A Novel Zebrafish-Based Platform for Anticonvulsant Drug Development
- Deborah Kurrasch, University of Calgary
- Jong Rho, University of Calgary
- Jeffrey Buchhalter, University of Calgary
- Mary Connolly, University of British Columbia
- Alberta Children's Hospital Research Institute
Epilepsy is a common neurological condition that affects 1-2% of the population. Despite dozens of anticonvulsant medications, over a third of patients remain refractory to drugs and our goal is to uncover novel therapies for this patient population. Here, we propose to create a national platform for anticonvulsant drug discovery. This platform will be comprised of a drug screen locally in Calgary using re-purposed drugs, and 50 Associate Members nationwide who will receive newly uncovered therapies to conduct open-label trials in their home institutions. Screening will be conducted using our innovative platform that relies on bioengineered zebrafish models that harbor genetic mutations that align with those observed in human counterparts of the disorder. All promising candidates will be verified in rodent models of epilepsy, prior to entry into the clinic. The project design will allow for the rapid translation of the findings to the clinic in small, open-label trials, which will provide rationale for launching larger, multi-center trials. This proposal has the potential to significantly improve epilepsy drug-discovery and treatment of patients throughout the world.