New gene-altering jab offers hope to children with drug-resistant epilepsy developed by researchers at University College London

Scientists have developed a gene therapy to cure a drug-resistant form of childhood epilepsy.

Focal cortical dysplasia occurs when areas in the brain develop abnormally, triggering frequent seizures. It can lead to learning difficulties and even death. While surgery to remove the faulty brain tissue can be effective, it also risks permanent brain damage.

But researchers at University College London (UCL) say they have developed a gene-altering medicine that could slash the number of seizures sufferers experience, with the potential to drastically improving the quality of life of epileptic children.

‘It could be deployed to thousands of children who are currently severely affected by uncontrolled seizure,’ says Dr Vincent Magloire, an epilepsy expert at UCL.

Some 600,000 people in the UK have epilepsy. Seizures are the most common symptom and occur when electrical impulses that ferry messages between cells in the brain get disrupted.

Roughly a third of patients do not respond to traditional drugs used to control epilepsy (file photo) 

Over the past decade a number of effective drugs have been rolled out on the NHS, meaning many patients are able to have relatively normal lives with few-to-no seizures.

But roughly a third of patients do not respond to these drugs. This includes those with focal cortical dysplasia, the most common cause of drug-resistant epilepsy in children, which affects around 35,000 children and young people.

Brain cells are supposed to form organised layers so 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 jumbled up, disrupting the signals and leading to seizures.

By analysing mice, researchers have discovered that increasing the brain’s levels of potassium – a natural chemical found in the body that helps regulate cells – reduces the rate of seizures.

This is because potassium appears to decrease the activity of brain cells, limiting the risk of the electrical impulses misfiring. Mice given the one-off injection had 87 per cent less seizures, on average, compared with those that were not treated.

Professor Dimitri Kullmann, a neurologist at UCL, says that plans for human clinical trials are under way and are expected to begin in the next five years.