Targeting XIAP-coordinated PKC signaling resensitizes PD-1-refractory tumors for rechallenge

Proc Natl Acad Sci U S A. 2026 Jun 9;123(23):e2600501123. doi: 10.1073/pnas.2600501123. Epub 2026 Jun 3.

Abstract

Despite revolutionizing oncology, PD-1/PD-L1-directed immune-checkpoint blockade (ICB) is limited by primary or acquired resistance that is routinely countered-without mechanistic clarity-by empiric rechallenge. Here, we identify substrate-based protein kinase C (PKC) activity as a prognostic biomarker in non-small cell lung cancer and a therapeutic target in anti-PD-1-refractory tumors. Pan-PKC inhibition overcomes anti-PD-1 resistance by inducing Caspase-3/GSDME-dependent immunogenic pyroptotic cell death, promoting tumor-intrinsic PD-L1 degradation via GSK3β activation, and enhancing CD8+ T cell recruitment and effector function through tumor-derived CCL4-CCR5 signaling. Mechanistically, PKC blockade destabilizes XIAP, relieving caspase inhibition, stabilizing PTEN, and suppressing Wnt/β-Catenin-ATF3 signaling to drive CCL4 expression. In resistant models, PKC inhibition synergizes more effectively with anti-CTLA-4 than with anti-PD-1/PD-L1, in association with reduced intratumoral regulatory T cells and reinforcement of CD8+ T cell effector function. These findings define the PKC-XIAP axis as a central regulator of immune resistance and provide a mechanistic rationale for PKC inhibition plus anti-CTLA-4 as a salvage strategy in ICB-refractory cancers.

Keywords: PD-L1; PKC; XIAP; immunotherapy; rechallenge.

MeSH terms

  • Animals
  • B7-H1 Antigen / metabolism
  • CD8-Positive T-Lymphocytes / immunology
  • Carcinoma, Non-Small-Cell Lung* / drug therapy
  • Carcinoma, Non-Small-Cell Lung* / immunology
  • Carcinoma, Non-Small-Cell Lung* / metabolism
  • Carcinoma, Non-Small-Cell Lung* / pathology
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm / drug effects
  • Humans
  • Immune Checkpoint Inhibitors / pharmacology
  • Lung Neoplasms* / drug therapy
  • Lung Neoplasms* / immunology
  • Lung Neoplasms* / metabolism
  • Lung Neoplasms* / pathology
  • Mice
  • Programmed Cell Death 1 Receptor* / antagonists & inhibitors
  • Programmed Cell Death 1 Receptor* / metabolism
  • Protein Kinase C* / antagonists & inhibitors
  • Protein Kinase C* / metabolism
  • Signal Transduction / drug effects
  • X-Linked Inhibitor of Apoptosis Protein* / metabolism

Substances

  • Protein Kinase C
  • Programmed Cell Death 1 Receptor
  • X-Linked Inhibitor of Apoptosis Protein
  • B7-H1 Antigen
  • Immune Checkpoint Inhibitors
  • PDCD1 protein, human
  • CD274 protein, human