Gene therapy-mediated reprogramming tumor infiltrating T cells using IL-2 and inhibiting NF-κB signaling improves the efficacy of immunotherapy in a brain cancer model

Neurotherapeutics. 2012 Oct;9(4):827-43. doi: 10.1007/s13311-012-0144-7.

Abstract

Immune-mediated gene therapy using adenovirus expressing Flt3 ligand and thymidine kinase followed by ganciclovir administration (Flt3/TK) effectively elicits tumor regression in preclinical glioma models. Herein, we assessed new strategies to optimize Flt3L/TK therapeutic efficacy in a refractory RG2 orthotopic glioblastoma model. Specifically, we aimed to optimize the therapeutic efficacy of Flt3L/TK treatment in the RG2 model by overexpressing the following genes within the brain tumor microenvironment: 1) a TK mutant with enhanced cytotoxicity (SR39 mutant TK), 2) Flt3L-IgG fusion protein that has a longer half-life, 3) CD40L to stimulate DC maturation, 4) T helper cell type 1 polarizing dendritic cell cytokines interleukin-12 or C-X-C motif ligand 10 chemokine (CXCL)-10, 5) C-C motif ligand 2 chemokine (CCL2) or C-C motif ligand 3 chemokine (CCL3) to enhance dendritic cell recruitment into the tumor microenvironment, 6) T helper cell type 1 cytokines interferon-γ or interleukin-2 to enhance effector T-cell functions, and 7) IκBα or p65RHD (nuclear factor kappa-B [NF-κB] inhibitors) to suppress the function of Foxp3+ Tregs and enhanced effector T-cell functions. Anti-tumor immunity and tumor specific effector T-cell functions were assessed by cytotoxic T lymphocyte assay and intracellular IFN-γ staining. Our data showed that overexpression of interferon-γ or interleukin-2, or inhibition of the nuclear factor kappa-B within the tumor microenvironment, enhanced cytotoxic T lymphocyte-mediated immune responses and successfully extended the median survival of rats bearing intracranial RG2 when combined with Flt3L/TK. These findings indicate that enhancement of T-cell functions constitutes a critical therapeutic target to overcome immune evasion and enhance therapeutic efficacy for brain cancer. In addition, our study provides novel targets to be used in combination with immune-therapeutic strategies for glioblastoma, which are currently being tested in the clinic.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Antiviral Agents / therapeutic use
  • Brain Neoplasms / genetics
  • Brain Neoplasms / immunology
  • Brain Neoplasms / therapy*
  • Dendritic Cells / immunology
  • Disease Models, Animal
  • Ganciclovir / therapeutic use
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Glioblastoma / genetics
  • Glioblastoma / immunology
  • Glioblastoma / therapy*
  • Humans
  • Immunotherapy / methods*
  • Interleukin-2 / immunology
  • Lymphocytes, Tumor-Infiltrating / immunology
  • Membrane Proteins / therapeutic use
  • NF-kappa B / immunology
  • Rats
  • Recombinant Proteins / therapeutic use
  • Signal Transduction*
  • T-Lymphocytes / immunology*
  • Thymidine Kinase / therapeutic use
  • Tumor Microenvironment / immunology

Substances

  • Antiviral Agents
  • Interleukin-2
  • Membrane Proteins
  • NF-kappa B
  • Recombinant Proteins
  • flt3 ligand protein
  • Thymidine Kinase
  • Ganciclovir