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Establishing an In Vitro Assay for Effectively Predicting ​Neurotoxicity Risks Using Functional Human iPSC-Derived Neurons​

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Hui Liu, PhD

HiPSC-derived neural cultures have shown great promise as an alternative to animal models and primary cultures for neurotoxicity testing. Here we present the development and validation of a highly efficient and predictive hiPSC-derived neuronal/glial in vitro co-culture approach for quantitative assessment of neurological liabilities of compounds in a medium-throughput setting.  

The system is composed of an established assay setup and optimized data analysis pipeline. We have tested 40 compounds on this assay system so far, for all of which our neurotoxicity risk prediction demonstrated excellent correlation with known clinical or animal data. We believe this may represent the future of fast and high throughput neurotoxicity assessment for early drug discovery for the pharmaceutical industry and the environmental impact of agricultural compounds.