Mechanistic studies of a novel human fusion toxin composed of vascular endothelial growth factor (VEGF)121 and the serine protease granzyme B: directed apoptotic events in vascular endothelial cells

Mol Cancer Ther. 2003 Oct;2(10):949-59.

Abstract

The serine protease granzyme B (GrB; 25 kDa) is capable of inducing apoptosis through both caspase-dependent and caspase-independent mechanisms. We designed a novel vascular-targeting fusion construct designated as GrB/vascular endothelial growth factor (VEGF)121, which is composed of a non-heparin-binding isoform of VEGF and the proapoptotic pathway enzyme GrB fused via a short, flexible tether (G4S). The chimeric fusion gene was then cloned into a bacterial vector, and the protein was expressed in Escherichia coli and purified by nickel-NTA metal affinity chromatography. Western blotting confirmed incorporation of both VEGF121 and GrB proteins into the construct. GrB/VEGF121 specifically bound (ELISA) to porcine aortic endothelial (PAE)/FLK-1 cells overexpressing the FLK-1/KDR receptor but not to cells overexpressing the FLT-1 receptor. Immunofluoresence studies showed that the GrB moiety of GrB/VEGF121 was delivered efficiently and rapidly into the cytosol of PAE/FLK-1 cells but not into that of PAE/FLT-1 cells after 4 h treatment with GrB/VEGF121. Treatment of cells with GrB/VEGF121 showed that the IC50 was approximately 10 nM against PAE/FLK-1 cells; however, there were no cytotoxic effects observed on PAE/FLT-1 cells at doses up to 200 nM. GrB/VEGF121 induced apoptotic events specifically on PAE/FLK-1 as assessed by terminal deoxynucleotidyl transferase-mediated nick end labeling assay, DNA laddering, and cytochrome c release from mitochondria. In addition, the fusion construct mediated the cleavage of caspase-8, caspase-3, and poly(ADP-ribose) polymerase in target endothelial cells within 4 h after treatment. In conclusion, delivery of the human proapoptotic pathway enzyme GrB to tumor vascular endothelial cells or to tumor cells may have significant therapeutic potential and represents a potent new class of targeted therapeutic agents with a unique mechanism of action.

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Aorta / cytology
  • Apoptosis*
  • Base Sequence
  • Blotting, Western
  • Caspase 3
  • Caspase 8
  • Caspases / metabolism
  • Cloning, Molecular
  • Cytochromes c / metabolism
  • DNA / chemistry
  • DNA Fragmentation
  • Dose-Response Relationship, Drug
  • Electrophoresis, Polyacrylamide Gel
  • Endothelium, Vascular / cytology*
  • Endothelium, Vascular / pathology
  • Enzyme-Linked Immunosorbent Assay
  • Escherichia coli / metabolism
  • Genetic Vectors
  • Granzymes
  • Humans
  • In Situ Nick-End Labeling
  • Inhibitory Concentration 50
  • Microscopy, Fluorescence
  • Mitochondria / metabolism
  • Molecular Sequence Data
  • Poly(ADP-ribose) Polymerases / metabolism
  • Protein Isoforms
  • Proto-Oncogene Proteins / metabolism
  • Proto-Oncogene Proteins c-bcl-2*
  • Recombinant Fusion Proteins / metabolism
  • Recombinant Fusion Proteins / pharmacology*
  • Serine Endopeptidases / chemistry*
  • Swine
  • Time Factors
  • Toxins, Biological / pharmacology*
  • Vascular Endothelial Growth Factor A / chemistry*
  • bcl-2-Associated X Protein

Substances

  • Protein Isoforms
  • Proto-Oncogene Proteins
  • Proto-Oncogene Proteins c-bcl-2
  • Recombinant Fusion Proteins
  • Toxins, Biological
  • Vascular Endothelial Growth Factor A
  • bcl-2-Associated X Protein
  • Cytochromes c
  • DNA
  • Poly(ADP-ribose) Polymerases
  • GZMB protein, human
  • Granzymes
  • Serine Endopeptidases
  • CASP3 protein, human
  • CASP8 protein, human
  • Caspase 3
  • Caspase 8
  • Caspases