Clinical trials for cannabis: Doctors weigh in on Epidiolex

Bottles line the shelves at the lab of Dr. Eric Marsh, assistant professor of neurology and pediatrics and attending physician at the Children’s Hospital of Philadelphia.  (Photo by Calah Kelley/News21)
Bottles line the shelves inthe lab of Dr. Eric Marsh, assistant professor of neurology and pediatrics and attending physician at the Children’s Hospital of Philadelphia. (Photo by Calah Kelley/News21)

By Lex Talamo

PHOENIX – Parent advocates nationwide are clamoring for access to medical marijuana for children who suffer from severe and intractable disorders.

In response, the Food and Drug Administration authorized 450 children to participate in clinical trials involving Epidiolex, a marijuana-derived oil treatment that is high in cannabidiol, the non-psychoactive component of marijuana.

Early results from the ongoing clinical trials are mixed – some children appear to benefit from using Epidiolex while others do not – and some members of the medical community remain concerned about whether or not marijuana is a safe treatment for children.

Twenty-five children with Dravet or  Lennox-Gastaut syndromes recently participated in a clinical trial of Epidiolex at the Children’s Hospital of Philadelphia.

“We had lots of families who were interested, and we actually had to do a lottery to select the 25 patients,” said Dr. Eric Marsh, assistant professor of neurology and pediatrics and attending physician at the hospital.

A similar clinical trial took place at the Primary Children’s Hospital in Utah. Chief Medical Officer and Chair of Pediatrics Dr. Ed Clark said finding the 25 participants took three to four months.

Both clinical trials had similar results: More than half of the children who received the oil showed an average 50 percent reduction in seizures. A few children experienced minor side effects like drowsiness or diarrhea. Marsh and Clark, however, said researchers need to conduct further studies before they would consider Epidiolex a valid treatment option.

“One of the tensions is we want the best scientific evidence we can have versus what we can do for a child and family at the time,” Clark said. “We’re trying to balance risk with compassion. Balancing all the issues, our goal is to help children and their families. The child is first, always.”

The doctors said they were concerned about the small number of children in the trials and the parent-reported results.

“There was so much excitement about this, you have to be concerned about a large placebo effect,” Marsh said. “All the media hype and the social media hype and the individual hype really could skew results. Without the randomized, double-blind, controlled trials, it’s virtually impossible to know whether or not any medicine is working.”

Dr. Charles Cutler, vice president of the Pennsylvania Medical Society, agreed that anecdotal success stories can be misleading. Cutler cited multiple cases where parents reported that medical marijuana helped their child, but doctors and electroencephalograms – considered the gold standard in measuring seizure activity – still showed significant seizure activity.

“One of the most heartbreaking experiences for a doctor is to see a sick child,” Cutler said. “When I walk through the hospital floors and see children who are hospitalized, hooked up to an IV, receiving oxygen with prongs in their nose, my feelings are the same, I think, as every doctor. We want children to be healthy, but we simply can’t use medication that isn’t proven.”

Another complication is how little is known about epilepsy itself.

“Epilepsy is like a roller coaster,” Marsh said. “There are good months and bad months. Some of these kids would get better over time naturally, so (of those) who’ve responded, did they respond because of the drug or because they were going to get a little better anyway?”

Dr. John Knight, a pediatrician at Boston Hospital, encouraged parents to wait until doctors conduct more research and clinical trials.

“My heart goes out to parents,” Knight said. “The problem is that they may be trading away an immediate relief for their child’s future. (Marijuana) is untested and the scientific evidence is that it can be devastating to the developing brain.”

Steve Schultz, vice president of investor relations at GW Pharmaceuticals, the manufacturers of Epidiolex, said parents will have a consistent pharmaceutical medicine they can trust once Epidiolex gains FDA approval.

But until that happens, many medical experts urge parents to wait.

Lex Talamo is a Hearst Fellow. Follow her on Twitter @LexTalamo. Come back in August to see the full News21 report on “America’s Weed Rush.”