Gene therapy has been suggested as a plausible novel approach to achieve seizure control in patients with focal epilepsy that do not adequately respond to pharmacological treatment.
In this study, Melin et.al have investigated the seizure-suppressant potential of combinatorial neuropeptide Y and Y2 receptor single vector gene therapy, first in a dose-response study, and second in an efficacy study.
The latter demonstrated a favorable outcome of the gene therapy, with a 31% responder rate and 13% seizure-freedom rate. In addition, the seizure duration was significantly reduced and the intervals between the seizures were prolonged in the treated group compared to the controls.
This study establishes the suppressant ability of the single vector combinatorial neuropeptide Y/Y2 receptor gene therapy in a clinically relevant chronic model of epilepsy.