New gene therapy could significantly reduce seizures in severe childhood epilepsy

UCL researchers have developed a new gene therapy to cure a devastating form of childhood epilepsy, which a new study shows can significantly reduce seizures in mice. The study, published in Brain , sought to find an alternative to surgery for children with focal cortical dysplasia. Focal cortical dysplasia is caused by areas of the brain that have developed abnormally and is among the most common causes of drug-resistant epilepsy in children. It frequently occurs in the frontal lobes, which are important for planning and decision-making. Epilepsy in focal cortical dysplasia is associated with comorbidities, including learning disabilities. Although surgery to remove the affected brain malformation is effective, its use is severely limited by the risk of permanent neurological deficit and does not always result in seizure freedom. Consequently, researchers at the UCL Queen Square Institute of Neurology evaluated a gene therapy based on the overexpression of a potassium channel which regulates neuronal excitability in a mouse model of focal cortical dysplasia in the frontal lobe.