A vaccine targeting mutant IDH1 induces antitumour immunity

Nature. 2014 Aug 21;512(7514):324-7. doi: 10.1038/nature13387. Epub 2014 Jun 25.

Abstract

Monoallelic point mutations of isocitrate dehydrogenase type 1 (IDH1) are an early and defining event in the development of a subgroup of gliomas and other types of tumour. They almost uniformly occur in the critical arginine residue (Arg 132) in the catalytic pocket, resulting in a neomorphic enzymatic function, production of the oncometabolite 2-hydroxyglutarate (2-HG), genomic hypermethylation, genetic instability and malignant transformation. More than 70% of diffuse grade II and grade III gliomas carry the most frequent mutation, IDH1(R132H) (ref. 3). From an immunological perspective, IDH1(R132H) represents a potential target for immunotherapy as it is a tumour-specific potential neoantigen with high uniformity and penetrance expressed in all tumour cells. Here we demonstrate that IDH1(R132H) contains an immunogenic epitope suitable for mutation-specific vaccination. Peptides encompassing the mutated region are presented on major histocompatibility complexes (MHC) class II and induce mutation-specific CD4(+) T-helper-1 (TH1) responses. CD4(+) TH1 cells and antibodies spontaneously occurring in patients with IDH1(R132H)-mutated gliomas specifically recognize IDH1(R132H). Peptide vaccination of mice devoid of mouse MHC and transgenic for human MHC class I and II with IDH1(R132H) p123-142 results in an effective MHC class II-restricted mutation-specific antitumour immune response and control of pre-established syngeneic IDH1(R132H)-expressing tumours in a CD4(+) T-cell-dependent manner. As IDH1(R132H) is present in all tumour cells of these slow-growing gliomas, a mutation-specific anti-IDH1(R132H) vaccine may represent a viable novel therapeutic strategy for IDH1(R132H)-mutated tumours.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibody Specificity
  • Antigens, Neoplasm / genetics
  • Antigens, Neoplasm / immunology
  • Cancer Vaccines / immunology*
  • Cancer Vaccines / therapeutic use*
  • Female
  • Glioma / enzymology
  • Glioma / genetics
  • Glioma / immunology*
  • Glioma / therapy*
  • Histocompatibility Antigens Class II / immunology
  • Humans
  • Immunity, Humoral
  • Immunotherapy / methods
  • Isocitrate Dehydrogenase / genetics*
  • Isocitrate Dehydrogenase / immunology*
  • Male
  • Mice
  • Mutant Proteins / genetics
  • Mutant Proteins / immunology*
  • Mutation
  • T-Lymphocytes, Helper-Inducer / immunology
  • Xenograft Model Antitumor Assays

Substances

  • Antigens, Neoplasm
  • Cancer Vaccines
  • Histocompatibility Antigens Class II
  • Mutant Proteins
  • Isocitrate Dehydrogenase
  • IDH1 protein, human