Epilepsy study sees benefit through hard-to-obtain drug
WINCHESTER — The results of an epilepsy study being conducted in Winchester have not been confirmed yet, but the signs have been encouraging.
So far, epilepsy medication Epidiolex, a controlled substance because it’s derived from the cannabis plant, has had a welcome effect on program participants, said Dr. Paul Lyons, medical director of the Virginia Comprehensive Epilepsy Program and attending physician at Winchester Medical Center.
“Overall it’s been terribly well tolerated,” Lyons said. “At the end of the day, the children have more seizure-free days, meaningfully, and the seizures they’re having are shorter.”
The three-month compassionate use study on two Virginia children with treatment-resistant, catastrophic forms of epilepsy is an unfunded, pro-bono effort by Lyons and Jennifer Stanford, a registered nurse and director of the Valley Health Clinical Research Department.
It started in November and monitors the effect of Epidiolex on treatment regimens the children were already following, Stanford said.
Epidiolex, dispensed by GW Pharmaceuticals in the United Kingdom, is the first drug approved by the Federal Drug Administration for medical research in epilepsy, Lyons said Wednesday at a news conference held at his office in Winchester Neurological Consultants.
It’s highly regulated because, as a derivative of the same plant that produces the psychoactive ingredient tetrahydrocannabinol (THC) in marijuana, it’s illegal for undocumented use in the United States.
“The big misperception of medical marijuana is the confusion the public has about what it is,” Lyons said. The drug he administers for the study is purified medical grade extract from the cannabis plant.
Patients in the study take the medication like they would cough syrup, Lyons said.
Side effects he mentioned are an upset stomach that can be alleviated by changing the dosage, and improved alertness.
Both participants — 9-year-old Courtney Iser, of Gore, and 12-year-old Caleb Thomas, of Virginia Beach — experienced mild stomach pain, but Lyons said neither had any other adverse response.
In fact, their parents have seen improvements.
Courtney, diagnosed with a severe form of epilepsy called Lennox-Gastaut syndrome, had her first seizure when she was 6 months old and was diagnosed, her mother Sherri Iser said. Courtney has tried 12 separate pharmaceutical medications; the high-fat, low-carb ketogenic diet used primarily for treating refractory epilepsy; and vagus nerve stimulation, which sends mild pulses of electrical energy to the brain via the vagus nerve.
“And with all that she still continues to have seizures,” Iser said.
Tracie Thomas’ son Caleb has experienced seizures since he was 3 and contracted viral encephalitis — an inflammation of the brain caused by virus.
He has tried all 24 anti-convulsive medications used for treating epilepsy, some more than once. He’s also tried the ketogenic diet and vagus nerve stimulation.
Since starting the Epidiolex study, Courtney and Caleb have both improved.
“He’s more alert, more engaged,” Thomas said. “… You can see a difference in his eyes, like he’s alive.”
“[Courtney] has had 23 seizure-free days of the 63 days we’ve been in the study,” Iser said. “Seven days have been consecutive.”
On Wednesday, moments before the news conference, Courtney had a seizure lasting a couple minutes. Her father said the seizure was much shorter than ones she had before beginning the study — some lasting hours and requiring emergency room treatment.
But Lyons is confident Epidiolex will have needed effects on the study of epilepsy — a condition the medical field has labored with for 102 years.
The need for better treatment of epilepsy inspired him to endure 13 months of phone calls, background checks and certifications to legally be able to possess and administer Epidiolex.
Classified a Schedule 1 controlled substance, it’s stored alone in a 500-pound safe that only Lyons can access.
Though the compassionate use study ends in another month, he said GW Pharmaceuticals has agreed to supply Courtney and Caleb with a year’s amount of Epidiolex free of charge, as Lyons monitors their conditions.
He also plans a fully funded study for treating patients of all ages with Dravet syndrome and Lennox-Gastaut syndrome, beginning in March or April and allows for more participants.
The randomized, placebo-controlled study will administer Epidiolex to half of study participants and a medically ineffective pill to the others.
After completing the study, he said all participants will be considered for access to Epidiolex.
Though other studies of Epidiolex are happening in New York, Chicago and San Francisco, Lyons’ office in Winchester is the only location in Virginia and the surrounding states certified to administer Epidiolex.
He said he hopes to also extend the study to his other offices in Purcellville and Charlottesville.
“If you can drive here, fly here, come here,” Lyons said.
To be considered for inclusion in the randomized study, contact Lyons at 866-948-7055 or at http://www.winchesterneurological.com, or Jennifer Stanford at 540-536-8978.
Contact staff writer Josette Keelor at 540-465-5137 ext. 176, or firstname.lastname@example.org
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