Scientists say the experimental drug Zorevunersen for Dravet Syndrome significantly reduced seizures in children during early clinical trials in the United Kingdom, offering hope for treating the rare, drug-resistant genetic epilepsy.
Early Trial Shows Major Seizure Reduction in Children
Researchers report that zorevunersen cut seizure frequency in children with Dravet syndrome by about half in early clinical trials, marking a potential breakthrough for a condition that often resists current treatments.
The study, led by University College London and Great Ormond Street Hospital, involved 81 children between the ages of two and 18. Before treatment, participants experienced an average of 17 seizures a month.
After receiving a 70-milligram dose of the drug, seizures dropped by about 50 percent on average. After three doses, researchers observed about an 80 percent reduction in seizure frequency.
Scientists also reported improvements in quality of life indicators, including motor skills, communication, and daily functioning. The findings were published in The New England Journal of Medicine.
Dravet syndrome is a rare genetic disorder that causes severe epilepsy and developmental delays. About 3,000 people in the United Kingdom are believed to have the condition.
Researchers Say Drug Could Transform Treatment
Lead author Helen Cross, director of childhood epilepsy at the UCL Institute of Child Health and an honorary consultant in pediatric neurology at Great Ormond Street Hospital, said the results highlight the urgent need for new therapies.
“I regularly see patients with hard-to-treat genetic epilepsies who can have multiple seizures a week,” Cross said. “Many are unable to do anything independently for themselves and require around-the-clock care.”
Children with the condition face a higher risk of sudden unexpected death in epilepsy, she added.
Cross said the next phase of research will determine whether the treatment can provide lasting benefits and confirm its long-term safety.
“If the phase three trials are successful, this treatment could help children with Zorevunersen for Dravet Syndrome lead much healthier and happier lives,” she said.
Experts Call Findings a Step Toward Genetic Epilepsy Therapies
Independent epilepsy experts welcomed the results, calling them an important step toward treatments that target the root genetic cause of the disorder rather than simply controlling symptoms.
Jowinn Chew, a researcher at London South Bank University, described the trial results as “a clinically significant step forward” in developing targeted therapies Zorevunersen for Dravet Syndrome.
Dr. Alfredo Gonzalez-Sulser of the University of Edinburgh said the findings could influence research on many other rare epilepsies.
“There are now more than 800 genetic epilepsies that need therapeutics similar to zorevunersen,” he said. “This sets a clear path to achieve effective interventions for these severe life-altering diseases for both patients and carers.”
Deb Pal, a professor of epilepsy at King’s College London, said the research offers hope for families affected by rare genetic seizure disorders.
“This landmark study gives enormous hope for the families of thousands of children and young people affected by monogenic epilepsies worldwide,” Pal said.
Researchers say a larger phase three clinical trial will begin to examine the drug’s long-term safety and effectiveness and determine which patients may benefit the most.
