The section chief of pediatric neurology at Nationwide Children

Recently, long-term data from an open-label extension trial of cannabidiol (CBD; Epidiolex, GW Pharmaceuticals) in patients with Lennox-Gastaut Syndrome (LGS) showed that the therapy remains well-tolerated through approximately 4 years of use—a valuable finding for a medication with such a highly publicized FDA approval.

The study revealed maintained reductions in drop and total seizure frequency through 72 weeks of exposure, as well as a high proportion of improvement in the overall condition of the patients, as reported by the patients themselves and their caregivers.

To discuss the meaning of these findings further, Anup D. Patel, MD, the section chief of pediatric neurology at Nationwide Children’s Hospital and an associate professor of clinical pediatrics and neurology at The Ohio State University College of Medicine spoke to 

: What new information was gleaned from this long-term, open-label extension study?

 Now we have long-term data to say, “Hey, if you're going to prescribe this medication for your patients, you can expect to see continued efficacy over time. You're not going see a drop-off. It's going to continue to work, and, more importantly, the adverse effect profile you see early on will stay the same later on. You're not going to learn anything new or have to watch out for anything new.”

Obviously, more data on that is going to be needed, so we're going to continue to collect data moving forward so if there is any new information that changes our thoughts on this, we'll be sure to address it. One of the things we saw similar to the other studies was that there are patients who can have an increase in their liver enzymes. But, in the majority—and in the case of our study, all but 3&mdash;of those results went back down to normal. They went back down to normal by doing 1 of 3 things: either reducing your CBD medication or stopping it; reducing or stopping your concomitant valproic acid; or actually doing nothing. When you do nothing some of those patients also self-resolve. That's an important thing that we learned, as far as long-term data, that we didn't quite know as it relates to the short-term data.

Were any of the data surprising or unexpected in any way?

It was interesting—but I think expected for somebody who does pediatric epilepsy&mdash;that fever was a common adverse effect that was reported. That's important for me to explain because fever is a very common occurrence in all pediatric patients. For anybody who has kids, it's very rare to go 1 year without their child having a fever, so I think it was more related to that than associated with the medication. I don't want people to be confused by that. But one of the things we saw that was similar to the other studies was tiredness remained as the No. 1 reported adverse effect.

Ultimately, what does this study mean for the epilepsy community?

What we now have seen, and demonstrated, is that this could be a treatment for patients with Lennox-Gastaut syndrome and Dravet syndrome, and that efficacy and safety can be maintained over time. Those are the most important points that I want to convey from this and it's reassuring to me that we saw that.

What is the potential impact of this therapy moving forward?

I think it's really important to have a new therapy for patients with Lennox-Gastaut syndrome. Lennox-Gastaut syndrome is a very difficult to treat epilepsy syndrome. There are FDA-approved treatments currently available, but many patients often fail on these medications, so having more treatment options is always of great benefit.

Most importantly, this medication works differently than any other medication for seizures that's been FDA-approved previously. That's exciting from a clinician standpoint and a scientist standpoint because we're tackling the same problem, but from a different angle, so perhaps there will be a better option when you have failed other medications used for Lennox-Gastaut syndrome. It gives patients and their families the most important thing that we can give them: hope.

What’s next for CBD—do you envision it being successful elsewhere?

Right now, the trial on tuberous sclerosis is looking at end points with Epidiolex—that enrollment has closed so I'm excited to see what those data points show. I'm not involved with those studies, but that's something that interests me. The sky's the limit&mdash;I'm really excited, now that this product is FDA-approved, about where it can be studied using scientific methodology that we similarly used. But because it's rescheduled that and now available via prescription, the regulations and hurdles and for regulatory purposes and research will, hopefully, be lessened because of our work. I'm hopeful that we can finally answer the questions of, “Does this product this molecule work for other disease states?” But the jury is still out, so obviously we'll wait and see.

Patel A, Gil-Nagel A, Chin R, et al. Long-term safety and efficacy of add-on cannabidiol (CBD) treatment in patients with Lennox-Gastaut Syndrome (LGS) in an open-label extension (OLE) trial (GWPCARE5). Presented at: American Epilepsy Society Annual Meeting; New Orleans, Louisiana; November 30 to December 4, 2018. aesnet.org/meetings_events/annual_meeting_abstracts/view/500065

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