I-Corps: Drug discovery pathway for neural cells

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The broader impact/commercial potential of this I-Corps project is the development of novel technology to increase the probability of clinical success in developing novel therapeutics for neurodegenerative disorders and neurotrauma using our novel screening system. This project is expected to bring several more compounds to the preclinical stages in 2022 and move compounds into the clinical stages by 2025. The broader impact of our technology beyond the development of novel therapeutics is the change in paradigm our drug discovery platform will cause. If our method is successful in discovering compounds with high clinical efficacy, many drug developers will switch from cell lines to using clinically relevant primary cell cultures, resulting in an increase in the value of our technology as it will be needed during the active patent window to conduct screenings in adult neural cells.This I-Corps project is based on the development of a novel screening system, the first of its kind, to test compounds directly in adult neural cells. Current neural screens use embryonic or newborn neural cells which leads to a very high clinical failure rate as those cells do not represent the target adult neural population in those with neurological diseases and disorders. The proposed novel screening system can consistently provide 10s of millions of neural cells of high purity and viability to conduct drug screenings directly in adult neural cells in place of embryonic/newborn neural cells. The benefits of this novel screening system in adult neural cells are 1) reduced chances of positive hits not being effective at pre-clinical stages; 2) increased odds of finding age/sex specific compounds; 3) ability to find novel compounds/targets unique to adult neural cells; 4) reduced overall cost of drug development. We are using this technology to discover novel drugs/compounds/targets that can treat neurodegenerative diseases and neurotrauma.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.