Site-specific PEGylation endows a mammalian ribonuclease with antitumor activity

Cancer Biol Ther. 2011 Aug 1;12(3):208-14. doi: 10.4161/cbt.12.3.15959. Epub 2011 Aug 1.

Abstract

Mammalian ribonucleases are emerging as cancer chemotherapeutic agents. Their cationicity engenders cell permeability, and their enzymatic activity destroys the biochemical information encoded by RNA. The pharmacologic potential of ribonucleases is, however, obviated by their high sensitivity to a cytosolic inhibitor protein (RI) and their small size, which limits their residence in serum. We reasoned that site specific conjugation of a poly(ethylene glycol) (PEG) chain could both reduce sensitivity to RI and increase serum half-life. We found that appending a PEG moiety can enable bovine pancreatic ribonuclease (RNase A) to evade RI, depending on the site of conjugation and the length and branching of the chain. Although a pendant PEG moiety decreases antiproliferative activity in vitro, PEGylation discourages renal clearance in vivo and leads to nearly complete tumor growth inhibition in a mouse xenograft model. These data demonstrate that a pendant PEG moiety can be beneficial to the action of proteins that act within the cytosol, and that strategic site-specific PEGylation can endow a mammalian ribonuclease with potent antitumor activity.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology*
  • Cattle
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Cytosol
  • Drug Screening Assays, Antitumor
  • Drug Stability
  • Half-Life
  • Humans
  • Male
  • Mice
  • Placental Hormones / pharmacology
  • Polyethylene Glycols / chemistry
  • Protein Conformation
  • Ribonuclease, Pancreatic / antagonists & inhibitors
  • Ribonuclease, Pancreatic / chemistry*
  • Ribonuclease, Pancreatic / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Placental Hormones
  • placental ribonuclease inhibitor
  • Polyethylene Glycols
  • Ribonuclease, Pancreatic