LSD1 inhibitors: a patent review (2010-2015)

Expert Opin Ther Pat. 2016 May;26(5):565-80. doi: 10.1517/13543776.2016.1165209. Epub 2016 Mar 28.


Introduction: Lysine demethylase 1 (LSD1) plays an important role in mediating the expression of genes involved in cancer and non-cancer diseases such as viral infections, cardiovascular and neurodegenerative disorders. It is involved in a number of processes from adipogenesis to cell adhesion to viral latency, regulating several cell pathways related with proliferation, development, and cell cycle control. Numerous chemical entities have been studied in recent years and some of them entered the clinical arena.

Areas covered: This review summarizes recent efforts in the drug development of LSD1 inhibitors reported in the patent literature covering the 2010-2015 period, including their potential use as therapeutics in cancerous, neurological, inflammatory, cardiovascular, and viral diseases.

Expert opinion: The development of novel potent and selective LSD1 inhibitors is ongoing, in order to improve their potency and selectivity against specific types of cancer or non-cancer diseases. More in-depth studies are required to assess the role of LSD1 inhibitors in the expression of LSD1 target genes, for a better assessment of the biochemistry underlying their efficacy, and to provide evidence for any possible side effects. Furthermore, an interesting therapeutic approach is the use of LSD1 inhibitors in conjunction with other epidrugs to combine their therapeutic potential, leading to innovative, personalized treatments.

Keywords: Cancer; epigenetics; lysine specific demethylase 1; small molecule inhibitors; viral infections.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Design*
  • Gene Expression Regulation / drug effects*
  • Histone Demethylases / antagonists & inhibitors*
  • Histone Demethylases / metabolism
  • Humans
  • Neoplasms / drug therapy
  • Neoplasms / pathology
  • Patents as Topic
  • Precision Medicine


  • Histone Demethylases
  • KDM1A protein, human