Eradicating Drug-tolerant Persister Cells in EGFR-Mutated Non-Small Cell Lung Cancer by Targeting TROP2 with CAR-T Cellular Therapy

Cancer Discov. 2025 Nov 3;15(11):2235-2250. doi: 10.1158/2159-8290.CD-24-1515.

Abstract

EGFR tyrosine kinase inhibitors have dramatically improved outcomes for patients with EGFR-mutated non-small cell lung cancer (NSCLC), but relapse frequently occurs because of drug-tolerant persister (DTP) cells that can evolve and develop diverse mechanisms of drug resistance. In samples from patients with EGFR-mutated NSCLC treated with EGFR tyrosine kinase inhibitors in the neoadjuvant setting, we observed enriched expression of the cell surface protein TROP2, a target of clinically active antibody-drug conjugates (ADC). We confirmed these findings across multiple EGFR-mutated NSCLC cell line and patient-derived xenograft models treated with osimertinib in vivo. Treatment with the TROP2 ADC sacituzumab govitecan at the time of osimertinib-induced minimal residual disease only modestly delayed tumor recurrence in vivo, whereas a single infusion of sacituzumab-based TROP2-directed chimeric antigen receptor (CAR) T cells significantly prolonged relapse-free survival, with evidence of cure. These data highlight the potential of engineering TROP2 CAR T-cell therapy to eliminate EGFR DTPs in patients.

Significance: We provide a rationale for targeting TROP2 in EGFR-mutated NSCLC DTPs. In contrast to TROP2 ADC therapy, targeting of TROP2 with CAR-T cells can eliminate osimertinib-induced DTPs in vivo, revealing the promise of developing novel TROP2-based CAR-T cells to promote durable response and prevent disease relapse in patients.

MeSH terms

  • Acrylamides / pharmacology
  • Aniline Compounds / pharmacology
  • Animals
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Antigens, Neoplasm* / immunology
  • Antigens, Neoplasm* / metabolism
  • Camptothecin / analogs & derivatives
  • Carcinoma, Non-Small-Cell Lung* / drug therapy
  • Carcinoma, Non-Small-Cell Lung* / genetics
  • Carcinoma, Non-Small-Cell Lung* / immunology
  • Carcinoma, Non-Small-Cell Lung* / pathology
  • Carcinoma, Non-Small-Cell Lung* / therapy
  • Cell Adhesion Molecules* / antagonists & inhibitors
  • Cell Adhesion Molecules* / metabolism
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm
  • ErbB Receptors / genetics
  • Female
  • Humans
  • Immunoconjugates / pharmacology
  • Immunotherapy, Adoptive* / methods
  • Indoles
  • Lung Neoplasms* / drug therapy
  • Lung Neoplasms* / genetics
  • Lung Neoplasms* / immunology
  • Lung Neoplasms* / pathology
  • Lung Neoplasms* / therapy
  • Mice
  • Mutation
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrimidines
  • Receptors, Chimeric Antigen
  • Xenograft Model Antitumor Assays

Substances

  • TACSTD2 protein, human
  • ErbB Receptors
  • Antigens, Neoplasm
  • Cell Adhesion Molecules
  • EGFR protein, human
  • osimertinib
  • Aniline Compounds
  • Immunoconjugates
  • Protein Kinase Inhibitors
  • Acrylamides
  • sacituzumab govitecan
  • Receptors, Chimeric Antigen
  • Antibodies, Monoclonal, Humanized
  • Camptothecin
  • Indoles
  • Pyrimidines