Small cleavable adapters enhance the specific cytotoxicity of a humanized immunotoxin directed against CD64-positive cells

J Immunother. 2008 May;31(4):370-6. doi: 10.1097/CJI.0b013e31816a2d23.

Abstract

The most potent immunotoxins (ITs) developed to date contain bacterial or plant cytotoxic components. As these are potentially immunogenic in man, human proteins are preferred for the long-term treatment of cancer. We have developed the first humanized IT for the treatment of CD64 malignancies such as acute myeloid leukemia. The bacterially expressed IT is composed of a humanized anti-CD64 single chain fragment [h22(scFv)] genetically fused to the human RNase angiogenin. As angiogenin lacks a dedicated translocation domain responsible for the higher potency of bacterial and plant-derived toxins, we have incorporated a recombinant adapter that contains a synthetic translocation domain flanked by proteolytically cleavable endosomal and cytosolic consensus sites. Although insertion of the adapter increased the cytotoxicity by up to 20-fold, serum stability was markedly reduced. Therefore, we designed a modified adapter variant with the endosomal-cleavable peptide deleted. The IT containing the truncated adapter showed significantly higher cytotoxicity than the adapter-free IT and superior serum stability to facilitate the potential applications in patients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Proliferation
  • Cytotoxicity, Immunologic*
  • Epitopes
  • Genetic Engineering
  • Humans
  • Immunoglobulin Fragments / genetics
  • Immunotherapy*
  • Immunotoxins* / administration & dosage
  • Immunotoxins* / genetics
  • Immunotoxins* / metabolism
  • Membrane Transport Modulators / immunology
  • Membrane Transport Modulators / metabolism
  • Neoplasms / immunology
  • Neoplasms / therapy
  • Protein Binding
  • Receptors, IgG* / immunology
  • Receptors, IgG* / metabolism
  • Recombinant Fusion Proteins / administration & dosage
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / metabolism
  • Ribonuclease, Pancreatic / genetics
  • U937 Cells

Substances

  • Epitopes
  • Immunoglobulin Fragments
  • Immunotoxins
  • Membrane Transport Modulators
  • Receptors, IgG
  • Recombinant Fusion Proteins
  • immunoglobulin Fv
  • angiogenin
  • Ribonuclease, Pancreatic