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Discovery of Novel 3-Phenylpiperidine Derivatives Targeting the β-Catenin/B-Cell Lymphoma 9 Interaction as a Single Agent and in Combination with the Anti-PD-1 Antibody for the Treatment of Colorectal Cancer

Hao Zhang, Chenglong Liu, Qiushi Chen, Lian Shen, Wenting Xiao, Jiayi Li, Jiayi Li, Yonghui Wang, Di Zhu, Qingwei Zhang, Jianqi Li, Jianqi Li

2023Journal of Medicinal Chemistry13 citationsDOIOpen Access PDF

Abstract

Direct disruption of the β-catenin/B-cell lymphoma 9 (BCL9) protein–protein interaction (PPI) is a potential strategy for colorectal cancer (CRC) treatment through inhibiting oncogenic Wnt activity. Herein, a series of 3-phenylpiperidine derivatives were synthesized and evaluated as β-catenin/BCL9 PPI inhibitors. Among them, compound 41 showed the best IC 50 (0.72 μM) in a competitive fluorescence polarization assay and a K D value of 0.26 μM for the β-catenin protein. This compound selectively inhibited the growth of CRC cells, suppressed Wnt signaling transactivation, and downregulated oncogenic Wnt target gene expression. In vivo, 41 showed potent anti-CRC activity and promoted the infiltration and function of cytotoxic T lymphocytes while decreasing the infiltration of regulatory T-cells (Tregs). Furthermore, the combination of 41 and the anti-PD-1 antibody (Ab) efficiently enhanced anti-CRC efficacy, first verifying the in vivo efficacy of the small-molecule β-catenin/BCL9 PPI inhibitor and anti-PD-1 Ab in combination.

Topics & Concepts

ChemistryTransactivationWnt signaling pathwayIn vivoCancer researchLymphomaColorectal cancerCateninT cellAntigenCytotoxic T cellAntibodyMolecular biologyIn vitroCancerGene expressionSignal transductionBiochemistryImmune systemImmunologyGeneInternal medicineBiologyBiotechnologyMedicineWnt/β-catenin signaling in development and cancerToxin Mechanisms and ImmunotoxinsGalectins and Cancer Biology
Discovery of Novel 3-Phenylpiperidine Derivatives Targeting the β-Catenin/B-Cell Lymphoma 9 Interaction as a Single Agent and in Combination with the Anti-PD-1 Antibody for the Treatment of Colorectal Cancer | Litcius