By the time he was six, Sam, the son of Evelyn Nussenbaum, Berkeleyside’s video storytelling producer, was having dozens of epileptic seizures a day. Nussenbaum and her husband, Fred Vogelstein, discovered that a British-made, cannabis-based drug stopped the convulsions. But they couldn’t use the medicine in the U.S. because it was illegal. Their push to bring the medicine to the U.S. eventually proved successful. Sam is now 17 and, earlier this week, the Food and Drug Administration approved its first cannabis-derived drug. Nussenbaum has written about her family’s experience.
My son Sam Vogelstein pioneered Epidiolex, the first cannabis-derived drug approved by the FDA.
By pioneered I mean this: our family found the medication, convinced its British maker to let us try it, and helped set in motion its launch and path to the United States. It’s changed everything for us. I hope it changes things for many more patients and, just as importantly, leads to a new way to think about and study cannabis in this country.
Sam started Epidiolex to treat his seizures in May of 2013. He’s been completely seizure-free two and a half years. He has, or had, Epilepsy with Myoclonic Absence, that caused him to have up to 100 blinky, jerky seizures a day. He cracked a window pane with his head; went face down, hard, into a plate of spaghetti; had a seizure at the top of the stairs and fell down the entire flight.
“The uncontrolled epilepsy interfered with his learning, playing and socializing, and terrorized our entire family.”
The seizures started when he was 4 and got worse when he was 6. Nothing we did stopped them — not two dozen seizure medications, the ketogenic diet, or the corticosteroids that gave him moonface like a cancer patient. By the time we found our way to Epidiolex he’d had uncontrolled epilepsy for eight years. It interfered with his learning, playing and socializing, and terrorized our entire family.
Many adults with epilepsy smoke pot to control their seizures. This is no secret to neurologists; with a third of the epilepsy population unresponsive to epilepsy medications, patients take matters into their own hands. But children have the same poor seizure control rates as adults and they shouldn’t smoke pot; the little we know about THC’s effect on the developing brain suggests it’s not good.
There have been rumblings about the anti-seizure properties of CBD, or cannabidiol (another part of the cannabis plant and the active ingredient in Epidiolex) since the 19th century. But governments have made it nearly impossible to study. I learned about it in a 2012 issue of the British medical journal Seizure, which had a paper about how the compound worked as an anticonvulsant in rodents. It was a ridiculously slim thread to hang my hopes on, but we didn’t have any other options. There were no other medications to try and Sam was not a surgery candidate.
But getting a reliable source was tough. First, we tried CBD tinctures we bought locally. But when we had them lab tested, they all had less CBD than their labels claimed, and some had none at all. Then we bought pot with a scientist friend who also had a child with epilepsy. She found chemists to extract the CBD and they gave us each what we estimated was enough for two weeks. Both kids responded, but the two weeks went fast and the chemists couldn’t risk helping us again.
Then I read about GW Pharmaceuticals in London, a company studying the cannabis plant with the British government’s permission. Epilepsy wasn’t their focus then. There was no such thing as Epidiolex. But they had greenhouses, their own plant stock, labs, and were extracting cannabidiol and other cannabis compounds regularly and systematically.
It’s easy to hate “evil” drug companies. I’m not pro or con. But they are good at something crucial when you’re trying to treat a poorly understood, life-threatening disease — quality control. The medicine they make is exactly the same from batch to batch, bottle to bottle, and dose to dose. You don’t think about how important that is until you can’t rely on it.
I’m not pro or con cannabis either. I never aspired to treat Sam with any or all of the marijuana plant. I don’t get a warm fuzzy when people congratulate us on treating him with what they see as a natural substance. I’m relieved that doctors are overseeing the administration of this plant compound, isolated from the other compounds with which it normally occurs. If I’d found good science that a motor oil extract could help seizures I would have pursued that.
“If I’d found good science that a motor oil extract could help seizures I would have pursued that.”
But I pursued Dr. Geoffrey Guy instead. And when I finally got through to GW Pharmaceuticals’ chairman, in the fall of 2012, he wasn’t surprised. While he’d been developing cannabis-derived drugs for cancer pain and multiple sclerosis, he’d been reading the same seizure studies I had. When he learned that Sam had responded to lab-extracted CBD he was open to letting him try GW’s; just not in the United States. Anti-cannabis sentiment here was too strong.
But it was legal for us to try it in London under a doctor’s supervision. So we did, during Sam’s fifth-grade Christmas vacation. And it worked. The day before Sam started GW Pharmaceuticals’ CBD extract he had 68 seizures. After three days, he had one in one day. We stayed for two amazing weeks, most entirely seizure-free. In between multiple blood tests and EEGs Sam ziplined over London at an amusement park. We spent a day at the science museum. We rode the London Eye ferris wheel. And then we had to leave. It wasn’t legal in the U.S., so GW Pharmaceuticals came to our hotel and collected the remainder. I considered stealing it but chickened out.
Once home, we submitted applications to the DEA and the FDA to make Sam his own one-boy trial so he could continue to get the drug. We got approval a few months later, in mid-2013. Inspired by Sam’s success on the drug, GW Pharmaceuticals then began extensive trials at UCSF, New York University, Massachusetts General Hospital in Boston and some 30 other hospitals. The company briefly considered naming its compound SamCan but ultimately settled on the more corporate-sounding Epidiolex.
Earlier this year, Sam and I traveled to Washington D.C. to testify about our experiences in front of the FDA. Some 30 people testified. Sam spoke and was the only person who got applause. Some parents came up to us afterwards and thanked him for speaking on behalf of their children, many of whom were too sick to speak for themselves.
Now that Epidiolex is FDA-approved it could be in Walgreens by September. Still on the drug, Sam is 17, six feet tall and will sleep until noon on the weekends if we let him. He fences and his coach just told him he’s ready to start competing in tournaments. He’ll start 11th grade in the fall. He has had some minor setbacks, prompting us to start him on a second drug a few years ago, but he’s not having seizures anymore. It’s all so normal. Gratitude doesn’t begin to describe what I feel.
Questions remain. Is Sam free of this terrifying disease for good? Will Epidiolex always work for him? Will insurance companies cover it for him or anyone else once GW Pharmaceuticals prices it? Will it cost a fortune? I don’t have a clue.
But I do know that I want the legalization of Epidiolex to do more than help stop seizures. I want its status as a legal drug to make it easier for scientists to get their hands on cannabis and pull it apart in the lab. With pot legal for recreation in so many states we should understand it better. Maybe we could quantify when someone is too stoned to drive. It turns out that cannabis high in THC can provoke seizures in people who have never had epilepsy. We should know why. Maybe more medications can be made from cannabis. Maybe there’s something in it that works better than Epidiolex. Maybe not. But it’s crazy not to try and find out.
