A new drug called zorevunersen has shown promise in reducing seizures by up to 90 percent in children with Dravet syndrome, a rare and devastating form of epilepsy. The findings, published March 4 in The New England Journal of Medicine, represent a potential breakthrough in treating a condition characterized by severe neurodevelopmental delays and a high risk of sudden death.
Dravet syndrome is caused by a genetic mutation in the SCN1A gene, which controls sodium channels required for proper interneuron signaling in the brain. Zorevunersen works by increasing protein production from the working copy of the SCN1A gene, directly addressing the underlying cause of the disease. This approach makes it potentially the first drug with the ability to alter the disease’s trajectory, rather than merely reducing seizures.
The early-stage trial involved 81 children ages 2 to 18 at hospitals in the U.K. and the U.S. The drug was delivered via lumbar puncture—a spinal injection that places the medication into cerebrospinal fluid bathing the brain. After 20 months of treatment, children who received the highest initial dose experienced between 59 percent and 91 percent fewer seizures. The study also noted improvements in neurodevelopmental outcomes and quality of life, particularly at higher doses. Side effects were mild, including occasional headaches or vomiting from the lumbar puncture procedure, and elevated protein levels in cerebrospinal fluid. Overall, the trial demonstrated the drug’s safety for children.
According to study leader Dr. Helen Cross, a professor of childhood epilepsy at University College London, the treatment improves multiple aspects of the disease beyond seizure reduction. A larger trial studying an additional 170 children is already underway, with results expected by October 2028.
This article is an AI-assisted summary. All facts and figures are drawn from the original report: https://www.livescience.com/health/groundbreaking-new-drug-shows-promise-for-treating-children-with-a-devastating-form-of-epilepsy