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11:36 AM

Could Component of Diet Pill 'Fen-Phen' Treat Epilepsy?

Cook Children's enrolling patients in follow-up trial for children with rare, devastating epilepsy

Could a component of the diet pill “fen-phen” be the answer for patients with a severe form of epilepsy known as Dravet syndrome?

Fenfluramine was originally used as a weight-loss drug before being pulled from the market in 1997 with reports that it caused severe damage to the heart.

Now being used at lower dosages, clinical trials in the United States, Canada and Europe have found the drug to be highly effective in the treatment of seizures in Dravet syndrome.

The results from the randomized, double-blind, placebo controlled phase 3 trial of Zogenix ZX008 (previously known as fenfluramine) showed it to be effective in the treatment of seizures in Dravet syndrome. Dravet syndrome is a rare, devastating epilepsy syndrome of childhood presenting with multiple seizures types, often resistant to most antiepileptic drugs (AEDs).

The most recent study included 119 patients between the ages of 2-18 years. After a six-week baseline, patients were randomized to placebo, a lower dose (0.2 mg/kg/d) or a slightly higher dose (0.8mg/kg/d) group added to their existing AEDs, which remained stable throughout the study.

Seventy percent of patients had a greater than 50-percent reduction in convulsive seizures in the high dose, 41 percent in the low and only 7.5 percent in placebo. Forty-five percent of the patients who participated had more than a 75-percent reduction in the high dose, 20.5 percent in the low, and 2.5 percent in the placebo. Finally, the high dose group experienced 20.5 days of seizure freedom as the longest median duration, 14 days in the low dose and only 9 in placebo.

This is intriguing news locally because Scott Perry, M.D., an epileptologist and medical director of the John and Jane Justin Neurosciences Center at Cook Children’s, is leading a second similar study at Cook Children's.

Currently, six patients are participating in the study at Cook Children's and the study remains open for enrollment. The study is open to children with Dravet syndrome with seizures uncontrolled on current AEDs.

"A number of inclusion/exclusion criteria are required to be selected for the trial. Patients may not be using cannabidiol (CBD) and they can’t have preexisting cardiac abnormalities because of past cardiac valve problems with the drug used as a diet pill," Dr. Perry said. "Based on the early open label results published from Belgium which prompted this present study, I’m quite excited. I’m pleasantly surprised that the results of this blinded/controlled trial were very favorable, much like the early open label results suggested. These children have a catastrophic form of epilepsy for which seizure control is very unlikely. This drug has demonstrated significant reduction in seizures which would be considered successful in any epilepsy syndrome. The results are that much more impressive given the difficulty these patients have in gaining seizure control.”

About Our Epilepsy Surgery Center

Cook Children's is a recognized Level 4 epilepsy center. The National Association of Epilepsy Centers recognizes Cook Children's Comprehensive Epilepsy Program as a Level 4 Pediatric Epilepsy Center. Level 4 epilepsy centers have the professional expertise and facilities to provide the highest level medical and surgical evaluation and treatment for patients with complex epilepsy. Click to learn more.

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