Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome

Among children and young adults with the Dravet syndrome, a developmental disorder that is associated with treatment-resistant seizures, cannabidiol reduced the frequency of convulsive seizures but caused sleepiness and elevated liver enzymes in some patients. Seizures are difficult to control in the Dravet syndrome, a rare genetic form of epileptic encephalopathy primarily due to loss-of-function mutations in the SCN1A gene. Interest in cannabidiol for the treatment of epilepsy was generated by media reports of efficacy in children with the Dravet syndrome. 1 Four small trials of cannabidiol had yielded mixed results. 2 – 5 A series of in vitro and in vivo preclinical models of seizure showed that cannabidiol had activity against convulsive seizures. 6 Subsequently, the safety and effectiveness of a standardized oral solution of cannabidiol was tested in an open-label trial involving 214 children and young adults . . .