Down-modulation of survivin expression and inhibition of tumor growth in vivo by EZN-3042, a locked nucleic acid antisense oligonucleotide

Nucleosides Nucleotides Nucleic Acids. 2010 Feb;29(2):97-112. doi: 10.1080/15257771003597733.

Abstract

Survivin plays an important role in preventing apoptosis and permitting mitosis, and is highly expressed in various human cancers. EZN-3042 is a locked nucleic acid antisense oligonucleotide (LNA-AsODN) against survivin. We report the effects of EZN-3042 in animal models. In a chemical-induced liver regeneration model, treatment with a mouse homolog of EZN-3042 resulted in 80% down-modulation of survivin mRNA. In A549 and Calu-6 lung xenograft models, treatment with EZN-3042 single agent induced 60% down-modulation of survivin mRNA in tumors and 37-45% tumor growth inhibition (TGI). In Calu-6 model, when EZN-3042 was combined with paclitaxel, an 83% TGI was obtained. EZN-3042 is currently being evaluated in a Phase 1 clinical trial as a single agent and in combination with docetaxel.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Combined Chemotherapy Protocols / pharmacology
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Disease Models, Animal
  • Down-Regulation / drug effects
  • Down-Regulation / genetics*
  • Female
  • Gene Expression Regulation, Neoplastic / drug effects
  • Humans
  • Inhibitor of Apoptosis Proteins
  • Liver Regeneration / drug effects
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / genetics
  • Lung Neoplasms / pathology*
  • Maximum Tolerated Dose
  • Mice
  • Mice, Inbred BALB C
  • Mice, Nude
  • Microtubule-Associated Proteins / genetics*
  • Microtubule-Associated Proteins / metabolism
  • Oligonucleotides / pharmacology
  • Oligonucleotides / therapeutic use*
  • Oligonucleotides, Antisense / pharmacology
  • Oligonucleotides, Antisense / therapeutic use*
  • Paclitaxel / pharmacology
  • Paclitaxel / therapeutic use
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Survivin
  • Treatment Outcome
  • Xenograft Model Antitumor Assays*

Substances

  • BIRC5 protein, human
  • EZN 3042
  • Inhibitor of Apoptosis Proteins
  • Microtubule-Associated Proteins
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • RNA, Messenger
  • Survivin
  • locked nucleic acid
  • Paclitaxel