One of the most painfully frustrating events in our practice of Neurology since the times of residency training has been the encounter with a refractory Epilepsy syndrome in a young patient. Not only we witness the suffering of the disgraced patients, who are losing intellectual capacity and the promise of a fruitful future, but also the terrible frustration of their parents and loved ones. Lenox-Gastaut syndrome is exactly that: an epilepsy variant that attacks young patients with “drop seizures” that are resilient to effective, long-term and non-toxic pharmacologic treatment.
A multi-center study that enrolled 171 patients form 24 clinical centers form the United States, the Netherlands and Poland studied the effects of the administration of Epidiolex, a CBD purified drug that does not contain any of the psychoactive components of Cannabis. Patients from the age of 2 and 55 years old who had failed to show improvement with at least two anti-epileptic drugs were eligible to participate; their mean age was 15 years old with six drug failures. They all had to have slow (less than 3 hertz) spike and wave patterns in their electroencephalogram, more than one type of seizure for at least six months and at least two drop seizures per week.
The participants were randomized to receive 20 mg/kg purified CBD oil daily (divided twice daily) or a matched placebo for 14 weeks. Their families actively participated as they helped them record the number of drop seizures and related events during the study. They were evaluated four times in their respective medical clinics and twice with a special telephone interview. The investigators studied the percentage change of drop seizures during the treatment period of 86 patients that received the drug and the 85 patients who were part of the placebo group. We must point out that all patients continued to receive their properly prescribed anti-epileptic treatment.
The median percent reduction in monthly drop seizures from baseline to the end of the study was 43.9% in the group receiving Epidiolex compared to 21.8% in the placebo group. Some patients receiving the drug even had a 50% reduction in seizures and three patients were even seizure-free during the whole study; nobody in the placebo group had a seizure-free period. There were some adverse events in both groups, including diarrhea, fatigue, fever and vomiting. Amongst the noted drug interactions, the patients taking Clobazam had an increase in its active metabolite, which led to higher sedation. Thirty-six treated patients that were also taking Valproic Acid had an increase in liver enzymes, which resolved after the CBD was stopped.
After the study was stopped in October 2015, an open-label extension study was started.
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