Synthesis of bisethylnorspermine lipid prodrug as gene delivery vector targeting polyamine metabolism in breast cancer

Mol Pharm. 2012 Jun 4;9(6):1654-64. doi: 10.1021/mp300001m. Epub 2012 Apr 30.

Abstract

Progress in the development of nonviral gene delivery vectors continues to be hampered by low transfection activity and toxicity. Here we proposed to develop a lipid prodrug based on a polyamine analogue bisethylnorspermine (BSP) that can function dually as gene delivery vector and, after intracellular degradation, as active anticancer agent targeting dysregulated polyamine metabolism. We synthesized a prodrug of BSP (LS-BSP) capable of intracellular release of BSP using thiolytically sensitive dithiobenzyl carbamate linker. Biodegradability of LS-BSP contributed to decreased toxicity compared with nondegradable control L-BSP. BSP showed a strong synergistic enhancement of cytotoxic activity of TNF-related apoptosis-inducing ligand (TRAIL) in human breast cancer cells. Decreased enhancement of TRAIL activity was observed for LS-BSP when compared with BSP. LS-BSP formed complexes with plasmid DNA and mediated transfection activity comparable to DOTAP and L-BSP. Our results show that BSP-based vectors are promising candidates for combination drug/gene delivery.

Publication types

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

MeSH terms

  • Animals
  • Breast Neoplasms
  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Gene Transfer Techniques
  • Genetic Vectors
  • Humans
  • Magnetic Resonance Spectroscopy
  • Mice
  • Polyamines / chemical synthesis*
  • Polyamines / pharmacology*
  • Prodrugs / chemical synthesis*
  • Prodrugs / pharmacology*
  • Spectrometry, Mass, Electrospray Ionization
  • Spermine / analogs & derivatives*
  • Spermine / chemical synthesis
  • Spermine / pharmacology*
  • TNF-Related Apoptosis-Inducing Ligand / genetics
  • TNF-Related Apoptosis-Inducing Ligand / metabolism
  • TNF-Related Apoptosis-Inducing Ligand / pharmacology

Substances

  • Polyamines
  • Prodrugs
  • TNF-Related Apoptosis-Inducing Ligand
  • TNFSF10 protein, human
  • Spermine