Nonpsychoactive Cannabidiol Helps Children with Severe Epilepsy

VBCN - May 2015 Volume 2, No 1

Washington, DC—Patients with treatment-resistant epilepsy may benefit from a treatment regimen that includes cannabidiol (Epidiolex), a compound in medical marijuana that does not contain psychoactive properties. According to results of a multicenter study presented at the 2015 annual meeting of the American Academy of Neurology, more than half of all patients who received cannabidiol experienced a 50% reduction in seizures.

“Cannabidiol showed reductions in seizure frequency across multiple drug-resistant epilepsy syndromes and seizure types and was generally well-tolerated in this open-label cohort,” said Matthew Wong, MD, Assistant Professor of Neurology at Wake Forest School of Medicine, Winston-Salem, NC.

According to the Centers for Disease Control and Prevention, approximately 5.1 million individuals in the United States have been diagnosed with epilepsy. About one-third of these patients experience seizures that cannot be controlled by medication. Severe forms of epilepsy, such as Lennox-Gastaut syndrome and Dravet syndrome, are marked by frequent, treatment-resistant seizures.

Cannabidiol is the most abundant nonpsychoactive component of Cannabis sativa and has shown anticonvulsant activity in preclinical models of epilepsy, independent of activity at known endogenous cannabinoid receptors, Dr Wong said. Anecdotal reports suggest that cannabidiol may be effective in treating children with treatment-resistant epilepsies, in particular Dravet syndrome, but the compound has rarely been studied in formal clinical trials.

The new study included 213 patients aged 2 to 26 years (median age, 11 years), and was conducted at 11 US centers that were granted FDA-approved open-label expanded access. To qualify for this study, patients had to have an early-onset, treatment-resistant epileptic condition, such as Lennox-Gastaut or Dravet syndrome.

Patients first entered into a 4-week, baseline phase where caregivers tracked countable motor seizures as well as other seizure types. After 4 weeks, patients received a 99% pure, oil-based extract of cannabidiol, which was titrated until intolerance to a maximum daily dose of 25 mg/kg.

“All patients were prescribed cannabidiol in a liquid daily dose that was gradually increased up to a potential maximum of 25 mg/kg for 12 weeks,” explained Dr Wong. “Seizures were recorded as convulsive (countable) or nonconvulsive. Atonic seizures were also specifically recorded.”

Study Results


Of the 137 patients who received at least 12 weeks of treatment, the number of seizures was reduced by a median of 54% during the 12-week period in all patients; the number of seizures in patients with Dravet syndrome decreased by 63%. A 50% responder analysis demonstrated that 51% of patients who completed the study had at least a 50% decrease in seizures. Dr Wong noted that at 3 months, 9% of all patients were seizure-free; 16% of patients with Dravet syndrome were seizure-free. In addition, there was a 66.7% decrease in the number of atonic seizures.

Surveillance laboratory tests were performed to identify problems with liver and kidney function, as well as anti­epileptic drug levels. The most common adverse events that occurred in more than 10% of patients who received cannabidiol included somnolence (21%), diarrhea (17%), fatigue (17%), and decreased appetite (16%). Serious adverse events occurred in 52 patients; 22 patients experienced serious adverse events that were possibly related to cannabidiol use, the most common of which included status epilepticus (N = 10) and diarrhea (N = 3). Overall, 10 (5%) patients discontinued the use of cannabidiol as a result of adverse events.

Future research with cannabidiol will include randomized, placebo-controlled clinical trials, Dr Wong said.

“Treatment with cannabidiol is associated with a meaningful reduction in seizure frequency in patients with severe treatment-resistant epilepsy,” Dr Wong said. “The tolerability also seemed good, with very few withdrawals due to adverse events.”

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