The FDA has approved its first cannabis drug. What's next?

Five years ago, I bought a safe for my son's doctor to store drugs. It was blue, about three square feet, and weighed 965 pounds, as the Acme Wile E. Coyote safes used to try to drop on Road Runner. My family and I had evidence that the drugs could cure our 11-year-old son from his relentless seizures. But because the drugs contained a derivative of a cannabis plant known as cannabidiol, or CBD, the drugs had to be handled as if they were heroin, or any other medicine called Schedule 1. Every few months, when a new shipment arrived, me or my wife Evelyn would appear at the door of the UCSF office, Roberta Cilio. She opened the safe, made us sign papers, and gave Sam's medicine in a brown paper bag to take him home.

Medicine helped our son, and many more. Sam's seizures fell from 100 a day to about five a day – and GW Pharmaceuticals, the maker, was so encouraged by his response that he started talking to the doctors about epilepsy about clinical tests. On Monday, the Food and Drug Administration approved the drug, now known as Epidiolex, for sale in the United States. It will probably be available on prescription in pharmacies in the fall.

Like any new drug, we will not know it's up to the hype for a while. What is clear is that it will definitely change the way we talk about cannabis in America. Cannabis is as legal as alcohol in Washington and eight states, including the entire West Coast. But doctors, scientists and hospital administrators are governed by federal and non-state laws, making cannabis research a risky and time-consuming venture. It's about to change permanently.

Preliminary data suggest that CBD could be useful not only for epilepsy, but also for a wide range of neurological diseases such as Parkinson's disease, Alzheimer's disease, multiple sclerosis and certain cancers. brain. "There are neuroscientists who drool to work on it," said Elizabeth Thiele, director of the Pediatric Epilepsy Program at Mbadachusetts General Hospital and one of the leading investigators of GW trials. Once the DEA reprogrammed Epidiolex, they will finally be able to do it.

The approval also allows GW to speed up its own internal research on cannabis. GW is already testing Epidiolex for the treatment of tubular sclerosis, a condition other than epilepsy that often causes convulsions. GW now has a political stake with US regulators that it plans to use to get approval for Sativex, a THC and CBD oral spray for cancer pain and multiple sclerosis. , available in the UK since 2010 and in other European countries like Germany since 2011. If this product is approved, US scientists will finally have a legal and relatively easy way to test the effects of THC dosed for many disorders .

For now, the biggest unknown is how insurance companies will decide to cover Epidiolex. The double-blind, placebo-controlled trials required for FDA approval covered only two rare pediatric types of epilepsy: Dravet's syndrome and Lennox Gastaut's syndrome. Approximately 50,000 patients are affected by both diseases. But there is already evidence that Epidiolex could help with dozens of different types of seizures and epileptic syndromes.

Indeed, two-thirds of the 1,756 patients who have tried Epidiolex in the past five years have not had Dravet or LGS, including Sam. The demand for Epidiolex was so high in patients who did not were not responding to drugs, that GW allowed neurologists from more than four dozen hospitals that were not part of the formal trials to conduct their own so-called open trials. He helped GW learn more about how Epidiolex worked in a larger population, and it allowed a large number of sick patients to have access to medications that could help them.

This parallel research should allow doctors to prescribe the drug for other diseases, a practice known as "prescription of a drug". It simply means that, according to the professional opinion of a doctor, a drug is worth the candle and epilepsy Thiele says.

The reason why pharmaceutical companies focus on rare diseases like Dravet and LGS – at least for epilepsy drugs – is motivated as much by commercial and bureaucratic as medical considerations, doctors and pharmaceutical executives alike. ;have said. It is difficult to obtain permission to test drugs in children unless these children have diseases like Dravet and LGS. In addition, companies can get FDA approval more quickly and benefit from patent protection longer if they develop drugs for rare diseases like Dravet or LGS.

What all this means is that GW will have to make sure that patients who are already taking Epidiolex for other forms of epilepsy do not wake up suddenly in the fall and become obligated to pay the high price for the drugs that suit them. It also means that physicians will need to mobilize their expertise and the data that already exists.

Thiele says insurance companies finally decide to cover epilepsy drugs for many more kinds of seizures than they were originally prescribed. The only question in his mind is how long will it take. The only thing she knows, is that the public pressure for insurance companies to cover Epidiolex will be intense. "Every epileptologist I know says that patients have asked when they can get Epidiolex for a year now," she adds.

As for Sam, he wants to become a neurologist when he grows up. Two and a half years ago, when we combined Epidiolex with another medication, Sam's fits stopped completely. Sam, now 17, has received medical clearance to drive a car, something with – well, everything – that seemed impossible to imagine five years ago.

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