Their work may have finally paid off.
GW Pharmaceuticals announced Monday that the first of its four major studies of the cannabis-based drug appeared to dramatically reduce seizures in patients with Dravet syndrome, a rare and severe form of epilepsy.
“This shows that cannabinoids can produce compelling and clinical important data and represent a highly promising new class of medications, hopefully in a range of conditions,” Justin Gover, GW Pharmaceuticals’ chief executive, told Reuters.
The trial, which involved 120 patients, showed that the median reduction in convulsive seizures was 39 percent in those taking the drug, called Epidiolex, versus 13 percent in those taking a placebo. The mean age of patients was 10 years old and they had previously tried and failed an average of more than four anti-epileptic treatments.
GW is also conducting other Phase 3 studies on Dravet syndrome with 150 patients and Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex.
Orrin Devinsky, a researcher with New York University Langone Medical Center’s Comprehensive Epilepsy Center who is the principal investigator, said that the data finally provides the epilepsy community “the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future.”
The company’s announcement that it will be seeking Food and Drug Administration approval for the treatment sent its shares soaring 120 percent by midday. The company has fast-track status for its application to treatment on newborns with epilepsy. Jim Cramer of “The Street” said on CNBC’s Squawk that the positive reaction may also be due to the hope that the drug could replace some addictive painkillers.
The theory that marijuana could be used to treat seizures has been around since the 19th century, but it wasn’t until about 2012, when a Colorado mom named Paige Figi began posting stories online about her daughter’s experiences with a cannabis extract, that the idea took off. Figi’s daughter, Charlotte, had Dravet syndrome and had been suffering from more than 300 seizures each week. She used a wheelchair, could only say a few words and had gone into cardiac arrest several times.
Desperate, the family began treating her with a few drops of an extract made from a strain of marijuana that was high in CBD, which is thought to be medicinal, and low in THC, the component that creates a high. The family reported that the seizures nearly stopped.
As their story spread across the country, parents held bake sales, benefit concerts and other fundraisers to try to raise money for their own children. Hundreds moved to Colorado while others tried to work within their own states to legalize the use of the treatment. Devinsky, who has a child with epilepsy himself, was one of the early proponents of trying to do rigorous clinical trials of the drug and teamed up with GW Pharmaceuticals, which had previously been working on cannabis-derived drugs for multiple sclerosis.
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