Seizure burden, especially in patients with poorly controlled, drug-resistant epilepsy, is a serious clinical problem.
In this regard, blood-brain barrier dysfunction is a critical factor that triggers a chain of events leading to seizures and contributes to the development of drug resistance.
We identified two FDA-approved drugs that attenuate barrier dysfunction in vivo. Our preliminary data suggest that repairing barrier dysfunction helps reduce seizure burden, yet therapeutic options to restore barrier function in patients are currently not available. We directly address this critical unmet need in our team science project by establishing a bench-to-bedside pipeline to develop novel drugs, test these drugs in preclinical epilepsy models, and conduct clinical pilot studies in patients with poorly controlled epilepsy.