The battle over medical marijuana, particularly when it involves children, has often been more about sentiment than science. But now researchers have results from the country’s first clinical trial using the drug for severe childhood epilepsy.
I met one of their success stories inside a ranch-style home in Pasadena. Her name is Grace Scout Rodriguez, and she looks like a typical three-year-old, wriggling around in her mom’s lap.
Her mother, Gloria, paused for a moment as we talked.
“Hold on, she’s having a little seizure,” she said.
“Right now?” I asked. I didn’t notice anything.
“Now.” But then it’s over, just like that. Grace started touching my microphone, trying to grab it. “Bah!” she yelled, all smiles again.
“They’re absence seizures, she doesn’t have them very often,” Gloria explained. “I can tell in her body, and her eyes blink really fast and her head drops down just a tiny bit.”
Grace has Dravet syndrome, a severe form of epilepsy related to a genetic mutation. She has several types of seizures, from the stereotypical clonic-tonic seizures, that involves convulsions, to these momentary episodes. Dravet syndrome is incurable, and sometimes children die when the seizures can’t be controlled.
“They call it ‘catastrophic,’ and I hate that word,” Gloria said. “It just sounds like you’re…doomed. And she’s not doomed. You see she’s happy, she’s all over the place, but I think that’s mostly due to Epidiolex.”
“It has to be,” her husband Ascencion chimed in.
“It has to be,” agreed Gloria.
Epidiolex is an experimental liquid medication made from cannabis. GW Pharmaceuticals manufactures it in England. The formulation contains no THC, the chemical in marijuana that causes a high. But it does contain a calibrated dose of cannabidiol, or CBD, which is believed to have anti-seizure properties.
“Coming from someone who’s never tried it recreationally or otherwise, I didn’t know what to think,” Gloria said. “But I just know that at 400 seizures a day, I thought, there’s just no way my baby can keep living like this.”
When Grace has a bad seizure, she has to be hospitalized and sometimes put on a ventilator. But Gloria said even smaller seizures are dangerous.
“Several times we thought she’d broken her nose,” Gloria said about the falls, “That’s why we don’t have a coffee table anymore, because she hit her head on it so many times. She had a little black eye once.”
But since Grace started taking Epidiolex last May, the frequency has gone down, her parents said.
During the three-month, double-blind, randomized trial, no one, including Grace’s doctor, parents or teenage sister, knew if Grace was getting Epidiolex or a placebo.
But her dad Ascension said he noticed a change right away.
“I knew it was the real deal because she was acting totally different,” he said. “She wasn’t ‘dropping’ as many times.”
Gloria said her daughter also became more focused. Grace still has developmental delays associated with Dravet syndrome, and knows only two dozen words, but she can interact more, and play more.
“Prior to the trial, she would look at us but not see us,” Gloria said. “We would talk to her and she would just gaze at us. There was really no life in her eyes, she was just like a little zombie on all this medication. And it’s almost like we met her for the first time when she started on this medication.”
After the trial finished, the family learned Grace had indeed been getting the actual drug.
She now takes it twice a day, mixed into yogurt or applesauce.
Dr. Angus Wilfong directs the Comprehensive Epilepsy Program at Texas Children’s Hospital. He said the clinical results from the trial for patients with Dravet syndrome were significant. The drug reduced severe seizures by 39 percent, compared to a reduction on placebo of 13 percent.
“In a medication trial for epilepsy, that’s really as good as any medicine ever is,” Wilfong says. “So the results were very strongly positive.”
Side effects were less severe than other epilepsy drugs, he added.
Wilfong has heard the stories and reports about desperate families moving to Colorado, or parents of children with severe epilepsy creating their own cannabis oils to give to their children. But Wilfong cautions that those remedies can be dangerous, even if the plant is marketed as high in CBD and low in THC.
“With Epidiolex, we know exactly how much CBD, we know that it’s only CBD and there’s no THC, we know there aren’t contaminants or pesticides in it. So I strongly encourage families to not use botanical formulations of medical marijuana.”
Last June, Governor Greg Abbott signed a bill to allow some patients in Texas access to cannabis oil, but only for a few conditions like epilepsy. The state still needs to set up the distribution system.
Dr. Wilfong says Epidiolex might just make it to the market first. New drugs need at least two successful clinical trials to get FDA approval. Grace was in the first one, and the second one is wrapping up soon, Wilfong said.