FDA-Approved Medical Marijuana Clinical Trial for Epilepsy
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 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.
November is National Epilepsy Awareness Month!
The evidence for the efficacy of cannabis in the treatment of epilepsy is overwhelming. Dr. Margaret Gedde has studied the effects of cannabis on various forms of epilepsy such as Dravet syndrome, Doose syndrome, Lennox-Gastaut syndrome, metachromatic leukodystrophy, cortical dysplasia, and idiopathic epilepsy. Dr. Gedde employed a compound of cannabis known as cannabidiol (CBD) in her studies, and each patient saw improvement where traditional treatments had failed. The results were dramatic. Eight out of 11 patients saw a 98-100% reduction in seizures. One patients saw a 75% reduction, and two reported a 20-24% reduction.
In another recent study, the NYU School of Medicine, in conjunction with the director of the NYU Comprehensive Epilepsy Center, published a report on a trial sponsored by GW Pharmaceuticals. The study is imperative for GW Pharmaceuticals, as they are attempting to pass the FDA approval process for a new drug called Epidiolex – a liquid form of pure CBD – in order to treat epilepsy.
To test the effectiveness of CBD on epilepsy patients, they sponsored the NYU study of 137 Dravet and Lennox-Gastaut patients during a 12 week period. The patients in the study had been previously unresponsive to epilepsy medications that only succeeded in producing side-effects such as drowsiness, diarrhea, decreased appetite, and fatigue. Like Dr. Gedde’s patients, the subjects in this study experienced dramatic improvement. Dravet patients saw an average 53% decrease in seizures, and Lennox-Gastaut patients experienced an average 55% decrease.
Because of the DEA’s Schedule I classification of cannabis (which ironically puts it in a category classified as having no currently accepted medical treatment use in the U.S.), it has been challenging to do extensive testing. In the place of laboratory studies, there are hundreds of personal anecdotes about the effectiveness of cannabis in the treatment of epilepsy.
Medical cannabis has been used to treat neurological conditions, such as epilepsy, for years – often with dramatic effective results, and garnered worldwide attention in 2013 after the mainstream media focused on the remarkable story of Charlotte Figi: a child with a severe form of epilepsy known as Dravet Syndrome. At the age of 5, Charlotte was having nearly 300 seizures per day, and her family had been told there were no other treatment options that could help her any further. The family turned to cannabinoid therapy, giving her cannabis oil twice a day. Charlotte is now 8 years old and continues to thrive, with seizures happening only 2 to 3 times per month.
John Malanca, founder of United Patients Group commented “What Charlotte’s story did was give a worldwide platform to the truth behind the efficacy of medical cannabis for devastating conditions such as epilepsy. We are excited to lend our support and to help further enlighten the epilepsy community and others on the truth behind medical cannabis.”
During UPG’s Medical Cannabis Conference held in May of 2016, they featured a speaker who had a very personal experience with epilepsy, and braved the stigma and outright backlash to treat her two children – one with epilepsy and the other with autism. Rebecca Gonzalez is a mother who advocates strongly for medical marijuana and for the use of full plant cannabis extract for treating her children and others like them. Her story is can be seen in this interview we did with Rebecca following our conference here.
Continuing the fight for access to medical marijuana
To address the severe need for an effective epilepsy treatment, GW Pharmaceuticals is continuing their trials of Epidiolex. In 2015, the company launched their third round of trials that will test 150 patients during a period of 14 weeks in a Epidiolex vs placebo study. This is just another step in order to get approval from the FDA for the cannabidiol-based drug.
But with the Schedule I classification of cannabis still in place, this may be difficult. And parents such as Rebecca Gonzalez and others don’t have time to wait. Perhaps the most unfortunate aspect of the ban on medicinal marijuana at the federal level is that, with so few studies, dosing and prescribing cannabis is not yet an exact science. Many families must undergo a trial and error process to discover the correct administration, strain, and dosage. And just like with traditional medication, each treatment must be tailored to each patient, since every patient is unique in their medical needs.
However, Bonni Goldstein, MD, who is the director of Canna-Centers and a UPG Advisory Board member, prescribes CBD to children with epilepsy and suggests that there is little to lose and much to gain when trying CBD. Unlike traditional pharmaceuticals, cannabis has fewer and less severe side-effects. She notes that cannabis can have side effects of drowsiness and fatigue, but that traditional epilepsy medications sometimes come with more severe symptoms such as liver failure, vomiting, etc. She even goes so far as to say that the side effects of CBD are less severe than those of epilepsy itself.
This week, United Patients Group, along with the Utah Association for Responsible Cannabis Legislation will host an evening of information, education and awareness for lawmakers, healthcare providers, public safety officials and others at the University of Utah to focus on the science of cannabinoid medicine and how its positive effects can improve the health of Utahans. Dr. Goldstein will be one of several featured speakers who will address how cannabis has had a profound and positive effect on treating patients who suffer from epilepsy among other chronic conditions such as Autism, Alzheimer’s and opioid addiction.
Celebrating National Epilepsy Awareness Month
As supporters of cannabis in the treatment of epilepsy and educators on the medical benefits of cannabis for various illnesses, we at United Patients Group are proud to participate in National Epilepsy Awareness Month. We will continue to battle epilepsy with knowledge, science, and support of the most effective treatments available.
Epilepsy Information: Epilepsy and Medical Marijuana Treatments
Epilepsy is a brain disorder that causes people to have recurring seizures. The seizures happen when clusters of nerve cells, or neurons, in the brain send out the wrong signals. People may have strange sensations and emotions or behave strangely. They may have violent muscle spasms or lose consciousness.
There is a wealth of new scientific understanding regarding how medical marijuana or cannabis can be beneficial for treating Epilepsy.
Doctors use brain scans and other tests to diagnose epilepsy. It is important to start treatment right away. There is no cure for epilepsy, but medicines can control seizures for most people. When medicines are not working well, surgery or implanted devices such as vagus nerve stimulators may help. Special diets can help some children with epilepsy.