Safety and Efficacy of a Novel MicroRNA-Based Therapy for Epilepsy in a Translational Animal Model

Current treatments for epilepsy work mainly by reducing brain excitability. They fail to control seizures in many patients and must be taken repeatedly. Dr. Gutierrez-Quintana and his team propose to test a completely new class of drug targets for epilepsy called microRNAs. Instead of changing brain excitability, these naturally occurring molecules coordinate signaling processes inside brain cells by controlling expression of various genes. The team has previously found that, in brain tissue from people with temporal lobe epilepsy, there was a consistent increase in the level of one of these molecules called microRNA-134. The team developed an inhibitor of microRNA-134, called Ant-134, that when injected into mice and rats significantly suppressed seizures. Remarkably, Ant-134 has been shown to have potent and long-lasting anti-convulsant effects in numerous rodent models after a single dose.

Dr. Gutierrez-Quintana’s team will now test the effects of Ant-134 in a population of dogs with naturally occurring drug-resistant epilepsy, an important step towards translating their findings into treatments for humans. If successful, this project will pave the way for human clinical trials for a treatment that has the potential of providing long-lasting seizure control after a single dose and may be even cure some forms of epilepsy.