AAN 2026 | Cannabidiol effective across genetic epilepsies beyond Lennox-Gastaut syndrome

So, as you know, there were two trials looking at purified CBD in treating Lennox-Gastaut patients. Both of those trials were positive and led to the FDA approval in the United States and EU for CBD and the treatment of seizures and those disorders. Kind of since those trials were done, there’s been more and more of a focus on what are now called DEEs, or developmental and epileptic encephalopathies, as now we have over 1,000 genes associated with epilepsy. And we realize that many of those mutations in those genes can lead a person to not only have significant epilepsy, but significant cognitive impairment and other issues. So going back to an LGS trial, where, you know, LGS is an epilepsy syndrome caused by many, many different etiologies, we knew that it was very likely that many of the patients in that trial actually had DEEs, genetic DEEs, and thought it was important to go back and see that. So we were able to identify over 50 patients who enrolled in the LGS trial who did have pathogenic mutations in other genes, and we were able to show that those genes were associated with various DEs. I think there were close to over 30 different genes identified, showing that, yes, CBD can be effective in treating patients with Lennox-Gastaut. It also clearly is effective in treating DEEs, which is really important with this changing view of treating this group of epilepsy, because we’re kind of moving away from Lennox-Gastaut to the more specific etiologies and treating DEEs. And again, we saw that the efficacy with regard to seizure control was very similar to the overall LGS population. And also the tolerability safety profile in those with genetic DEEs in the cohort was also similar to the LGS trial. So I think it was really useful analysis to do. As you know, there’s an ongoing trial now, a basket trial, first basket trial in epilepsy, enrolling patients with different DEEs. So this kind of shows that really, in some ways, every LGS trial has been a basket trial.

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