Neoadjuvant anti-OX40 (MEDI6469) therapy in patients with head and neck squamous cell carcinoma activates and expands antigen-specific tumor-infiltrating T cells

Nat Commun. 2021 Feb 16;12(1):1047. doi: 10.1038/s41467-021-21383-1.

Abstract

Despite the success of checkpoint blockade in some cancer patients, there is an unmet need to improve outcomes. Targeting alternative pathways, such as costimulatory molecules (e.g. OX40, GITR, and 4-1BB), can enhance T cell immunity in tumor-bearing hosts. Here we describe the results from a phase Ib clinical trial (NCT02274155) in which 17 patients with locally advanced head and neck squamous cell carcinoma (HNSCC) received a murine anti-human OX40 agonist antibody (MEDI6469) prior to definitive surgical resection. The primary endpoint was to determine safety and feasibility of the anti-OX40 neoadjuvant treatment. The secondary objective was to assess the effect of anti-OX40 on lymphocyte subsets in the tumor and blood. Neoadjuvant anti-OX40 was well tolerated and did not delay surgery, thus meeting the primary endpoint. Peripheral blood phenotyping data show increases in CD4+ and CD8+ T cell proliferation two weeks after anti-OX40 administration. Comparison of tumor biopsies before and after treatment reveals an increase of activated, conventional CD4+ tumor-infiltrating lymphocytes (TIL) in most patients and higher clonality by TCRβ sequencing. Analyses of CD8+ TIL show increases in tumor-antigen reactive, proliferating CD103+ CD39+ cells in 25% of patients with evaluable tumor tissue (N = 4/16), all of whom remain disease-free. These data provide evidence that anti-OX40 prior to surgery is safe and can increase activation and proliferation of CD4+ and CD8+ T cells in blood and tumor. Our work suggests that increases in the tumor-reactive CD103+ CD39+ CD8+ TIL could serve as a potential biomarker of anti-OX40 clinical activity.

Publication types

  • Clinical Trial, Phase I
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biopsy
  • CD8-Positive T-Lymphocytes / immunology
  • Cell Proliferation
  • Clone Cells
  • Disease-Free Survival
  • Epitopes / immunology*
  • Human papillomavirus 16 / physiology
  • Humans
  • Kaplan-Meier Estimate
  • Lymphocyte Activation / immunology
  • Lymphocyte Subsets / immunology
  • Lymphocytes, Tumor-Infiltrating / immunology
  • Neoadjuvant Therapy* / adverse effects
  • Receptors, Antigen, T-Cell / metabolism
  • Receptors, OX40 / antagonists & inhibitors*
  • Receptors, OX40 / metabolism
  • Squamous Cell Carcinoma of Head and Neck / blood
  • Squamous Cell Carcinoma of Head and Neck / immunology*
  • Squamous Cell Carcinoma of Head and Neck / pathology
  • Squamous Cell Carcinoma of Head and Neck / therapy*
  • Stromal Cells / metabolism

Substances

  • Epitopes
  • Receptors, Antigen, T-Cell
  • Receptors, OX40

Associated data

  • ClinicalTrials.gov/NCT02274155