A Parent's Perspective: the LGS Epidiolex Study

GW Pharmaceuticals recently submitted a new drug application (NDA) to the U.S. Food and Drug Application (FDA) for the medication Epidiolex^®. This oil-based, 99% purified cannabidiol (CBD) extract from the cannabis plant allows for giving known known and consistent amounts of medication in each dose.

The following is an interview epilepsy.com Medical Cannabis Editor Anup Patel MD conducted with a parent of a child living with Lennox-Gastaut syndrome. The child participated in a placebo-controlled research trial of Epidiolex^®. The mother details what she thought about the research trial process and gives an update on how her daughter is doing now.

Tell us a little about your daughter and her Lennox-Gastaut syndrome.

My daughter developed seizures at 3 months of age. She had infantile spasms and was having clusters of 50 to 100 seizures daily. At 18 months, she was diagnosed with Aicardi Syndrome, a rare genetic disorder that included various seizure types that were difficult to control.

Her lifetime has been spent weaning her on and off antiepileptic medications and trying to find that right mixture of medications to provide some relief. Many medications would work for a while, but lost their efficacy over time. The combination of these medicines would make her tired and unable to concentrate. However, this was an improvement and we had to accept this form of treatment because we were out of options. We found ourselves waiting for new drugs to be FDA approved.

Why did you want to participate in the study on Epidiolex^®, a purified CBD?

I had heard in our Aicardi community that some families were trying an experimental drug that contained cannabidiol. They were having success. It provided their girls with better seizure control, without the side effects other medications can cause. I also liked the idea that it was a natural, plant-based medication.

Editor’s note: While derived from the cannabis plant, Epidiolex^® is a manufactured medication produced in a controlled setting to ensure consistent dosing. Unlike products available in state cannabis dispensaries, Epidiolex^® is subject to the same stringent safety testing that any medication receives. If approved by the FDA, it would only be available by prescription from a health care provider.

What was your experience in the study?

Our experience with the study has been very positive. The doctors, nurses, and pharmacist that provide the routine examinations and dispense the CBD have been very helpful, courteous, and respectful of our privacy. The whole process has been simple, from applying to be in the study, to reporting my daughter’s seizure count two years later. The research and study staff have been very attentive to my daughter’s needs and available 24/7 for any questions or concerns we have had.

Has the study medication helped your daughter?

Absolutely. Prior to the study, my daughter’s best control was 5 to 8 seizures per day and she usually had 50 to 100 seizures a day. In addition, we used emergency medications approximately 2 to 5 times a month to stop a seizure lasting longer than 10 minutes. This was no way for her to live her life. We were out of options.

I am happy to say that my daughter now has 2 to 5 seizures a week and some days where she is seizure free. Emergency medications are being used 2 to 3 times a year. In addition to improved seizure control, she is more alert and can concentrate on her surroundings. She has regained some lost milestones, such as sitting unassisted for a minute, using a switch toy to say, “turn the page” in a book, and, my personal favorite, smiling again!

Does she have any side effects in the study?

She has had some gastritis (inflammation of the stomach lining), but this has been manageable with reflux and stomach coating medications, along with taking the CBD with meals. In addition, she has had much more manageable and frequent bowel movements. This is a plus, as she has fought constipation all her life.

Would you do this study again or participate in another research study?

We would participate in the study again. It has been a very productive and positive experience, not only for my daughter, but for the entire family. Without a doubt, we would participate in another research study. The whole experience has been very positive and rewarding.

What do you hope to gain from being in this study?

We achieved what we set out to do: reduce my daughter’s seizures in number and in duration. Plus we have been able to reduce the amount of other antiepileptic medications she takes. We hope to stop one or more of the antiepileptic medicines she is currently prescribed.

Why is it important for people to participate in research studies?

It is extremely important that folks participate in research studies. By participating, the field of medicine advances. We gain the knowledge to treat and even cure some of the worst conditions and diseases. We must strive to give those affected individuals hope and a better quality of life.

What would it mean if this medication were to become FDA approved?

My first thought would be a sense of relief. Other individuals and families struggling with epilepsy would now have access to a more natural alternative to seizure control. One that has been proven effective for the toughest intractable epilepsy, with minimal side effects.