Prescription CBD Shows Promise for Rare Epilepsies Beyond Its Approved Uses

CBD reduced seizure frequency in at least one patient across 47 of 57 studies, spanning 37 different developmental and epileptic encephalopathies (DEEs) and treatment-resistant epilepsies (TREs). Twenty-two studies reported at least one patient seizure-free for 48+ days. Most common adverse effects were gastrointestinal.

Systematic literature review per PRISMA guidelines searching Embase, PubMed, and Cochrane through March 2024. Evaluated studies reporting CBD effectiveness and tolerability in DEEs/TREs beyond Lennox-Gastaut, Dravet, and tuberous sclerosis (the approved indications). 57 studies included, covering 971 patients.

Epidiolex is only approved for three specific epilepsies, but many patients with other rare seizure disorders lack effective treatment options. This review suggests CBD may help a broader range of epilepsy patients than currently indicated.

With over 37 rare epilepsy types showing potential benefit, this review supports clinical investigation of CBD across the broader spectrum of treatment-resistant epilepsies. For families dealing with rare epilepsies, this offers hope that approved treatment options may eventually expand.

Most studies (33/57) were case reports or small case series — the lowest evidence tier. Heterogeneous study designs and outcome definitions. No placebo-controlled data for most conditions. Publication bias likely favors positive results.