Oligonucleotide N3' --> P5' thio-phosphoramidate telomerase template antagonists as potential anticancer agents

Nucleosides Nucleotides Nucleic Acids. 2003 May-Aug;22(5-8):577-81. doi: 10.1081/NCN-120021958.

Abstract

Human telomerase is a reverse transcriptase that is expressed in essentially all cancer cells, but not in the vast majority of normal somatic cells. Therefore, the specific inhibition of telomerase activity in tumors might have significant beneficial therapeutic effects. We have designed and evaluated oligonucleotide N3' --> P5' thio-phosphoramidates as telomerase template antagonists. In biochemical cell-free assays 11-13-mer thio-phosphoramidate oligonucleotides demonstrated sequence specific and dose dependent inhibition of telomerase with pico-molar IC50 values. Optimization of the oligonucleotide sequence and length resulted in the identification of a 13-mer-oligonucleotide thio-phosphoramidate GRN163 as a drug development candidate. In cell cultures GRN163 was able to inhibit telomerase activity in the absence of cationic lipid with approximately 1 microM IC50 values. Telomerase inhibition by GRN163 produced gradual telomere shortening, followed by cellular senescence and/or apoptosis of cancer derived cell lines.

MeSH terms

  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology
  • Base Sequence
  • Cell Survival / drug effects*
  • Drug Design
  • Humans
  • Oligonucleotides / chemical synthesis
  • Oligonucleotides / chemistry
  • Oligonucleotides / pharmacology*
  • Phosphates / chemistry
  • Phosphates / pharmacology*
  • Telomerase / antagonists & inhibitors*
  • Templates, Genetic*
  • Tumor Cells, Cultured

Substances

  • Antineoplastic Agents
  • Oligonucleotides
  • Phosphates
  • Telomerase
  • thiophosphoric acid