USP30 inhibition augments mitophagy to prevent T cell exhaustion

Sci Adv. 2025 Aug 15;11(33):eadv6902. doi: 10.1126/sciadv.adv6902. Epub 2025 Aug 15.

Abstract

The exhaustion of tumor-infiltrating CD8+ T cells poses a substantial challenge in cancer immunotherapy, with mitochondrial health essential for sustaining T cell functionality. Mitophagy, a critical process for mitochondrial quality control, is severely impaired in exhausted CD8+ T cells, yet the underlying mechanisms remain unclear. We identified ubiquitin-specific protease 30 (USP30), a mitochondrial deubiquitinase that inhibits mitophagy, as a key factor up-regulated in exhausted CD8+ T cells. Notably, prolonged antigen stimulation triggers the T cell receptor and nuclear factor of activated T cell 1 signaling, which drives the transcriptional up-regulation of USP30. Excitingly, our interventions targeting USP30 through genetic deletion or pharmacological inhibition effectively restored mitophagy, improved mitochondrial fitness, and rejuvenated CD8+ T cell effector functions. These interventions reinvigorated antitumor responses and markedly suppressed tumor growth. Our findings establish USP30 as a critical regulator of mitophagy and a promising therapeutic target for reversing T cell exhaustion and enhancing the efficacy of cancer immunotherapy.

MeSH terms

  • Animals
  • CD8-Positive T-Lymphocytes* / immunology
  • CD8-Positive T-Lymphocytes* / metabolism
  • Cell Line, Tumor
  • Humans
  • Immunotherapy
  • Lymphocytes, Tumor-Infiltrating / immunology
  • Mice
  • Mitochondria / metabolism
  • Mitochondrial Proteins* / genetics
  • Mitophagy* / drug effects
  • Mitophagy* / genetics
  • Signal Transduction
  • T-Cell Exhaustion
  • Thiolester Hydrolases

Substances

  • Usp30 protein, human
  • Mitochondrial Proteins
  • Thiolester Hydrolases