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Small-Molecule Modulators Targeting Coactivator-Associated Arginine Methyltransferase 1 (CARM1) as Therapeutic Agents for Cancer Treatment: Current Medicinal Chemistry Insights and Emerging Opportunities
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    Small-Molecule Modulators Targeting Coactivator-Associated Arginine Methyltransferase 1 (CARM1) as Therapeutic Agents for Cancer Treatment: Current Medicinal Chemistry Insights and Emerging Opportunities
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    • Shuqing Li
      Shuqing Li
      Key Laboratory of Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Jiangxi Provincal Key Laboratory of Tissue Engineering, School of Pharmacy, Gannan Medical University, Ganzhou 341000, P. R. China
      More by Shuqing Li
    • Wanyi Pan
      Wanyi Pan
      Key Laboratory of Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Jiangxi Provincal Key Laboratory of Tissue Engineering, School of Pharmacy, Gannan Medical University, Ganzhou 341000, P. R. China
      More by Wanyi Pan
    • Chengpeng Tao
      Chengpeng Tao
      Key Laboratory of Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Jiangxi Provincal Key Laboratory of Tissue Engineering, School of Pharmacy, Gannan Medical University, Ganzhou 341000, P. R. China
    • Zhihao Hu
      Zhihao Hu
      Key Laboratory of Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Jiangxi Provincal Key Laboratory of Tissue Engineering, School of Pharmacy, Gannan Medical University, Ganzhou 341000, P. R. China
      More by Zhihao Hu
    • Binbin Cheng*
      Binbin Cheng
      School of Medicine, Hubei Polytechnic University, Huangshi 435003, China
      *[email protected]
      More by Binbin Cheng
    • Jianjun Chen*
      Jianjun Chen
      School of Pharmaceutical Sciences, Guangdong Provincial Key Laboratory of New Drug Screening, Southern Medical University, Guangzhou 516000, China
      *[email protected]
      More by Jianjun Chen
    • Xiaopeng Peng*
      Xiaopeng Peng
      Key Laboratory of Prevention and Treatment of Cardiovascular and Cerebrovascular Diseases, Jiangxi Provincal Key Laboratory of Tissue Engineering, School of Pharmacy, Gannan Medical University, Ganzhou 341000, P. R. China
      *[email protected]
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    Journal of Medicinal Chemistry

    Cite this: J. Med. Chem. 2024, XXXX, XXX, XXX-XXX
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    https://doi.org/10.1021/acs.jmedchem.4c02106
    Published November 7, 2024
    © 2024 American Chemical Society

    Abstract

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    Overexpression of coactivator associated arginine methyltransferase 1 (CARM1) is associated with various diseases including cancer. Therefore, CARM1 has emerged as an attractive therapeutic target and a drug response biomarker for anticancer drug discovery. However, the development of conventional CARM1 inhibitors has been hampered by their limited clinical efficacy, acquired resistance, and inability to inhibit nonenzymatic functions of CARM1. To overcome these challenges, new strategies such as isoform-selective inhibitors, dual-acting inhibitors, targeted protein degradation technology (e.g., PROTACs), and even activators, are essential to enhance the anticancer activity of CARM1 modulators. In this perspective, we first summarize the structure and biofunctions of CARM1 and its association with cancer. Next, we focus on the recent advances in CARM1 modulators, including isoform-selective CARM1 inhibitors, dual-target inhibitors, PROTAC degraders, and activators, from the perspectives of rational design, pharmacodynamics, pharmacokinetics, and clinical status. Finally, we discuss the challenges and future directions for CARM1-based drug discovery.

    © 2024 American Chemical Society

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    Journal of Medicinal Chemistry

    Cite this: J. Med. Chem. 2024, XXXX, XXX, XXX-XXX
    Click to copy citationCitation copied!
    https://doi.org/10.1021/acs.jmedchem.4c02106
    Published November 7, 2024
    © 2024 American Chemical Society

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