy when he was 4 years old.

SAM VOGELSTEIN: When I did have a seizure, I would sort of - everything went black kind of.

MCCLURG: For about 20 seconds - just long enough to tumble down a flight of stairs at his house in Berkeley, plunge into a dinner plate, crack his head on a window.

VOGELSTEIN: I don't remember a lot of it really.

MCCLURG: Doctors tried nearly two dozen different medications to treat Sam. Nothing worked long term. And the side effects from many were severe - full-body rashes, fits of rage, strange visuals.

VOGELSTEIN: I hallucinated that my bug sheets came to life and that I had holes in my body.

MCCLURG: Seven exhausting years passed. And then Evelyn came across a study using cannabidiol, or CBD, to successfully treat seizures in rats. CBD is an extract from the cannabis plant that doesn't make you high.

NUSSEMBAUM: And I thought my son needs access to that. I got to get this.

MCCLURG: She dug around and found a British pharmaceutical company that was making highly concentrated CBD for multiple sclerosis patients. The company agreed to let Sam try the drug in the U.K. under a doctor's supervision for two weeks.

NUSSEMBAUM: After one day, his seizures were down to 30. After two days, they were down to 10. After three days, he had one seizure.

MCCLURG: Sam is now 17. The drug still works. And he doesn't have any side effects. For the past six years, the FDA has allowed what's called a compassionate use for Sam. Along the way, hundreds of other patients have tried the drug in clinical trials, which eventually led to its recent FDA approval. The brand name for the CBD drug is Epidiolex.

JOE SIRVEN: This is what everyone asked about.

MCCLURG: Dr. Joe Sirven is a neurologist at the Mayo Clinic in Arizona.

SIRVEN: This almost had like instant name recognition.

MCCLURG: He says his patients read about Epidiolex studies on social media, and then they begged to try it.

SIRVEN: It showed really, really great results - particularly with certain larger seizures, the big convulsions.

MCCLURG: Now, many patients are using CBD from marijuana dispensaries. But these aren't regulated, and the dose and consistency can vary widely. Still, Sirven doesn't necessarily recommend switching.

SIRVEN: I would never change it if it's working for you. If it's not though, here's an option.

MCCLURG: Epidiolex isn't right for everyone. It only reduces seizures in about 30 percent of epilepsy patients. And the drug can cause side effects - like fatigue, nausea, diarrhea, rashes, insomnia. And it's not on the market just yet. First, the Drug Enforcement Administration needs to reclassify CBD. It's currently a Schedule I drug, meaning it's illegal under federal law. That's expected to happen by early fall.

NUSSEMBAUM: So once that's done, it could potentially be in Walgreens or Rite Aid. But there are still big holes - there are big gaps in this.

MCCLURG: The price has not been announced yet. You will need a prescription. And Nussenbaum worries some insurance companies may not cover Epidiolex.

UNIDENTIFIED PERSON: So it looks like we got an order for 10 bottles here.

MCCLURG: For now, Sam still gets his drugs at the investigational pharmacy at UC San Francisco.

UNIDENTIFIED PERSON: All right. And if I could have you sign there for me please.

NUSSEMBAUM: One more time, OK.

MCCLURG: Some day Sam hopes he's the one prescribing Epidiolex.

VOGELSTEIN: I want to be an epilepsy doctor.

MCCLURG: First, the 17-year-old is going to get his driver's license. He was just cleared to get behind the wheel. He hasn't had a seizure in more than two years. For NPR News, I'm Lesley McClurg in San Francisco.