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.

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.

It is very exciting to see that this new gene therapy could potentially be used as an effective alternative to surgery in patients with focal cortical dysplasia.

Co-corresponding author, Professor Gabriele Lignani (UCL Queen Square Institute of Neurology)

Gene therapies have previously been shown to work in another form of epilepsy where seizures arise in the temporal lobes, but have not been tested in focal cortical dysplasia.

The researchers found that the gene therapy reduced seizures by an average of 87% when compared with the control group, without affecting the mouse’s memory or behaviour.

Following the successful study in mice, we believe the treatment is suitable for clinical translation, and, taking into account the size of the unmet need, it could be deployed to thousands of children who are currently severely affected by uncontrolled seizures.

Lead author Dr Vincent Magloire, UCL Queen Square Institute of Neurology

Plans for a first in human clinical trial are underway and are planned in the next five years.

Co-corresponding author, Professor Dimitri Kullmann, UCL Queen Square Institute of Neurology

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