Cannabidiol cuts seizures for half of those with rare pediatric epilepsies
Large study finds 50% reduction for those helped by add-on therapy
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Use of cannabidiol — a chemical from the hemp plant used for treating epilepsy — as an add-on treatment for individuals with developmental and epileptic encephalopathies (DEEs), including Dravet syndrome, leads to meaningful reductions in seizures for about half of patients, a new study has found.
“Adjunctive pharmaceutical-grade cannabidiol is associated with clinically meaningful seizure reduction in a substantial proportion of treated individuals,” the researchers wrote, noting that there’s “at least a 50% reduction in seizures” for half of those given the drug in addition to other therapies.
A smaller number of people with these rare pediatric epilepsies saw reductions of 75% or more, with some achieving “seizure freedom,” the team noted.
The researchers’ analysis, which involved dozens of previously published studies, generally found results consistent with those of earlier clinical trials. However, the scientists could not identify factors, such as disease type or age, that reliably affected the effectiveness of cannabidiol treatment.
“Future work should prioritize adequately powered, prospectively collected, syndrome-defined cohorts, standardized outcome reporting, and ideally individual-participant data sharing to better define clinically meaningful effect modifiers and refine patient selection,” the team wrote.
The study, “Cannabidiol Use in Developmental and Epileptic Encephalopathies: A Syndrome- and Age-Stratified Systematic Review and Meta-analysis,” was published in the journal CNS Drugs.Â
DEEs are a group of severe pediatric-onset epilepsies characterized by frequent, treatment-resistant seizures, along with a range of other developmental issues. People with DEEs typically experience learning problems, movement difficulties, and behavioral challenges.
Some of the most well-recognized DEEs are Dravet syndrome and Lennox-Gastaut syndrome (LGS), but there are many others, each with its own underlying genetic cause.
Treatment for DEEs usually involves a combination of different antiseizure medications, but many patients continue to experience seizures despite being on several different therapies.
Epidiolex, a cannabidiol therapy, approved in US
Pharmaceutical-grade cannabidiol or CBD, sold in the U.S. as Epidiolex and in Europe as Epidyolex, has emerged in recent years as a promising add-on therapy for treatment-resistant epilepsy. CBD is the major nonpsychoactive component of the cannabis plant.
Data from clinical trials involving people with Dravet and LGS, along with other real-world findings, show that CBD can help reduce seizure frequency. However, its use in a broader group of DEEs has not been systematically studied.
To address this knowledge gap, researchers in Brazil and Mexico conducted a review and analysis of 46 previously published studies examining outcomes among people with DEEs treated with CBD as an add-on to standard medications. The studies ranged from randomized controlled trials to compassionate use programs and other real-world analyses.
“By providing … estimates of CBD effectiveness and safety, this study aims to offer clinicians more precise evidence to guide treatment decisions in this highly vulnerable group,” the researchers wrote.
More than 2,500 people with DEEs were involved in the various trials and programs reviewed. Most were children with Dravet or LGS. Across the studies, most patients had highly drug-resistant epilepsy and were on multiple antiseizure medications, according to the authors.
Overall, half of the patients achieved at least a 50% reduction in seizure frequency after starting treatment with cannabidiol. Slightly more than one-quarter achieved at least a 75% reduction. A small proportion of the patients — about 6% in all — became entirely seizure-free.
In studies that looked only at people with Dravet, 41% had at least a 50% seizure reduction, while 5% were estimated to be seizure-free. These findings are consistent with Dravet clinical trials, the team noted.
More research needed on other factors affecting treatment
The scientists also examined whether factors such as age, disease type, dose, or the simultaneous use of clobazam —an anticonvulsant therapy approved for use in LGS — might influence medication efficacy.
While some early analyses suggested such factors might play a role, the data were highly variable. Ultimately, the researchers couldn’t confirm a consistent or robust relationship between CBD’s effectiveness and other factors.
“Our results support CBD as a potential treatment option across DEEs, but do not justify preferential expectations of benefit according to syndrome, age, dose stratum, treatment duration, or clobazam co-medication.”
The scientists noted that more studies are needed to accurately define which factors influence CBD effectiveness in order to inform patient selection for treatment.
Side effects of cannabidiol were generally consistent across studies, with common ones including drowsiness, decreased appetite, diarrhea, fatigue, and behavioral changes. Most of these were mild or moderate in severity and could be managed with a dose adjustment.
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