Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

Nature. 2017 Jul 13;547(7662):222-226. doi: 10.1038/nature23003. Epub 2017 Jul 5.

Abstract

T cells directed against mutant neo-epitopes drive cancer immunity. However, spontaneous immune recognition of mutations is inefficient. We recently introduced the concept of individualized mutanome vaccines and implemented an RNA-based poly-neo-epitope approach to mobilize immunity against a spectrum of cancer mutations. Here we report the first-in-human application of this concept in melanoma. We set up a process comprising comprehensive identification of individual mutations, computational prediction of neo-epitopes, and design and manufacturing of a vaccine unique for each patient. All patients developed T cell responses against multiple vaccine neo-epitopes at up to high single-digit percentages. Vaccine-induced T cell infiltration and neo-epitope-specific killing of autologous tumour cells were shown in post-vaccination resected metastases from two patients. The cumulative rate of metastatic events was highly significantly reduced after the start of vaccination, resulting in a sustained progression-free survival. Two of the five patients with metastatic disease experienced vaccine-related objective responses. One of these patients had a late relapse owing to outgrowth of β2-microglobulin-deficient melanoma cells as an acquired resistance mechanism. A third patient developed a complete response to vaccination in combination with PD-1 blockade therapy. Our study demonstrates that individual mutations can be exploited, thereby opening a path to personalized immunotherapy for patients with cancer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antibodies, Monoclonal / pharmacology
  • Antibodies, Monoclonal / therapeutic use
  • B7-H1 Antigen / immunology
  • CD8 Antigens / immunology
  • Cancer Vaccines / genetics*
  • Cancer Vaccines / immunology*
  • Cancer Vaccines / therapeutic use
  • Epitopes / genetics
  • Epitopes / immunology
  • Humans
  • Immunotherapy / methods
  • Melanoma / genetics
  • Melanoma / immunology*
  • Melanoma / therapy*
  • Mutation / genetics*
  • Neoplasm Metastasis
  • Neoplasm Recurrence, Local / prevention & control
  • Nivolumab
  • Precision Medicine / methods*
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors
  • RNA / genetics*
  • T-Lymphocytes / immunology
  • Vaccination
  • beta 2-Microglobulin / deficiency

Substances

  • Antibodies, Monoclonal
  • B7-H1 Antigen
  • CD274 protein, human
  • CD8 Antigens
  • Cancer Vaccines
  • Epitopes
  • Programmed Cell Death 1 Receptor
  • beta 2-Microglobulin
  • Nivolumab
  • RNA