Reacción a "Reactions to study testing gene therapy in mice to treat epilepsy"

Epilepsy affects about 1 % of the population worldwide and for almost one third of people with epilepsy, medication is not enough to control seizures and have a good quality of life. Over the past 30 years, more than 20 different treatments for epilepsy have been developed. However, these have failed to reduce the proportion of refractory epilepsies (those that do not respond to drug treatments). This makes the need for new avenues of treatment all the more necessary.  

In recent years, gene therapies or other genetically targeted therapies are being developed for many types of epilepsies caused by specific mutations (pathogenic variants), such as Dravet syndrome (related to the SCN1A gene). However, these treatments will only work for specific causes, and we will need a different therapy design for each epilepsy (most of them rare or infrequent diseases). This work is encouraging, especially because if confirmed to be effective in patients, it would be useful for the most common group of epilepsies, the so-called focal epilepsies (related to a focus or lesion in the brain), in both children and adults.  

Colleagues at the University College of London have made it possible with this treatment, using a viral vector (adeno-associated), to silence a gene that is only activated by seizures, so it could treat seizures without "switching off" the healthy activity of the patient's neurons. They have shown that this can be useful in animal models. The next step will be to conduct clinical trials in patients with refractory epilepsy. Caution must always be exercised with these preclinical (laboratory) studies and clinical development is still years away, but the data they show and the novel approach give hope to the whole epilepsy community.