Novel pharmacologic approaches to hepcidin genetic disorders

Genetic hemochromatosis and thalassemia suffer of iron overload partially caused by a defect of hepcidin, the protein hormone that controls systemic iron homeostasis and availability. On the other hand, hepcidin excess occurs in a genetic disorder with iron deficiency anemia, named IRIDA. The mechanisms controlling the expression of this hormone are now characterized in sufficient detail to allow pharmacological approaches to rescue from both hepcidin excess and deficiency. This project proposes to explore the use in animal models of two drugs already widely used in clinics. Heparin as a strong suppressor of hepcidin expression, that has to be improved by removing its anticoagulant activity, and Tacrolimus, an immunosuppressive molecule that stimulates hepcidin expression possibly even at subclinical concentrations. These drugs might be useful for the treatments of genetic iron-deregulation disorders.