A number of publications have appeared recently reporting on the efficacy and safety of cannabidiol (CBD) as an add-on therapy, as its clinical trials are concluding and its use is extending.  This paper describes an “expanded access” program – that is, when the FDA allows access to a drug before approval.  This is of interest because it is more like the normal, clinical use of a drug, as opposed to the way it is administered during a formal controlled trial.

CBD was utilized in conjunction with a variety of other drugs, with the most common partners being clobazam (ex. Onfi) (66%); valproic acid (ex. Depakote) (43%); and levetiracetam (ex. Keppra) (34%).  At the first time-point of 12 weeks, the percentages of LGS/DS patients with ≥50%, ≥75%, and 100% reduction in major motor seizures were 53%, 23%, and 6%.  Numbers for reductions of total seizures were actually similar (46%, 26%, and 5%).  These levels of response remained quite stable throughout the study period (to week 96).  The most common adverse effects were sleepiness (30%) (especially when in combination with clobazam) and diarrhea (24%).  41% experienced “serious AE’s”, including convulsion (14%), status epilepticus (9%), pneumonia (5%), and fever (4%).

All of these results are quite similar to those found in the controlled clinical trials, and those now being reported from formal extension trials focused on Dravet patients.

Laux LC, Bebin EM, Checketts D, Chez M, Flamini R, Marsh ED, Miller I, Nichol K, Park Y, Segal E, Seltzer L, Szaflarski JP, Thiele EA, Weinstock A; CBD EAP study group. Long-term safety and efficacy of cannabidiol in children and adults with treatmentresistant Lennox-Gastaut syndrome or Dravet syndrome: Expanded access program results. Epilepsy Res. 2019 Mar 25;154:13-20. doi: 10.1016/j.eplepsyres.2019.03.015 PMID: 31022635.