Improving therapeutic efficacy of IL-12 intratumoral gene electrotransfer through novel plasmid design and modified parameters

Gene Ther. 2018 Apr;25(2):93-103. doi: 10.1038/s41434-018-0006-y. Epub 2018 Mar 9.

Abstract

The use of immunomodulatory cytokines has been shown effective in regressing a wide range of tumors. However, systemic delivery of recombinant cytokines results in serious, potentially life-threatening, adverse effects. By contrast, nucleic acid transfer via electroporation (EP) is a safe and effective method of delivering plasmid-encoded cytokines to tumors. Intratumoral delivery of IL-12 plasmid DNA by electroporation (IT-pIL12-EP) produced objective response rates in Phase 2 clinical trials in metastatic melanoma. However, only 17.9% of patients receiving IT-pIL12-EP show a complete therapeutic response. Here, we sought to improve the antitumor efficacy of our clinical IT-pIL12-EP plasmid electroporation platform. We evaluated multiple plasmid designs for IL-12 expression. IL-12 expression from a plasmid incorporating a picornavirus-derived co-translational P2A site was the most effective in expressing IL-12p70. In addition, modifying the electroporation parameters improved transfection efficiency and expression of plasmid-derived IL-12p70, as well as its downstream effector IFN-γ in vivo. Finally, using a murine melanoma model that is representative of the intended target patient population, we show that combining modified electroporation conditions with the pIL12-P2A plasmid expression enhances the systemic antitumor response. These improvements to the IT-pIL12-EP platform may improve patient clinical response rates and survival when translated to clinical trials.

MeSH terms

  • Animals
  • CD4-CD8 Ratio
  • Electroporation / methods*
  • Enzyme-Linked Immunosorbent Assay
  • Gene Transfer Techniques*
  • HEK293 Cells
  • Humans
  • Injections, Intralesional
  • Interferon-gamma / blood
  • Interferon-gamma / metabolism
  • Interleukin-12 / biosynthesis
  • Interleukin-12 / genetics*
  • Internal Ribosome Entry Sites
  • Melanoma, Experimental / immunology
  • Melanoma, Experimental / therapy*
  • Mice
  • Picornaviridae / genetics
  • Plasmids*

Substances

  • Internal Ribosome Entry Sites
  • Interleukin-12
  • Interferon-gamma