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Developing cutting-edge gene therapy for severe childhood epilepsy

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Epilepsy affects around 70,000 babies, children and young people under 18 years old in the UK.[1,2] Sadly, medication doesn’t work in up to one in three young people and other treatments, such as surgery, are not always effective, meaning life can be difficult and unpredictable.[3] Professor Dimitri Kullmann of University College London is leading a team aiming to develop gene therapies for children with a severe type of epilepsy called focal cortical dysplasia (FCD) who may have no other treatment options. They hope this laboratory work will ultimately lead to effective new treatments that can help control seizures – transforming the lives of children and their families affected by this devastating condition.

This project is jointly funded by Action Medical Research and LifeArc.

How are children’s lives affected now?

Children with focal cortical dysplasia (FCD) have disorganised nerve cells in an area of their brain, which puts them at a much higher risk of seizures. This severe form of epilepsy is caused by problems with brain development while the baby is still in the womb – and is sometimes due to faulty genes.

“Seizures can be scary for children and their families – and can put them at risk of injury or even death,” says Professor Kullmann. “There’s no way of knowing when they may occur, which can impact hugely on their everyday lives – such as going to school or taking part in social activities.”

Unfortunately, some children with FCD will continue having seizures despite trying medication – and other treatments, such as surgery, are risky and aren’t suitable for everyone.

“There is an urgent need for effective new treatment options for children with FCD whose seizures can’t be controlled by existing treatments,” says Professor Kullmann.

How could this research help?

“Our goal is to develop gene therapy as a safe and effective treatment for children with FCD,” says Professor Kullmann.

The approach involves using a harmless virus to deliver a working copy of the faulty gene into nerve cells in the area of the brain affected by FCD. The researchers have already tested several such gene therapies developed for treating adult epilepsies in the laboratory – and are about to start a clinical trial in adults.

 “Building on our previous work, we now plan to investigate if any of these gene therapies could be repurposed for treating children with FCD,” says Professor Kullmann.

The team will carry out laboratory experiments to identify which of their gene therapies has the most potential to become a safe and effective treatment for children with FCD.

If our results are encouraging, it could pave the way for future clinical trials in children affected by this devastating condition.

Professor Kullmann


  1. Young Epilepsy: Information about epilepsy; [website accessed 6th June 2023]
  2. Office for National Statistics; Estimates of the population for the UK, England and Wales, Scotland and Northern Ireland - Office for National Statistics ( [website accessed 6th June 2023]
  3. NHS Epilepsy treatment: [website accessed 20 October 2022]

Research table

Project details

Project Leader Professor Dimitri M Kullmann, FMedSci FRS
Location Department of Clinical and Experimental Epilepsy, UCL Queen Square Institute of Neurology, University College London
Project Team Professor Matthew C Walker, PhD FRCP
Professor Stephanie Schorge, PhD
Dr Gabriele Lignani, PhD
Other Locations Department of Neuroscience, Physiology and Pharmacology, University College London
Grant Awarded
Grant Amount £247,521
Start Date
End Date
Duration 36 months
Grant Code (GN number) GN2930


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