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Mouse and cell culture studies suggest cannabidiol or inhibiting GPR55 could help treat Dravet syndrome, a disease involving pediatric seizures and life-long autism-like behaviors caused by loss-of-function mutations in Nav1.1 (SCN1A). In a genetic mouse model of Dravet syndrome, cannabidiol increased the frequency of inhibitory post-synaptic currents in the hippocampus and decreased autism-like social interaction deficits compared with no treatment and vehicle. In ex vivo hippocampal slices from the model, cannabidiol or a GPR55 antagonist tool compound decreased action potential frequency and increased the frequency of inhibitory post-synaptic currents compared with vehicle. Next steps could include identifying and testing other GPR55 antagonists in mouse models of Dravet syndrome.

GW Pharmaceuticals plc has Epidiolex cannabidiol, an oral formulation of cannabidiol, in registration to treat pediatric epilepsy in Dravet syndrome, Lennox-Gastaut syndrome (LGS) and treatment-resistant epilepsy; Phase III testing to treat seizures associated with tuberous sclerosis complex; and Phase II testing to treat infantile spasms. GW Pharmaceuticals has other cannabidiol formulations in Phase II testing to treat glioma and schizophrenia with partner Otsuka Pharmaceutical Co. Ltd., and Phase I testing to treat neonatal hypoxic-ischemic encephalopathy...