Granzyme B-H22(scFv), a human immunotoxin targeting CD64 in acute myeloid leukemia of monocytic subtypes

Mol Cancer Ther. 2008 Sep;7(9):2924-32. doi: 10.1158/1535-7163.MCT-08-0554.

Abstract

Acute myeloid leukemia (AML) cells of subtypes M4 and M5 show enhanced expression of CD64 (FcgammaRI), the high-affinity receptor for IgG, which is normally expressed at high levels only on activated cells of the myeloid lineage. CD64 is therefore a prime target for the specific delivery of cytotoxic agents. A promising toxin candidate is granzyme B, a human serine protease originating from cytotoxic granules of CD8+ T lymphocytes and natural killer cells. After evaluating the sensitivity of the AML-related cell line U937 toward cytosolic granzyme B, we genetically fused granzyme B to H22, a humanized single-chain antibody fragment (scFv) specific for CD64, to obtain Gb-H22(scFv), a fusion protein lacking the immunogenic properties of nonhuman immunofusions. Gb-H22(scFv) was successfully expressed in human 293T cells, secreted, and purified from cell culture supernatants. The purified protein bound specifically to CD64+ U937 cells. Despite linkage to the binding domain, the proteolytic activity of functional Gb-H22(scFv) was identical to that of free granzyme B. Target cell-specific cytotoxicity was observed with a half-maximal inhibitory concentration (IC50) between 1.7 and 17 nmol/L. In addition, the induction of apoptosis in U937 cells was confirmed by Annexin A5 staining and the detection of activated caspase-3 in the cytosol. Finally, apoptosis was observed in primary CD64+ AML cells, whereas CD64(-) AML cells were unaffected. This is the first report of a completely human granzyme B-based immunotoxin directed against CD64, with activity against an AML-related cell line and primary AML cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Apoptosis / drug effects
  • Caspase 3 / metabolism
  • Cell Death / drug effects
  • Drug Screening Assays, Antitumor
  • Enzyme Activation / drug effects
  • Granzymes / pharmacology*
  • Humans
  • Immunoglobulin Variable Region / pharmacology*
  • Immunotoxins / pharmacology*
  • Leukemia, Monocytic, Acute / enzymology
  • Leukemia, Monocytic, Acute / immunology
  • Leukemia, Monocytic, Acute / metabolism*
  • Protein Processing, Post-Translational / drug effects
  • Receptors, IgG / antagonists & inhibitors*
  • Recombinant Fusion Proteins / pharmacology*
  • U937 Cells

Substances

  • Immunoglobulin Variable Region
  • Immunotoxins
  • Receptors, IgG
  • Recombinant Fusion Proteins
  • Granzymes
  • Caspase 3