Benzodiazepines interfere with the efficacy of pembrolizumab-based cancer immunotherapy. Results of a nationwide cohort study including over 50,000 participants with advanced lung cancer

Oncoimmunology. 2025 Dec;14(1):2528955. doi: 10.1080/2162402X.2025.2528955. Epub 2025 Jul 4.

Abstract

We previously reported that elevated levels of diazepam binding inhibitor (DBI), also called 'endozepine' because it acts as an endogenous benzodiazepine equivalent on the gamma-aminobutyric acid type A receptor, constitutes a potential risk factor for the diagnosis of non-small cell lung cancer (NSCLC). Antibody-mediated neutralization of DBI improved the immunosurveillance of NSCLC in preclinical models with and without immunotherapy targeting programmed cell death protein 1 (PD-1). A pilot study in a small French-Canadian cohort (n = 205) suggested that benzodiazepine (BZD) use correlates with reduced progression-free survival in NSCLC patients receiving PD-1/PD-L1 blockade. Here, we report a retrospective analysis of the nation-wide French registry of advanced NSCLC patients treated with pembrolizumab. Among the eligible NSCLC patients surviving ≥2 months after treatment initiation (n = 31,479), 37.7% (n = 11,878) received at least two prescriptions of benzodiazepines within 90 days before to 30 days after treatment initiation. Compared to non-users (n = 19,601), BZD users had significantly reduced overall survival (hazard ratio = 1.08, 95% CI: 1.04-1.12, p < 0.001), an effect that persisted after correction using inverse probability of treatment weighting (IPTW) on sociodemographic, clinical, oncologic, and comedication variables. In a subset of 556 patients from the ONCOBIOTICS study, benzodiazepine use was associated with signs of intestinal dysbiosis and alterations in the TOPOSCORE, a prognostic marker linked to poorer outcomes in cancer patients receiving immunotherapy. We conclude that benzodiazepine use may be an independent negative prognostic factor for NSCLC patients under pembrolizumab-based immunotherapy. Future studies must determine whether withdrawal of benzodiazepines or neutralization of DBI improves the clinical response to immunotherapy.

Keywords: ACBP; NSCLC; acyl CoA binding protein; benzodiazepines; lung adenocarcinoma; lung squamous carcinoma; psychotropic agents.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antibodies, Monoclonal, Humanized* / therapeutic use
  • Antineoplastic Agents, Immunological* / therapeutic use
  • Benzodiazepines* / administration & dosage
  • Benzodiazepines* / adverse effects
  • Benzodiazepines* / therapeutic use
  • Carcinoma, Non-Small-Cell Lung* / drug therapy
  • Carcinoma, Non-Small-Cell Lung* / mortality
  • Carcinoma, Non-Small-Cell Lung* / pathology
  • Female
  • France / epidemiology
  • Humans
  • Immune Checkpoint Inhibitors / therapeutic use
  • Immunotherapy* / methods
  • Lung Neoplasms* / drug therapy
  • Lung Neoplasms* / mortality
  • Lung Neoplasms* / pathology
  • Male
  • Middle Aged
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors
  • Retrospective Studies
  • Treatment Outcome

Substances

  • pembrolizumab
  • Antibodies, Monoclonal, Humanized
  • Benzodiazepines
  • Antineoplastic Agents, Immunological
  • Immune Checkpoint Inhibitors
  • Programmed Cell Death 1 Receptor