Marijuana drug shows promise for rare seizure disorder

A new study shows promising results in treating patients suffering a rare seizure disorder with cannabidiol, a non-psychoactive cannabis compound.

Conducted by researchers in the U.S., England and Australia, the study is one of the few peer-reviewed, scientific works to evaluate the use of cannabis compounds in treating seizure disorders.

The article, “Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet syndrome,” was published Wednesday in The New England Journal of Medicine.

Dravet syndrome is a rare form of epilepsy that usually presents in infants between 5 and 6 months old. It causes frequent, prolonged seizures that can lead to developmental issues, requiring constant supervision and care for the child.

There is no specific or Food and Drug Administration-approved treatment for Dravet syndrome despite anecdotal stories of cannabis products treating other epilepsy disorders.

Patients rely on a cocktail of anti-convulsive medications and, in some instances, a diet high in fats and low in carbohydrates.

In the study, patients who were put on a cannabidiol treatment plan showed a 50 percent decrease in seizures.

For Nicole Villas, whose son suffers from Dravet syndrome, the study is exciting not only for the reduced number of seizures but also for the spotlight it puts on the syndrome and the challenges faced by the patients and their families.

“As a mother, the challenges are it’s a rare syndrome, so nobody understands when you say your child has a severe form of epilepsy, nobody understands how all-encompassing that is,” said Ms. Villas, scientific director of the Dravet Syndrome Foundation. “So you end up having to educate nurses, schools, therapists, pediatricians, pretty much everybody, sometimes even your pediatric neurologist. You end up becoming an expert on something that nobody else is, and that’s tough. That’s challenging.”

Dr. Elaine Wirrell, a co-participant and principal investigator in the study at the Mayo Clinic, said that its “modest” findings offer at least one more option for patients and families hit by the disease.

“Even though it did reduce seizure burden, it did not stop the seizures completely, and so you still have that uncertainty: When is that next seizure coming? When is the next prolonged seizure coming?” said Dr. Wirrell, a pediatric epileptologist. “We haven’t by any means fixed this, but it certainly gives us one more option.”

The study was conducted at 23 centers in the U.S. and Europe with 108 patients undergoing randomized selection to receive either the cannabidiol treatment or a placebo. Fifty-two patients completed the cannabidiol treatment and 56 followed through with the placebo.

The patients ranged in age from 2 to 18 and had a baseline of 13 seizures per month.

Researchers concluded that the frequency of seizures for the cannabidiol group decreased by half, from 12.4 to 5.9. The placebo group had a slight decrease, from 14.9 to 14.1.

“The percentage of patients who became seizure-free was 5 percent with cannabidiol and 0 percent with placebo,” the researchers wrote.

Side effects of cannabidiol treatment included diarrhea, vomiting, fatigue, fever, sleepiness and “abnormal results on liver-function tests,” but some of these issues were resolved when dosage was adjusted, the researchers noted.

The study focused on the efficacy of Epidiolex, an oral cannabidiol solution from GW Pharmaceuticals. The British drugmaker was the primary funding source for the study and responsible for the trial design.

“The study we’ve conducted and the data generated are specific to this product,” GW Pharmaceuticals CEO Justin Gover told The Washington Times. “It’s not a study of medical marijuana, it’s not a study of sorts of CBD oils, which contain various other things. This is a very specific product which has been made to FDA standards and purity to ensure that it is a specific active ingredient in a pharmaceutical form which has been studied.”

The company hopes to have FDA approval for the drug and its availability for prescription by 2018.