Reengineering an antiarrhythmic drug using patient hiPSC cardiomyocytes to improve therapeutic potential and reduce toxicity

WL McKeithan, DAM Feyen, AAN Bruyneel… - Cell Stem Cell, 2020 - cell.com
WL McKeithan, DAM Feyen, AAN Bruyneel, KJ Okolotowicz, DA Ryan, KJ Sampson, F Potet…
Cell Stem Cell, 2020cell.com
Modeling cardiac disorders with human induced pluripotent stem cell (hiPSC)-derived
cardiomyocytes is a new paradigm for preclinical testing of candidate therapeutics.
However, disease-relevant physiological assays can be complex, and the use of hiPSC-
cardiomyocyte models of congenital disease phenotypes for guiding large-scale screening
and medicinal chemistry have not been shown. We report chemical refinement of the
antiarrhythmic drug mexiletine via high-throughput screening of hiPSC-CMs derived from …
Summary
Modeling cardiac disorders with human induced pluripotent stem cell (hiPSC)-derived cardiomyocytes is a new paradigm for preclinical testing of candidate therapeutics. However, disease-relevant physiological assays can be complex, and the use of hiPSC-cardiomyocyte models of congenital disease phenotypes for guiding large-scale screening and medicinal chemistry have not been shown. We report chemical refinement of the antiarrhythmic drug mexiletine via high-throughput screening of hiPSC-CMs derived from patients with the cardiac rhythm disorder long QT syndrome 3 (LQT3) carrying SCN5A sodium channel variants. Using iterative cycles of medicinal chemistry synthesis and testing, we identified drug analogs with increased potency and selectivity for inhibiting late sodium current across a panel of 7 LQT3 sodium channel variants and suppressing arrhythmic activity across multiple genetic and pharmacological hiPSC-CM models of LQT3 with diverse backgrounds. These mexiletine analogs can be exploited as mechanistic probes and for clinical development.
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