Data suggests that treatment with cannabidiol is associated with other benefits besides a decrease in seizure frequency.
Eric Marsh, MD, PhD
Treatment with cannabidiol (CBD, Epidiolex; GW Pharmaceuticals) is associated with improvements in non-seizure related behavioral and quality of life measures in patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), according to survey data presented at the 2019 American Epilepsy Society Annual Meeting, December 6-10, 2019, in Baltimore, Maryland.1
In June 2018, the FDA approved CBD oral solution for the treatment of seizures associated with DS and LGS. It is the first FDA-approved drug indicated for the treatment of patients with DS, and the first approved drug to contain a purified drug substance derived from marijuana.2
Data previously presented at the 2019 American Academy of Neurology Annual Meeting in May showed that CBD reduced seizure occurrence by nearly 50% in patients with DS. "The children in this study had already tried an average of 4 epilepsy drugs with no success and at the time were taking an average of 3 additional drugs, so to have this measure of success with cannabidiol is a major victory," Ian Miller, MD, director, Epilepsy and Neurophysiology Program, Nicklaus Children’s Hospital, said in a statement in May.
In the current study, investigators led by Eric Marsh, MD, PhD, of Children’s Hospital of Philadelphia, assessed 76 patients across 4 programs at the Children’s Hospital of Philadelphia, Children’s Hospital of Pittsburgh, and Geisinger Medical Center who were enrolled in open-label, compassionate use, expanded access studies of Epidiolex. Changes in non-seizure outcomes were collected using qualitative surveys and a validated pediatric side effect questionnaire. Additionally, investigators documented the relationship between seizure response, behavior changes, and side effects. The study group featured 44 men and 32 women with a mean age range of 10.24 years, ranging between 2 and 20 years. On average, patients were on 2.5 concomitant medications (range, 1-3).
Data showed that after 3 months to 4 years of CBD treatment, 57 (75%) of the 76 enrolled patients reported positive non-seizure related changes via qualitative surveys and the side effect questionnaire. Among the improvements were reported of improved attention, better sleep, decreased drooling, improved mood, and improved fine and gross motor skills.
The results indicate that treatment with CBD has significant potential to affect more than just seizure outcomes. Larger scale studies using placebo-controlled cohorts are required to determine whether these outcomes are sustainable over the long-term.
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1. Hagopian SJ, Little K, Thomas D, et al. Is this the CBD? Qualitative results of positive non-seizure related outcomes in four expanded access programs. Presented at: 2019 American Epilepsy Society Annual Meeting. December 6-10, 2019; Baltimore, Maryland. Abstract 2.339.
2. FDA approves first drug comprised of an active ingredient derived from marijuana to treat rare, severe forms of epilepsy. FDA. June 25, 2018. fda.gov/news-events/press-announcements/fda-approves-first-drug-comprised-active-ingredient-derived-marijuana-treat-rare-severe-forms. Accessed: December 9, 2019.