A nationally renowned pediatric neurologist at Saint Barnabas has gotten FDA approval to study whether a cannabis-based drug could prevent seizures in children diagnosed with severe forms of epilepsy.
The development is sure to be watched closely in New Jersey, where a number of families whose children have Dravet syndrome, a potentially deadly form of epilepsy, say they cannot obtain yet a useful form of medicinal marijuana through the state Department of Health.
The U.S. Food and Drug Administration gave Orrin Devinsky, director of the New York University and Saint Barnabas Comprehensive Epilepsy Center, Roberta Cilio of the University of California – San Francisco’s Neurology Department, and GW Pharmaceuticals of the United Kingdom permission to use the experimental drug, Epidiolex, to treat 125 children with seizure disorders for whom traditional medicines have failed.
Enrollment in the trial will begin next month, Devinsky said.
“I think this is a big step forward in the science of Cannabidiods,” said Devinsky, referring to the spectrum of active ingredients in marijuana. “We will finally get some data on epilepsy, and this should provide the basis to plan a randomized double-blind study.”
The drug is a liquid form of pure cannabidiol, a non-psychoactive component of the cannabis plant that according to anecdotal reports has shown to reduce the frequency and intensity of seizures in children. The FDA’s Nov. 15th approval also gives Epidiolex “orphan drug” status, providing GW full marketing rights to be the lone producer of the product for seven years, the company said in a statement.
Orphan diseases are rare, occurring in less than 200,000 of people in the nation. There are 5,440 documented cases of Dravet syndrome in the United States and 6,710 in Europe, although the actual number may be higher because the condition is under-diagnosed, according to a statement from the pharmaceutical company. The seizures can damage the brain, delay development and cause lifelong intellectual disabilities.
Meghan and Brian Wilson of Scotch Plains, who have fought a very public battle to obtain medicinal marijuana for their 2-1/2-year-old daughter Vivian, said they are eager to see the drug trial get underway. But Vivian will not be enrolled next month out of concern for her fragile health.
Based on the experiences of other families whose children have Dravet, the children who were weaned off the anti-seizure drug Benzodiazepine — a tranquilizer that prevents or stops seizures by slowing down the central nervous system — saw the most benefits from cannabidiol. Cutting back on the “Benzo,” even very slowly, has triggered many severe seizures, Vivian’s mother said.
“I think it’s a great step in the direction of valid scientific research with cannabis,” she said. “I’d like to see the data before we add on another pharmaceutical, because at the end of the day it is a another pharmaceutical.
These are the first trials in human for this particular compound.
Brian Wilson said getting Vivian into the trial would take a “hard core withdrawal” from the Benzo. “I’d hate to expose Vivian to that until we know it will work.”
Parents whose children have Dravet, Lennox-Gastaut syndrome and other serious seizure disorders, say they are eager to try cannabidiol produced by growers in Colorado that has helped reduce seizure activity in more than 100 children. But none of the three medical marijuana dispensaries in New Jersey — including one that opened Wednesday and another Oct. 28 – carry the strains of pot that have shown any promise. No one has submitted a proposal approved by the state to produce a pot-infused edible product, which under state law are available for children.The Wilsons purchased marijuana from Compassionate Care Foundation, Inc. in Egg Harbor, cooked it down into an oil, but cannot find a lab in the state willing to test its potency.
GW already has a track record producing marijuana-based medicine. Savitex, a cannabis-based spray used to treat muscle spasticity for patients with multiple sclerosis, is available in 22 countries. It is under FDA review.
“Through its efforts, GW aims to provide the necessary evidence to confirm the promise of CBD in epilepsy and ultimately enabling children to have access to an FDA-approved prescription,” according to a statement from Stephen Wright, GW’s research and development director.