Cannabidiol: Helping Kids with Hard-to-Treat Epilepsy

An international study my colleagues and I published today in the New England Journal of Medicine showed that cannabidiol can lead to a significant reduction in the number of seizures for patients with Lennox-Gastaut syndrome, a rare and difficult to treat form of epilepsy. A small percentage of children in the study treated with cannabidiol actually became seizure-free. If you are not familiar with epilepsy research, this might lead you to ask a few questions.

What Is Cannabidiol?

Cannabidiol, also known as CBD, is a purified derivative from the cannabis plant that has none of its psychoactive properties (properties that people feel high).

Cannabis? Is This Medical Marijuana?

No. It is a pharmaceutical formulation of pure, plant-based cannabidiol (CBD) and lacks the high associated with marijuana.

Desperate families, knowing of the potential of cannabidiol, sometimes try to obtain it on their own. There is no guarantee that a product labeled “cannabidiol” and sold on the streets is safe, or even contains what it claims.

What Is Lennox-Gastaut Syndrome?

Children with Lennox-Gastaut syndrome (LGS) can have hundreds of seizures every day. This rare, complex form of epilepsy often does not respond to epilepsy medication. The children in our study failed many treatments to stop their seizures in the past, so there was a significant need for something that could help them.

So What Did the Study Find Exactly?

The randomized, double-blind, placebo-controlled trial enrolled 225 patients with Lennox-Gastaut syndrome overall, including 25 at Nationwide Children’s. They were given different doses of a cannabidiol solution and one third of the patients – a control group – were given a placebo.

Of the patients who received the highest dose of cannabidiol, 39 percent of patients saw their number of seizures reduced by at least half. Thirty-six percent of patients received 10 mg/kg dose and had the same result. A total of 14 percent of patients in the placebo group had their number of seizures cut by half or more, demonstrating a significant difference between patients who received cannabidiol and those who did not.

So Does Cannabidiol Only Help Patients with Lennox-Gastaut Syndrome?

Nationwide Children’s also participated in a separate study published in 2017 in the New England Journal of Medicine that found cannabidiol can reduce seizures in Dravet syndrome as well, another complex childhood epilepsy disorder.  Also, we participated in an open label study published in Lancet neurology treating other forms of epilepsy as well. 

If My Child Has Lennox-Gastaut or Dravet Syndrome, What Do I Need to Know About the FDA Approval Process?

An advisory committee of the U.S. Food and Drug Administration in April recommended that this cannabidiol drug (called Epidiolex) be approved to treat seizures associated with both Lennox-Gastaut and Dravet syndromes. If the FDA follows the panel’s recommendation, Epidiolex will be the first non-synthetic, plant based, marijuana-derived product approved for prescription in the United States. The FDA has announced that it will make its decision this summer.

This medication has gone through the same rigorous research process that every other medication in the United States does. My fellow researchers and I wanted to give these families an answer, and we wanted to do it in a safe and proper way.