Phase I Trial of Viral Vector-Based Personalized Vaccination Elicits Robust Neoantigen-Specific Antitumor T-Cell Responses

Clin Cancer Res. 2024 Jun 3;30(11):2412-2423. doi: 10.1158/1078-0432.CCR-23-3940.

Abstract

Purpose: Personalized vaccines targeting multiple neoantigens (nAgs) are a promising strategy for eliciting a diversified antitumor T-cell response to overcome tumor heterogeneity. NOUS-PEV is a vector-based personalized vaccine, expressing 60 nAgs and consists of priming with a nonhuman Great Ape Adenoviral vector (GAd20) followed by boosts with Modified Vaccinia Ankara. Here, we report data of a phase Ib trial of NOUS-PEV in combination with pembrolizumab in treatment-naïve patients with metastatic melanoma (NCT04990479).

Patients and methods: The feasibility of this approach was demonstrated by producing, releasing, and administering to 6 patients 11 of 12 vaccines within 8 weeks from biopsy collection to GAd20 administration.

Results: The regimen was safe, with no treatment-related serious adverse events observed and mild vaccine-related reactions. Vaccine immunogenicity was demonstrated in all evaluable patients receiving the prime/boost regimen, with detection of robust neoantigen-specific immune responses to multiple neoantigens comprising both CD4 and CD8 T cells. Expansion and diversification of vaccine-induced T-cell receptor (TCR) clonotypes was observed in the posttreatment biopsies of patients with clinical response, providing evidence of tumor infiltration by vaccine-induced neoantigen-specific T cells.

Conclusions: These findings indicate the ability of NOUS-PEV to amplify and broaden the repertoire of tumor-reactive T cells to empower a diverse, potent, and durable antitumor immune response. Finally, a gene signature indicative of the reduced presence of activated T cells together with very poor expression of the antigen-processing machinery genes has been identified in pretreatment biopsies as a potential biomarker of resistance to the treatment.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Adenoviridae* / genetics
  • Adenoviridae* / immunology
  • Adult
  • Aged
  • Antigens, Neoplasm* / genetics
  • Antigens, Neoplasm* / immunology
  • CD8-Positive T-Lymphocytes / immunology
  • Cancer Vaccines* / administration & dosage
  • Cancer Vaccines* / immunology
  • Female
  • Genetic Vectors* / administration & dosage
  • Genetic Vectors* / genetics
  • Humans
  • Male
  • Melanoma / immunology
  • Melanoma / therapy
  • Middle Aged
  • Precision Medicine* / methods
  • T-Lymphocytes / immunology
  • Vaccination / methods

Substances

  • Cancer Vaccines
  • Antigens, Neoplasm

Associated data

  • ClinicalTrials.gov/NCT04990479

Grants and funding