Therapeutic potential of hepcidin - the master regulator of iron metabolism

Pharmacol Res. 2017 Jan;115:242-254. doi: 10.1016/j.phrs.2016.11.010. Epub 2016 Nov 17.


Iron is an essential biogenic element for both prokaryotic and eukaryotic cells. In humans iron is present in hundreds of different metalloproteins. The peptide hormone hepcidin serves as a master regulator of iron homeostasis on the level of single cells and whole organism - by altering cell surface expression of cellular iron exporter - protein ferroportin. Altered levels of extracellular hepcidin lead to pathological conditions such as hemochromatosis and iron loading or, on the other side, iron restrictive anemias. Therapeutic modulation of hepcidin is a new and promising approach to treatment of these conditions. In this review, a summary of the current knowledge of hepcidin function, regulation and pathological involvements are provided, followed by a section covering the therapeutic potential of hepcidin and the current strategies how to modulate its levels and biological functions for therapeutic purposes.

Keywords: Anemia of chronic disease; Hemochromatosis; Hepcidin; Iron; Iron deficiency anemia; Iron overload; Iron therapy; Thalassemia.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cation Transport Proteins / metabolism
  • Hepcidins / pharmacology*
  • Hepcidins / therapeutic use*
  • Homeostasis / drug effects
  • Humans
  • Iron / metabolism*


  • Cation Transport Proteins
  • Hepcidins
  • metal transporting protein 1
  • Iron