Promising Treatment Emerging for Intractable Childhood Epilepsy

UH Rainbow Babies & Children's one of few centers in the country to offer Phase 3 clinical trial of important new therapy

Innovations in Pediatrics | Fall 2019

Asim Shahid, MD

A medication being tested in Phase 3 clinical trials at UH Rainbow Babies & Children’s Hospital and a small number of other centers has the potential to revolutionize the standard of care for children and teens with rare, intractable forms of epilepsy.

The clinical trials under way here are evaluating the effectiveness of the medication fenfluramine, which has been used in Europe to treat patients with epilepsy, in patients with Dravet syndrome and Lennox-Gastaut syndrome.

“Both syndromes are refractory epilepsies, meaning no matter what we do in terms of medications and therapeutic interventions, these patients continue to have seizures,” says pediatric epilepsy specialist and Division Chief of Pediatric Neurology Asim Shahid, MD; and Associate Professor of Pediatrics and Assistant Professor of Neurology, Case Western Reserve University School of Medicine who is conducting the clinical trials at UH Rainbow. Dr. Shahid adds that in most cases these patients experience poorer outcomes, and  are at higher risk of sudden unexpected death in epilepsy (SUDEP), especially patients with Dravet syndrome.

“Currently these children are treated with the conventional medications -- valproic acid or clobazam or stiripentol -- but they continue to have seizures. There is a great need to find newer therapies to help them and that’s where this new medication comes in.”

So far, the double-blind, randomized, placebo-controlled trials of fenfluramine in Dravet syndrome and Lennox-Gastaut syndrome have yielded extremely encouraging results, Dr. Shahid says.

Beyond the aggregate data, Dr. Shahid says he’s also seen a dramatic difference in his individual patients.

“One of my patients, a 9-year-old boy who is enrolled in the trial, was having up to 90-odd seizures per month,” he says. “He would come to my office either foggy from seizure medications or just after having a seizure.“

The family moved his care hundreds of miles to be involved in the trial here,” Dr. Shahid adds. “He’s now in the open-label portion of the trial and experiencing about 23 or 24 seizures per month – a good, significant 70 percent decrease. Now when I see him, he’s interactive. He shakes my hand and says, “Hello, Doctor!” and tells me stories about Blue’s Clues. I could never have imagined such progress. The family is excited he is able to take steps by himself. Before he was in a wheelchair or a stroller. I also have a girl who was having 16 to 18 seizures each month, and now she’s down to one or two a month.”

Given positive results such as these, Dr. Shahid says he expects fenfluramine to win FDA approval for patients with Dravet syndrome and Lennox-Gastaut syndrome sometime in 2020.

For now, he says, it’s enough to enjoy seeing his difficult-to-treat patients improve so dramatically.

“We’re all doing this for the patients,” he says. “Anything we can do to help these children get better in terms of their seizures and improve their quality of life, that’s very gratifying.”

For more information about the Phase 3 clinical trials of fenfluramine as a treatment for Dravet syndrome or Lennox-Gastaut syndrome under way at UH Rainbow Babies & Children’s Hospital, please call, Pediatric Neurology at 216-553-1947 or email Peds.Innovations@UHhospitals.org.