Study Weighs Medical Marijuana Benefits for Epilepsy Patients

The trials, being conducted at the Jacobs School of Medicine and Biomedical Sciences and at Women and Children’s Hospital of Buffalo (WCHOB), build upon observational studies showing cannabidiol (CBD), a non-psychoactive marijuana derivative, may reduce seizures in patients with certain forms of refractory pediatric epilepsy.

Researchers will investigate how well CBD works when given with existing medications to treat seizures associated with severe epilepsies in children with Lennox Gastaut Syndrome and Dravet Syndrome.  

“We are doing new studies that bring new hope,” Weinstock says.

For patients with severe epilepsy, Weinstock says, pharmaceutical-grade CBD is the best option, since it allows for accurate dose prescription and titration.

“There could be wide variability between medical marijuana obtained from different dispensaries or even between batches from a single dispensary, which could lead to a significant difference in the ratio content of THC to CBD,” he says.

“That’s why regulation in the content ratio of the different cannabinoids is needed for administration of medical marijuana to patients with refractory epilepsy.”

In 2015, an open-label study by New York University researchers and others found that cannabidiol add-on therapy had a promising efficacy and safety profile in children with severe epilepsy. Weinstock acknowledges the importance of such studies, in which both the researcher and the patient knew the treatment was being given.

“However, the clinical data are still quite sparse,” he says. “A randomized, double-blind, placebo-controlled clinical trial, which is what we are now doing, is the gold standard for providing the needed scientific data regarding the efficacy and safety of cannabidiol in children with severe epilepsy.”

In a randomized, placebo-controlled trial, some patients receive the drug and others receive the placebo, and neither patients nor clinicians are privy to information about which patients are receiving the real treatment. That is a standard practice in clinical trials and is especially important with medical marijuana, Weinstock says.

“There is a very strong placebo effect with medical marijuana, since parents are so invested in the idea that this is going to work,” he explains. “There’s still a gap between what patients and their caretakers believe and what we know scientifically.”

The trials are funded by GW Pharma, a British pharmaceutical company that makes Epidiolex, an oil that is 99 percent CBD.

Data gathered by these studies will be used to support an application to the Food and Drug Administration for approval of the drug.

New York State’s Compassionate Use program is also making Epidiolex available to 100 children and young adults statewide with treatment-resistant epilepsy. Twenty of them will be screened and treated at the UB and WCHOB sites.

Weinstock, who is medical director of the Child Neurology and Pediatric Epilepsy Program, is the site principal investigator on both clinical trials and sees patients through UBMD Neurology.