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

This is a work of excellent quality carried out by a research group of recognized prestige in the field of epilepsy. The work has been led by researchers from the Department of Clinical and Experimental Epilepsy at the University College of London, one of the most advanced departments in clinical and epilepsy research in the world. The work involves experiments with an adeno-associated virus containing a promoter and a gene that, once activated by neuronal activity corresponding to an epileptic seizure, inhibit neuronal activity. In other words, the system works on demand of the epileptic activity. Thus, we would have a treatment that would inhibit neuronal epileptic activity only in the event of a spontaneous epileptic seizure. The experiments were performed on mouse models of epilepsy and on neuronal cells in culture.  

The work provides a very novel treatment approach for patients suffering from drug-resistant epilepsy, who represent 30% of the total number of patients with epilepsy, probably about 100,000 in Spain. This treatment route would be an alternative to the usual treatment for these patients, which consists of treatment with anti-crisis drugs or resective surgery. This involves the ablation of part of the brain tissue which, in addition to being aggressive, can affect different motor or cognitive functions. It also has the advantage that the physiological alteration introduced is only induced in the event of a crisis and not during normal brain activity.  

Although work remains to be done for its preclinical and clinical development, it is likely that this form of treatment will be established in the near future. In addition to the technical challenges in humans (e.g. brain area and injection system), it needs to be demonstrated that this form of treatment does not produce relevant adverse effects and to investigate how it could selectively target different neuronal groups.