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Syntheses of LSD1/HDAC Inhibitors with Demonstrated Efficacy against Colorectal Cancer: <i>In Vitro</i> and <i>In Vivo</i> Studies Including Patient-Derived Organoids

Po-Yu Chou, Mei-Jung Lai, Kelvin K. Tsai, Li‐Hsin Cheng, Yi-Wen Wu, Mei-Chuan Chen, Shiow‐Lin Pan, Hsiu‐O Ho, Kunal Nepali, Jing‐Ping Liou

2024Journal of Medicinal Chemistry15 citationsDOIOpen Access PDF

Abstract

High Resolution Image Download MS PowerPoint Slide Precedential evidence ascertaining the overexpression of LSD1 and HDACs in colorectal cancer spurred us to design a series of dual LSD1-HDAC inhibitors. Capitalizing on the modular nature of the three-component HDAC inhibitory model, tranylcypromine as a surface recognition motif was appended to zinc-binding motifs via diverse linkers. A compendium of hydroxamic acids was generated and evaluated for in vitro cytotoxicity against HCT-116 cells (human colorectal cancer cell lines). The most potent cell growth inhibitor 2 (GI 50 = 0.495 μMm HCT-116 cells) shows promising anticancer effects by reducing colony formation and inducing cell cycle arrest in HCT-116 cells. It exhibits preferential inhibition of HDAC6, along with potent inhibition of LSD1 compared to standard inhibitors. Moreover, Compound 2 upregulates acetyl-tubulin, acetyl-histone H3, and H3K4me2, indicative of LSD1 and HDAC inhibition. In vivo, it demonstrates significant antitumor activity against colorectal cancer, better than irinotecan, and effectively inhibits growth in patient-derived CRC organoids.

Topics & Concepts

In vivoChemistryIn vitroColorectal cancerOrganoidCancerPharmacologyCancer researchBiochemistryInternal medicineCell biologyMedicineBiotechnologyBiologyHistone Deacetylase Inhibitors ResearchChemical Synthesis and AnalysisAsymmetric Hydrogenation and Catalysis