According to the American Epilepsy Society, approximately 2.2 to 3 million individuals in the U.S. have epilepsy and it affects more children than adults. Furthermore, many suffer from a type of seizure disorder that is untreatable with traditional medications, and is often related to severe morbidity and increased mortality. There has been much interest in cannabis as a means of providing an alternative form of medicine in treating epilepsy. Data supporting the medicinal benefits of cannabis, and more specifically, the role that cannabidiol (CBD) could play in treating treatment-resistant convulsive and non-convulsive epilepsy, such as Dravet syndrome, Lennox-Gastaut syndrome in children is gradually increasing. Additionally, studies have pointed to the safety, effectiveness, and tolerability of CBD, and today, human clinical trials of CBD products such as Epidiolex, is validating early findings related to the potential of CBD as an epilepsy treatment.
Cannabidiol (CBD) Product Epidiolex May Treat Dravet Syndrome and Lennox-Gastaut Syndrome
Dravet syndrome is a rare, horrible, and enduring form of epilepsy that surfaces in the first year of a child’s life, and is characterized by frequent and or continued seizures. Dravet Syndrome affects 1:15,700 individuals, 80% of whom have a mutation in their SCN1A gene , and features a number of symptoms including, developmental delay and movement and balance issues, orthopedic conditions, delayed language and speech issues, and sleeping difficulties, among other symptoms.
In March of 2016, British-based GW Pharmaceuticals announced the positive findings of its pivotal Phase III trial on cannabinoid-based investigational drug, Epidiolex, in treating children with Dravet syndrome. The Phase 3 study randomized 120 patients into two arms, Epidiolex 20mg/kg/day (n=61) and placebo (n=59) during a 14-week treatment period, and the results showed that Epidiolex, a cannabidiol-based drug, achieved a median reduction in monthly convulsive seizures of 39 percent. Epidiolex was shown to significantly decrease the frequency of convulsive seizures with minimum adverse effects.
In another GW Pharmaceuticals Phase 3 clinical trial of Epidiolex for the treatment of seizures associated with Lennox-Gastaut syndrome, a rare and typically debilitating form of childhood-onset epilepsy, Epidiolex was added to the patient’s current treatment. During the trial, patients taking 20mg Epidiolex per day achieved a 42 percent median reduction in seizures monthly, and patients taking 10mg Epidiolex per day achieved a 37 percent median reduction of seizures monthly.
Cannabidiol Open-label Interventional Trial Across the U.S
In the U.S, medical cannabis in the form of CBD oil is of growing interest to researchers and patients seeking an alternative and natural way to treat their intractable epilepsy. Several states have adopted CBD-only laws and research into the medicinal benefits of CBD are being conducted to date. These new findings are going a long way to validate early animal studies.
In an open-label trial conducted between Jan 15, 2014 and Jan 15, 2015, patients aged 1-30 years with severe, treatment-resistant epilepsy, were enrolled in an expanded-access program at 11 epilepsy centers across the USA, and each patient was given oral cannabidiol at 2-5 mg/kg per day, and the medicine was gradually increased until intolerance or to a maximum dose of 25 mg/kg or 50 mg/kg per day. The trial served to establish the safety and tolerability of cannabidiol, along with its efficacy. The results showed that adverse events were reported with more than 10% of patients experienced somnolence (25%), decreased appetite (19%), diarrhea (19%), fatigue (13%), convulsion (11%), and (6%) epilepticus- a serious adverse effect; additionally, the median reduction in monthly motor seizures was 36.5% (IQR 0-64.7). These findings support the claim that CBD could reduce the frequency of seizures, and is adequately safe for use in children as well as adults.
Treatment-resistant epilepsy affects a significant segment of the population and one of the most vulnerable groups in society-children. There are different forms of epilepsy, including rare genetic conditions and those that are difficult to treat with traditional medication. Cannabidiol (CBD) continues to be the new source of potential epilepsy treatment that researchers are studying in hopes of leveraging it for its medicinal properties. In trials, CBD had been shown to significantly reduce the frequency of seizures and has a safety profile that researchers generally deems acceptable. GW Pharmaceuticals is paving the way with the first of its kind cannabidiol medication-Epidiolex, and researchers are still pursuing human trials into CBD as a potential treatment for epilepsy.