A new gene-editing vaccine offers hope for children with drug-resistant epilepsy, developed by researchers at University College London.
- About 600,000 people in Britain suffer from epilepsy and suffer from regular seizures
- A new injection could help children with a specific, drug-resistant type of epilepsy
Scientists have developed a gene therapy to treat a drug-resistant form of epilepsy in children.
Focal cortical dysplasia occurs when areas of the brain develop abnormally, resulting in recurrent seizures. It can lead to learning difficulties and even death. While surgery to remove defective brain tissue can be effective, it also risks permanent brain damage.
But researchers at University College London (UCL) say they have developed a gene-editing drug that could reduce the number of seizures patients suffer, with the potential to significantly improve the quality of life of children with epilepsy.
“It could be deployed to thousands of children who are currently severely affected by uncontrolled seizures,” says Dr. Vincent Magloire, an epilepsy expert at UCLA.
About 600,000 people in the UK have epilepsy. Seizures are the most common symptom and occur when electrical impulses that transmit messages between cells in the brain are disrupted.
Over the past decade, a number of effective medicines have been introduced into the NHS, meaning that many patients are able to live relatively normal lives with few or no seizures.
But approximately one-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 supposed 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 lobes, responsible for planning and decision-making – become mixed up, disrupting signals and leading to seizures.
By analyzing mice, researchers discovered that increasing levels of potassium in the brain – a natural chemical found in the body that helps regulate cells – reduces the rate of seizures.
This is because potassium appears to reduce the activity of brain cells, reducing the risk of failure of electrical impulses. Mice that were given a single injection had 87% fewer seizures on average, compared to those that were not treated.
Professor Dimitri Coleman, a neurologist at University College London, says plans for human clinical trials are underway and expected to begin in the next five years.
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