Kevin Reece, KHOU 11 News - Izaiah Ruiz can suffer a half dozen grand mal seizures a month. Lesser seizures happen daily and can be caused by changes in temperature, even changes in his mood.
Now at the age of 6, with the developmental age of just a 2-year-old, his next hope for a halt to his physical and cognitive decline might be a derivative of marijuana.
Izaiah, who lives with his grandparents in Montgomery County, enrolled Friday in the first worldwide cannabis trial to treat epilepsy. He suffers from Dravet Syndrome, a rare and catastrophic form of epilepsy that begins in infancy. Texas Children's Hospital, one of the largest epilepsy centers in the country, has been chosen as one of 10 sites for the first clinical trial of a marijuana-based drug called Epidiolex, a highly purified extract of the cannabis plant containing pure cannabidiol or CBD. Manufactured by the British company GW Pharmaceuticals (the only company that produces pure plant-derived CBD) it does not contain THC - tetrahydrocannabinol the psychoactive ingredient in marijuana.
"There is a desperate need for new treatments for epilepsy," said Dr. Angus Wilfong, Texas Children's Hospital pediatric neurologist and principal investigator for the study. "We need new treatments, new tools, to treat uncontrolled epilepsy. And we're really hopeful that CBD could be that new treatment that is really going to change people's lives."
"A chance to live to be a teenager. A chance to live to be a grown man," said Izaiah's grandmother Lori Fountain. "It might give my little boy a chance of a life. He might be able to outside and play and slide down a slide, and be on a swingset and ride on a tractor. Get to play like a normal little boy."
All of the initial 30 patients in the study also have Dravet. The first phase of the study will help determine the proper dosage of CBD. A second phase will include 90 participants at 50 centers like Texas Children's Hospital worldwide where a placebo-controlled study will help determine if the drug does help reduce or eliminate epileptic seizures.
"It's important for people to understand that we're not giving children marijuana or cannabis," said Wilfong. "I'm passionate, my team is passionate about doing everything we can, exploring all possible new leads to see if we can help these children lead a safer happier more healthy life with this condition."
Lori Fountain, like other parents desperate for a cure or at least a reduction in seizures, had considered moving her family to Colorado where CBD treatments are available. But the Texas Children's/GW Pharmaceuticals study is the first scientific approach taken to determine if the completely non-TCH extract is actually effective.
"And I don't care what people think. I mean, really I don't," said Fountain about those who might question her willingness to treat her grandson with a marijuana-derivative drug. "I'll hand him a pamphlet and tell them what's wrong with him. And tell them to come spend a whole day in my shoes."
Texas Children's began using Epidiolex earlier this year under compassionate use protocols for two adolescent patients with catastrophic forms of epilepsy that were not responsive to other drugs. Wilfong, the first physician in Texas to be able to administer Epidiolex to pediatric patients, says the results have been promising. Now, through the clinical trial funded by GW Pharmaceuticals, they hope to prove its efficacy.
"We are hopeful that in the next year, the results of this trial will show this drug has a positive impact on enrolled patients and also that it will have implications for patients with other forms of intractable epilepsy," said Dr. Gary Clark, chief of neurology at Texas Children's.
The Epidiolex trial is accepting only pediatric patients with confirmed Dravet syndrome. To learn more, visit clinicaltrials.gov