The results of a multi-center study led by researchers from UCSF Benioff Children’s Hospital San Francisco were announced on January 5th. The study indicated that children and young adults with severe forms of epilepsy who do not respond to conventional medical treatment have fewer seizures when treated with a purified cannabinoid.
The study results are significant. My conclusion is that the Food and Drug Administration is likely to approve the use of Epidiolex this year. If the FDA approves the use of Epidiolex, it will be a monumental event. It will be the first time that the FDA has approved any cannabis-derived pharmaceutical as a treatment.
While the preliminary results announced by UCSF are very positive, they potentially could have a devastating impact on all the states who have legalized medical marijuana, and to all growers, processors, extractors and dispensaries in America’s medical marijuana industry. It could also potentially deprive thousands of patients who have been relying on local sources of medical marijuana.
In announcing the results, Maria Roberto Cilio, MD, Ph.D., the senior author of the report and the director of research at the UCSF Pediatric Epilepsy Center stated, “Better treatment for children with uncontrolled seizures is desperately needed. It’s important to get seizure control at any age, but in children, uncontrolled seizures may impact brain and neurocognitive development, which can have an extraordinary effect on quality of life and contribute to progressive cognitive impairment.”
The researchers evaluated 162 children and young adults at 11 independent epilepsy centers across the United States. All of the children were treated with Epidiolex, a purified liquid cannabinoid that contained none of the psychoactive cannabinoid tetrahydrocannabinol (THC). The study took place over a 12-week period. The results indicated a median 36.5 percent reduction in monthly motor seizures, with a median monthly frequency of motor seizures decreasing from 30 motor seizures a month to under 16 over the period of the 12-week trial.
The patients in the trial were all between the ages of one and 30 with intractable epilepsies resistant to many if not all of the antiepileptic treatments. The trial included children with Dravet syndrome, a rare genetic disorder that manifests in early childhood with frequent, disabling seizures. These seizures often occur daily and can number into the hundreds, and the syndrome also can result in profound cognitive and social deficits.
Epidiolex is a liquid formulation of pure plant-derived Cannabidiol (CBD), formulated as a treatment for various orphan pediatric epilepsy syndromes by the UK’s GW Pharmaceuticals. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the treatment of Dravet syndrome and Lennox-Gastaut syndrome and has also granted Fast Track designation for Dravet syndrome.
UCSF Benioff Children’s Hospital San Francisco was the first site to administer Epidiolex to a child with epilepsy. In April 2013, the drug was first provided to a patient after obtaining a special approval from the U.S. Food and Drug Administration’s Investigational New Drug (IND) program. The preliminary results provided the framework for the current study. A second patient began taking Epidiolex at UCSF in July 2013, and in January 2014, UCSF and other centers started to enroll patients under an expanded access Investigational New Drug program.
In announcing the results, Cilia also stated, “This trial is pioneering a new treatment for children with the most severe epilepsies, for whom nothing else works. This is just the first step. This open-label study found that CBD both reduces the frequency of seizures and has an adequate safety profile in children and young adults. Randomized controlled trials are the next step to characterize the true efficacy and safety profile of this promising compound.”
Why are these successes a problem for America’s medical marijuana industry? It starts with Epidiolex itself. While Epidiolex is a proprietary formulation, it is essentially only pharmaceutical-grade, cannabis-derived CBD in liquid form, purified to remove all THC. While not manufactured to pharmaceutical standards, similar CBD-based formulations are available in many of the states that have legalized medical marijuana. While some have no THC, most have only minimal or trace amounts of THC. Some have no THC. Despite the position that GW Pharmaceuticals may take, there is nothing magical about Epidiolex as a plant-derived formulation.
So far the FDA has avoided regulation of marijuana-based medicine in the states that have approved it. While the FDA has the legal-basis for regulating medical marijuana, it is something that the agency has avoided, along with herbal-based medicines in general. It is clear that the FDA has a legal responsibility to regulate medical marijuana and all herbal medicines.
The threat to America’s medical marijuana industry is this. If the FDA approves Epidiolex, potentially two things could happen. Either the agency or GW Pharmaceuticals could start to force through regulatory action by the FDA or legal action by GW Pharmaceuticals, any other company from manufacturing or selling any CBD-based formulation for the same medical conditions. If either of these occur, it could be devastating to America’s emerging medical marijuana industry, compassionate care providers, and most importantly to thousands of patients who rely on local sources of CBD-based medicine.
Author: Jeffrey friedland (email@example.com, Tel. 1-970-779-4040)
Jeffrey Friedland is the author of “Marijuana: The World’s Most Misunderstood Plant,” which is available in print and Kindle editions at Amazon (www.amazon.com/author/jeffreyfriedland )