MALT1 Inhibitors and Degraders: Strategies for NF-κB-Driven Malignancies

J Med Chem. 2025 Mar 13;68(5):5075-5096. doi: 10.1021/acs.jmedchem.4c02873. Epub 2025 Feb 25.

Abstract

Mucosa-associated lymphoid tissue protein 1 (MALT1), a cysteine protease and the sole paracaspase in humans, plays a pivotal role in the survival and proliferation of NF-κB-dependent malignant cancers, particularly MALT lymphoma and diffuse large B-cell lymphoma (DLBCL). Dysregulated MALT1 activity is implicated in various malignancies, highlighting its importance as a therapeutic target. This Perspective provides an overview of MALT1's structural and functional characteristics, summarizes recent advancements in small-molecule inhibitors and degraders targeting this protein, and discusses compound structures, structure-activity relationship (SAR) analyses, and biological activities. We aim to inform future research efforts to enhance the activity, selectivity, and pharmacological properties of MALT1-targeting compounds, establishing a foundational framework for drug development in this critical area of cancer therapy.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Humans
  • Mucosa-Associated Lymphoid Tissue Lymphoma Translocation 1 Protein* / antagonists & inhibitors
  • Mucosa-Associated Lymphoid Tissue Lymphoma Translocation 1 Protein* / metabolism
  • NF-kappa B* / antagonists & inhibitors
  • NF-kappa B* / metabolism
  • Neoplasms / drug therapy
  • Neoplasms / metabolism
  • Neoplasms / pathology
  • Structure-Activity Relationship

Substances

  • Mucosa-Associated Lymphoid Tissue Lymphoma Translocation 1 Protein
  • NF-kappa B
  • MALT1 protein, human
  • Antineoplastic Agents