Polyamine analogues targeting epigenetic gene regulation

Essays Biochem. 2009 Nov 4:46:95-110. doi: 10.1042/bse0460007.

Abstract

Over the past three decades the metabolism and functions of the polyamines have been actively pursued as targets for antineoplastic therapy. Interactions between cationic polyamines and negatively charged nucleic acids play a pivotal role in DNA stabilization and RNA processing that may affect gene expression, translation and protein activity. Our growing understanding of the unique roles that the polyamines play in chromatin regulation, and the discovery of novel proteins homologous with specific regulatory enzymes in polyamine metabolism, have led to our interest in exploring chromatin remodelling enzymes as potential therapeutic targets for specific polyamine analogues. One of our initial efforts focused on utilizing the strong affinity that the polyamines have for chromatin to create a backbone structure, which could be combined with active-site-directed inhibitor moieties of HDACs (histone deacetylases). Specific PAHAs (polyaminohydroxamic acids) and PABAs (polyaminobenzamides) polyamine analogues have demonstrated potent inhibition of the HDACs, re-expression of p21 and significant inhibition of tumour growth. A second means of targeting the chromatin-remodelling enzymes with polyamine analogues was facilitated by the recent identification of flavin-dependent LSD1 (lysine-specific demethylase 1). The existence of this enzyme demonstrated that histone lysine methylation is a dynamic process similar to other histone post-translational modifications. LSD1 specifically catalyses demethylation of mono- and di-methyl Lys4 of histone 3, key positive chromatin marks associated with transcriptional activation. Structural and catalytic similarities between LSD1 and polyamine oxidases facilitated the identification of biguanide, bisguanidine and oligoamine polyamine analogues that are potent inhibitors of LSD1. Cellular inhibition of LSD1 by these unique compounds led to the re-activation of multiple epigenetically silenced genes important in tumorigenesis. The use of these novel polyamine-based HDAC or LSD1 inhibitors represents a highly promising and novel approach to cancer prevention and therapy.

Publication types

  • Review

MeSH terms

  • Amino Acid Sequence
  • Animals
  • DNA / chemistry
  • Epigenesis, Genetic*
  • Gene Expression Regulation*
  • Gene Silencing
  • Histone Deacetylases / metabolism
  • Humans
  • Models, Biological
  • Models, Chemical
  • Molecular Sequence Data
  • Neoplasms / metabolism
  • Polyamines / chemistry*
  • Polyamines / metabolism
  • Polyamines / pharmacology*
  • RNA / chemistry
  • Sequence Homology, Amino Acid

Substances

  • Polyamines
  • RNA
  • DNA
  • Histone Deacetylases