GENE THERAPY IN DRUG-REFRACTORY EPILEPSY: THE FOCUS ON NEUROPEPTIDE Y

Hippocampal NPY gene transfer as a therapeutic strategy for pharmacoresistant temporal lobe epilepsy Temporal lobe epilepsy represents both the most common but also unfortunately, the most intractable of all the epilepsies. The best current option for the majority of individuals who continue to have seizures despite optimal drug treatment, is that of temporal lobectomy, an operation which involves removing the region of the brain from which the seizures originate. This operation is highly successful for most cases, but fails in some, and moreover many individuals are reluctant to undergo surgery that involves removing part of their brain. Here, we are proposing experiments which we hope will show that there is a potentially safer and perhaps even more effective surgical approach, that of injecting directly into that same brain region, a harmless virus which contains a specific gene which encodes one of the brain’s natural anticonvulsants. The goal is to show that such gene delivery is not only safe and well tolerated, but also stops seizures. If we can be successful in these experiments, the same approach could rapidly be scaled up to human trials, hence this research is what we call translational, or focused on research specifically orientated towards clinical investigation and treating the human condition.