Trial Of Marijuana-Based Drug Offers Hope
Izaiah Ruiz’s epilepsy was so severe, his daily life so miserable, that his grandmother says she would have sold her home outside Houston and moved to Colorado for what many say is a new, miracle treatment: marijuana.
But Lori Fountain couldn’t make the finances work. As her 6-year-old grandson suffered, all she could do was follow online the progress of patients who sought treatment in Colorado, where marijuana is legal. Their families reported patients suddenly were seizure-free, verbal, able to dress and feed themselves thanks to treatment with a strain of marijuana, a preparation known as Charlotte’s Web, which has become something of a national phenomenon.
Seemingly without hope, Fountain a few weeks ago got unbelievable news from Texas Children’s Hospital: Izaiah would be one of the first patients in an international trial of an experimental drug derived from marijuana that has shown dramatic benefits in a select few patients who have already received it. The drug, Epidiolex, is a highly purified extract of cannabis that does not contain THC, the ingredient that alters mood.
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Fountain said she cannot wait to bring Izaiah to Texas Children’s for testing.
“If this drug could work and give Izaiah any semblance of a normal life, I can’t tell you how ecstatic I’ll be,” said Fountain, who described the boy’s constant seizures, inability to talk and failure to comprehend Christmas or other holidays. “I can’t explain what it’d mean to watch him playing outside, going down a slide, taking part in an Easter egg hunt.”
Texas Children’s is expected to enroll the most patients in the 10-site trial, which is being conducted on patients with a rare and severe form of epilepsy known as Dravet syndrome.
The syndrome, which occurs in about one in every 30,000 births, is characterized by dozens or even hundreds of seizures a day, some life-threatening. Patients typically struggle with developmental delays, a variety of health problems and an increased risk of what is known as Sudden Unexplained Death in Epilepsy.
Difficult to control
Dravet syndrome seizures are difficult to control and frequently don’t respond to medications available now. Some drugs that work in many epilepsy patients make Dravet syndrome worse.
Dr. Angus Wilfong, the study’s principal investigator at Texas Children’s, says he hopes the trial shows Epidiolex is the safe and effective treatment desperately needed — that it lives up to the expectations generated by the anecdotes.
“Charlotte’s Web has received a lot of attention, but there’s no proof it works,” said Wilfong, a pediatric neurologist at Texas Children’s and Baylor College of Medicine. “This will be a scientifically rigorous study of a standardized product that shouldn’t cause any issues of addiction.”
Two Dravet syndrome patients who previously received Epidiolex at Texas Children’s through a compassionate-use program approved by the Food and Drug Administration showed dramatic benefits, Wilfong said.
Though he said he thinks a drug synthesized from marijuana would be more potent than Charlotte’s Web, Wilfong cautioned he didn’t expect the drug would make any Dravet syndrome patient seizure-free.
Dr. Elson So, a neurologist and president of the American Epilepsy Society, said the Epidiolex trial is important “not to disprove Charlotte’s Web but to make sure we don’t miss out on a compound that could be very good for epilepsy.” He noted that without such a study, the politics of marijuana could blind people to a useful compound.
Two phases of study
The study is being conducted in two stages. The first part will enroll 30 children 4 to 10 years old and focus on determining safe and effective doses. Patients, tested the first month for the severity of their Dravet syndrome, thereafter will receive any of number of potencies of the drug or a placebo.
The second part, expected to begin in early 2015, will enroll 80 children 2 to 18 years old.
Epidiolex, a liquid medicine taken by mouth or through a feeding tube, is made by GW Pharmaceuticals. It has been studied in a previous “precursor” trial, in which researchers reported on results with patients, not all with Dravet syndrome, not randomized to either a treatment or placebo arm and taking other medications. The researchers showed some benefit.
The new trial is only for pediatric patients with confirmed Dravet syndrome. The first patient began testing in Atlanta last week. Izaiah will be the second patient.
Fountain said she hopes Izaiah gets Epidiolex from the start, rather than a placebo, but she takes solace that if he doesn’t, he’ll get the drug soon thereafter.
“I’ve been dealing with this since Izaiah was 2 months old, when he had his first seizure,” said Fountain, who stays with the boy around the clock, including when he’s sleeping. “We’ve almost lost him a couple times and know that without new treatment, he may not reach his teenage life. This drug is our hope.”