Antitumor activity and toxicity of anti-HER2 immunoRNase scFv 4D5-dibarnase in mice bearing human breast cancer xenografts

Invest New Drugs. 2011 Feb;29(1):22-32. doi: 10.1007/s10637-009-9329-2. Epub 2009 Sep 30.

Abstract

Ribonucleases (RNases) are a non-mutagenic alternative to harmful DNA-damaging anticancer drugs. Targeting of RNases with antibodies to surface antigens that are selectively expressed on tumor cells endows specificity to the cytotoxic actions of RNases. Barnase, a ribonuclease from Bacillus amyloliquefaciens, is a promising candidate for targeted delivery to cancer cells because of its insusceptibility to the ubiquitous cytoplasmic ribonuclease inhibitor, and its high stability and catalytic activity. Here, we characterized in vitro and in vivo an immunoRNase, scFv 4D5-dibarnase, which consists of two barnase molecules that are fused serially to the single-chain variable fragment (scFv) of humanized 4D5 antibody. The latter is directed against the extracellular domain of human epidermal growth factor receptor 2 (HER2), a cancer marker that is overexpressed in many human carcinomas. The scFv 4D5-dibarnase exerted a specific cytotoxic effect on HER2-overexpressing SKBR-3 and BT-474 human breast carcinoma cells (IC(50) = 4.1 and 2.4 nM, respectively) via induction of apoptosis. Ten doses of 0.7 mg/kg scFv 4D5-dibarnase to BALB/c nude mice that bore SKBR-3 human breast cancer xenografts resulted in a 76% reduction in tumor growth. A single injection of scFv 4D5-dibarnase at a total course dose of 7 mg/kg did not cause severe side effects in BALB/c nude or BDF1 mice. The cytotoxicity and selectivity of scFv 4D5-dibarnase merit consideration of this immunoRNase as a potent anticancer agent.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Bacterial Proteins
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / pathology
  • Cell Death / drug effects
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Female
  • Humans
  • Injections, Intravenous
  • Kidney / drug effects
  • Kidney / pathology
  • Mice
  • Receptor, ErbB-2 / immunology*
  • Recombinant Fusion Proteins / chemistry
  • Recombinant Fusion Proteins / metabolism*
  • Recombinant Fusion Proteins / pharmacology
  • Recombinant Fusion Proteins / therapeutic use*
  • Ribonucleases / chemistry
  • Ribonucleases / metabolism*
  • Ribonucleases / pharmacology
  • Ribonucleases / therapeutic use*
  • Single-Chain Antibodies / administration & dosage
  • Single-Chain Antibodies / chemistry
  • Single-Chain Antibodies / immunology*
  • Xenograft Model Antitumor Assays*

Substances

  • Antineoplastic Agents
  • Bacterial Proteins
  • Recombinant Fusion Proteins
  • Single-Chain Antibodies
  • immunoRNase scFv 4D5-dibarnase
  • Receptor, ErbB-2
  • Ribonucleases
  • Bacillus amyloliquefaciens ribonuclease