I had heard in our Aicardi community that some families were trying an experimental drug that contained cannabidiol. They were having success. It provided their girls with better seizure control, without the side effects other medications can cause. I also liked the idea that it was a natural, plant-based medication.
GW Pharmaceuticals recently submitted a new drug application (NDA) to the U.S. Food and Drug Application (FDA) for the medication Epidiolex ® . This oil-based, 99% purified cannabidiol (CBD) extract from the cannabis plant allows for giving known known and consistent amounts of medication in each dose.
What was your experience in the study?
Editor’s note: While derived from the cannabis plant, Epidiolex ® is a manufactured medication produced in a controlled setting to ensure consistent dosing. Unlike products available in state cannabis dispensaries, Epidiolex ® is subject to the same stringent safety testing that any medication receives. If approved by the FDA, it would only be available by prescription from a health care provider.
We would participate in the study again. It has been a very productive and positive experience, not only for my daughter, but for the entire family. Without a doubt, we would participate in another research study. The whole experience has been very positive and rewarding.
It is extremely important that folks participate in research studies. By participating, the field of medicine advances. We gain the knowledge to treat and even cure some of the worst conditions and diseases. We must strive to give those affected individuals hope and a better quality of life.
Safety data were made available on 313 patients (261 patients with 12 weeks treatment effect data plus 52 additional patients for whom 12 week treatment effect data are not yet available or who withdrew from treatment) and represent approximately 180 patient-years of exposure to Epidiolex.
Safety and efficacy data on 10 patients diagnosed with TSC from the expanded access program were presented by Massachusetts General Hospital for Children (Geffrey et al) on CBD treatment of refractory epilepsy in these patients. In this poster, there was a response rate of 50%, 50%, 40%, 60% and 66% at 2, 3, 6, 9, and 12 months of treatment with Epidiolex, respectively. Improvements were reported in alertness, verbal capacity/communication, vocalizations, cognitive availability, and initiation of emotional and physical connections, as well as heightened expression of emotion. Side effects were seen in 50% of patients (5) and most were resolved with anti-epileptic drug or CBD dose adjustment.
Data were presented on 44 patients with Dravet syndrome who had at least 12 weeks treatment data. Of these 44 patients, 25 patients had also reached 36 weeks treatment at the time of data analysis. Information collected on all seizures (convulsive and non-convulsive) is reported for each patient. Data are presented showing the median change in seizure frequency compared to a 4-week baseline period.
Clinical Effect Data – all patients in Epidiolex expanded access program 1
“It is reassuring that this Epidiolex data on almost twice the number of patients from the last presentation further reinforces and supports the encouraging signals of efficacy and safety reported previously. Specifically, in the patient populations that GW is targeting for development, Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex , Epidiolex treatment was associated with meaningful reductions in seizures as well as a sustained response,” stated
Data were presented on all 261 patients who had at least 12 weeks treatment. Treatment was open label. Of these 261 patients, 135 patients had also reached 36 weeks treatment at the time of data analysis. Information collected on all seizures (convulsive and non-convulsive) is reported for each patient. Data are presented showing the median change in seizure frequency compared to a 4-week baseline period.
Clinical Effect Data – Epileptic Spasms 3
In all three trials that followed — GWPCARE1 (NCT02091375), GWPCARE3 (NCT02224560) and GWPCARE4 (NCT02224690) — Epidiolex showed higher effectiveness in reducing the prevalence of seizures by 17-23% than a placebo, when administered in combination with conventional anti-epileptic drugs in children older than 2 years.
“These trials revealed CBD’s efficacy in reducing the frequency of the primary seizure type over a 14-week period compared with placebo, providing compelling evidence for the role of pure, plant-derived CBD in the management of DS and LGS,” the researchers said.
Researchers in this study reviewed the main findings of these trials regarding the safety, efficacy, and tolerability of Epidiolex for the treatment of children with Dravet and Lennox-Gastaut syndromes.
“The frequency and severity of seizures have profound impacts on quality of life, risk for injury (eg, convulsive seizures in [Dravet syndrome], drop seizures in [Lennox-Gastaut syndrome]), health care use, and increased risk for mortality,” the researchers wrote.
Dravet and Lennox-Gastaut syndromes are two rare medical conditions characterized by severe recurrent seizures that fail to respond to treatment with conventional anti-epileptic drugs.
Previous clinical trials have found that Epidiolex effectively reduces the frequency of epileptic seizures when used in combination with other anti-epileptic medications.
Common side effects associated with Epidiolex included somnolence, diarrhea, vomiting, decreased appetite/weight loss, and elevated hepatic transaminases (high levels of liver enzymes that might indicate liver damage).