Combined Flt3L/TK gene therapy induces immunological surveillance which mediates an immune response against a surrogate brain tumor neoantigen

Mol Ther. 2011 Oct;19(10):1793-801. doi: 10.1038/mt.2011.77. Epub 2011 Apr 19.

Abstract

Glioblastoma multiforme (GBM) is a primary brain tumor with a median survival of 14.6 months postdiagnosis. The infiltrative nature of GBM prevents complete resection and residual brain tumor cells give rise to recurrent GBM, a hallmark of this disease. Recurrent GBMs are known to harbor numerous mutations/gene rearrangements when compared to the primary tumor, which leads to the potential expression of novel proteins that could serve as tumor neoantigens. We have developed a combined immune-based gene therapeutic approach for GBM using adenoviral (Ads) mediated gene delivery of Herpes Simplex Virus Type 1-thymidine kinase (TK) into the tumor mass to induce tumor cells' death combined with an adenovirus expressing fms-like tyrosine kinase 3 ligand (Flt3L) to recruit dendritic cells (DCs) into the tumor microenvironment. This leads to the induction of specific anti-brain tumor immunity and immunological memory. In a model of GBM recurrence, we demonstrate that Flt3L/TK mediated immunological memory is capable of recognizing brain tumor neoantigens absent from the original treated tumor. These data demonstrate that the Flt3L/TK gene therapeutic approach can induce systemic immunological memory capable of recognizing a brain tumor neoantigen in a model of recurrent GBM.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Antigens, Neoplasm / immunology*
  • Brain Neoplasms / immunology
  • Brain Neoplasms / pathology
  • Brain Neoplasms / therapy*
  • Cell Line, Tumor
  • Genetic Therapy*
  • Glioblastoma / immunology
  • Glioblastoma / pathology
  • Glioblastoma / therapy*
  • Humans
  • Immunologic Memory
  • Interferon-gamma / metabolism
  • T-Lymphocytes / immunology
  • Thymidine Kinase / genetics*
  • fms-Like Tyrosine Kinase 3 / genetics*

Substances

  • Antigens, Neoplasm
  • Interferon-gamma
  • Thymidine Kinase
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3