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Updated: May 23, 2025

Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
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Statin Drugs Are Associated With Response to Immune Checkpoint Blockade in Recurrent/Metastatic Head and Neck Cancer

Tyler J Kristoff1,2, Sean Evans1,2, Pranay Nayi3

  • 1Department of Hematology and Medical Oncology, Emory University, Atlanta, Georgia, USA.

Cancer Medicine
|March 7, 2025
PubMed
Abstract

Insights

Statin use in head and neck squamous cell carcinoma (HNSCC) patients treated with immune checkpoint blockade (ICB) was associated with improved objective response rates. Further research is needed to confirm if specific statins enhance survival outcomes in HNSCC.

Area of Science:

  • Oncology
  • Immunology
  • Pharmacology

Background:

  • Statins, used for hyperlipidemia, show potential in improving survival for solid tumors like head and neck squamous cell carcinoma (HNSCC).
  • Preclinical data suggest statins can enhance anti-tumor immune responses by modulating cholesterol.
  • Retrospective studies indicate statins may improve responses to immune checkpoint blockade (ICB) in other cancers, but this remains unstudied in HNSCC.

Purpose of the Study:

  • To investigate the association between statin use and treatment outcomes in patients with recurrent/metastatic HNSCC receiving ICB.
  • To determine if statin therapy predicts objective response, progression-free survival (PFS), and overall survival (OS) in HNSCC patients treated with ICB.

Main Methods:

  • Retrospective analysis of pharmacy records for 158 patients with recurrent/metastatic HNSCC treated with pembrolizumab or nivolumab (2015-2022).
  • Exclusion of patients receiving fewer than 3 doses of ICB.
  • Univariate and multivariate analyses to assess the correlation between statin use and response metrics (objective response, PFS, OS).

Main Results:

  • Statin therapy was significantly associated with achieving an objective response in HNSCC patients.
  • Rosuvastatin and lovastatin showed the strongest associations with objective response.
  • Multivariate analysis confirmed statins as an independent predictor of objective response, but not PFS or OS.

Conclusions:

  • Statin therapy is an independent predictor of objective response to ICB in HNSCC.
  • Larger, prospective studies are warranted to explore specific statin drugs and their potential to improve survival outcomes in ICB-treated HNSCC patients.
Keywords:
PD‐1PD‐L1head and neck cancerimmune checkpoint blockadeimmunotherapysquamous cell carcinomastatins

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