A diet drug could reduce seizures for pediatric patients with Dravet syndrome.
A novel formulation of a drug that was initially used to treat children with a rare neurologic disease and eventually became part of a weight loss drug combination may be able to treat pediatric patients with severe seizures, according to a study conducted by the University of California, San Francisco.
In the phase 3 clinical trial, treatment with ZX0008 (fenfluramine) was linked to a drastic reduction in the number of seizures experienced by patients with Dravet syndrome.
Approximately 70% of patients experienced a 50% reduction in the number of seizures experienced.
“Dravet syndrome is a devastating condition that severely impacts the quality of life for both the children and their families,” principal investigator Joseph Sullivan, MD, said in a press release. “These results are very encouraging. Even reducing seizures for these children to once a month, instead of once or twice a week, makes a tremendous difference in their lives.”
Dravet syndrome is a severe form of pediatric epilepsy that is characterized by frequent seizures. The condition is often caused by defects in a gene that is required for normal brain cell function.
Patients with the condition tend to experience language and motor impairments, as well as hyperactivity and difficulty relating to their peers. Up to 15% of patients with the condition will die by age 25 commonly due to sudden unexpected death, according to the authors.
Included in the study were 119 children under 19 years of age with Dravet syndrome who were randomized to receive 0.8-mg/kg or 0.2-mg/kg of the investigational drug or placebo per day.
Among patients taking a higher dose of ZX008, 45% had at least a 75% reduction in monthly seizures, while only 20.5% of patients taking a lower dose achieved this, according to the study. In comparison, only 2.5% of patients in the placebo cohort had such a dramatic reduction in seizures.
Patients taking higher doses of the drug went an average of 20.5 days without experiencing a seizure. Patients taking the lower dose of the drug and those taking the placebo went 14 days and 9 days without seizures, respectively, according to the study.
Previously, fenfluramine was used to treat self-induction epilepsy, but researchers could not confirm that the drug had a direct anti-epileptic mechanism. It was also part of a combination of the weight loss drug fenfluramine/phentermine in the 1980s.
Although the drug was withdrawn from the market due to cardiovascular damage in patients with obesity, the authors reported that the new formulation did not damage the heart, according to the study.
These findings suggest that the investigational drug may be able to effectively reduce the number of seizures and associated disability young patients may experience.
“Since the degree of cognitive impairment may correlate with the frequency of seizures, our hope is that more effective treatments may also reduce these impairments,” Dr Sullivan said in the release.