Cannabidiol Has Long-Term Dose-Dependent Efficacy That Does Not Impact Behavior or Depend on Type of SCNA1 Mutation


A pharmaceutical grade, highly purified cannabidiol (CBD) (Epidiolex; Greenwich Biosciences, CA) was recently approved and made available for treatment of patients with seizures related to Lennox-Gastaut syndrome (LGS) and patients with Dravet syndrome (DS). Several new study results, including Phase 3 and expanded access program (EAP), regarding this therapeutic were presented at the American Epilepsy Society Meeting in New Orleans, LA. 

 “In our EAP, we have provided treatment to over 1,500 children and have reassurance of long-term efficacy and safety,” commented Justin Gover, CEO of GW Pharmaceuticals.

In open-label extension studies, treatment effects of CBD were maintained through 72 weeks of exposure for both patients with DS and LGS, with efficacy beginning in the first 6 to 10 days of treatment. 

The relationship between seizure control and CBD level was examined in an open-label single-center study of 56 adults and 38 children with treatment refractory epilepsy treated with CBD. A linear correlation between dose and seizure control was seen in patients who had weight-based dosing of CBD that could be increased every 2 weeks by 5 mg per kg per day to a maximum dose of 50 mg per kg per day. Blood levels of CBD were measured regularly and a strong linear correlation between dose and CBD level was also observed in all participants. 

Concerns about the effect of CBD on behavior in children were also addressed in an open-label extension study of 35 children, age 3 to 19 years, with medically refractory epilepsy who received CBD as adjunctive treatment. Behavioral functioning was assessed with the Total Problems T-score and no significant changes from pretreatment baselines were observed.  

A study of 120 patients with Dravet syndrome identified the presence and type of mutation in the SCN1A channel that are found in the majority of patients with DS and found no difference in response to treatment between those with a truncating mutation (n = 61) or a missense mutation (n = 50). 


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