New gene-altering jab offers hope to children with drug-resistant epilepsy developed by researchers at University College London
- About 600,000 people in Britain suffer from epilepsy and experience regular seizures
- The new shot can help children with a specific drug-resistant form of epilepsy
Scientists have developed a gene therapy to cure a drug-resistant form of epilepsy in children.
Focal cortical dysplasia occurs when areas of the brain develop abnormally, causing frequent seizures. It can lead to learning problems and even death. Although surgery to remove the defective brain tissue can be effective, it also risks permanent brain damage.
But researchers from University College London (UCL) say they have developed a gene-altering drug that could reduce the number of epilepsy seizures, with the potential to dramatically improve the quality of life for children with epilepsy.
“It could be used in thousands of children who are currently seriously affected by uncontrolled seizures,” said Dr Vincent Magloire, an epilepsy expert at UCL.
About 600,000 people in Britain have epilepsy. Seizures are the most common symptom and occur when electrical impulses that transmit messages between cells in the brain are disrupted.
About 600,000 people in Britain have epilepsy. Seizures are the most common symptom and occur when electrical impulses that transmit messages between cells in the brain are disrupted
About a third of patients do not respond to traditional medications used to control epilepsy (file photo)
Over the past decade, a number of effective medications have been launched on the NHS, meaning that many patients can lead relatively normal lives with few to no seizures.
But about a third of patients do not respond to these medications. This includes people with focal cortical dysplasia, the most common cause of drug-resistant epilepsy in children, affecting around 35,000 children and young people.
Brain cells are thought to form organized layers so that electrical impulses can travel smoothly between parts of the brain. However, in patients with focal cortical dysplasia, these layers – often in the frontal lobe, which is responsible for planning and decision-making – are confused, disrupting signals and leading to seizures.
By analyzing mice, researchers have found that increasing levels of potassium in the brain – a natural chemical found in the body that helps regulate cells – reduces the number of seizures.
This is because potassium appears to reduce the activity of brain cells, limiting the risk of electrical impulse failure. Mice that received the single injection had an average of 87 percent fewer seizures, compared to mice that were not treated.
Professor Dimitri Kullmann, a neurologist at UCL, says there are plans for clinical trials on humans and are expected to begin within the next five years.