Efficacy of systemic temozolomide-activated phage-targeted gene therapy in human glioblastoma

EMBO Mol Med. 2019 Apr;11(4):e8492. doi: 10.15252/emmm.201708492.

Abstract

Glioblastoma multiforme (GBM) is the most lethal primary intracranial malignant neoplasm in adults and most resistant to treatment. Integration of gene therapy and chemotherapy, chemovirotherapy, has the potential to improve treatment. We have introduced an intravenous bacteriophage (phage) vector for dual targeting of therapeutic genes to glioblastoma. It is a hybrid AAV/phage, AAVP, designed to deliver a recombinant adeno-associated virus genome (rAAV) by the capsid of M13 phage. In this vector, dual tumor targeting is first achieved by phage capsid display of the RGD4C ligand that binds the αvβ3 integrin receptor. Second, genes are expressed from a tumor-activated and temozolomide (TMZ)-induced promoter of the glucose-regulated protein, Grp78 Here, we investigated systemic combination therapy using TMZ and targeted suicide gene therapy by the RGD4C/AAVP-Grp78 Firstly, in vitro we showed that TMZ increases endogenous Grp78 gene expression and boosts transgene expression from the RGD4C/AAVP-Grp78 in human GBM cells. Next, RGD4C/AAVP-Grp78 targets intracranial tumors in mice following intravenous administration. Finally, combination of TMZ and RGD4C/AAVP-Grp78 targeted gene therapy exerts a synergistic effect to suppress growth of orthotopic glioblastoma.

Keywords: Grp78; bacteriophage; glioblastoma; targeting; temozolomide.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bacteriophages / genetics*
  • Brain Neoplasms / drug therapy
  • Brain Neoplasms / mortality
  • Brain Neoplasms / pathology
  • Brain Neoplasms / therapy*
  • Cell Line, Tumor
  • Combined Modality Therapy
  • Dependovirus / genetics
  • Endoplasmic Reticulum Chaperone BiP
  • Gene Expression / drug effects
  • Genetic Therapy*
  • Genetic Vectors / genetics
  • Genetic Vectors / metabolism*
  • Glioblastoma / drug therapy
  • Glioblastoma / mortality
  • Glioblastoma / pathology
  • Glioblastoma / therapy*
  • Heat-Shock Proteins / genetics
  • Heat-Shock Proteins / metabolism
  • Herpesvirus 1, Human / genetics
  • Herpesvirus 1, Human / metabolism
  • Humans
  • Kaplan-Meier Estimate
  • Mice
  • Mice, Inbred C57BL
  • Mice, Nude
  • Peptides / chemistry
  • Peptides / genetics
  • Promoter Regions, Genetic
  • Temozolomide / pharmacology
  • Temozolomide / therapeutic use*
  • Thymidine Kinase / genetics
  • Unfolded Protein Response / drug effects
  • Xenograft Model Antitumor Assays

Substances

  • Endoplasmic Reticulum Chaperone BiP
  • HSPA5 protein, human
  • Heat-Shock Proteins
  • Hspa5 protein, mouse
  • Peptides
  • Thymidine Kinase
  • Temozolomide