STOCKHOLM, June 14, 2013 /PRNewswire/ --
Dr Elizabeta Nemeth will present findings of a study in patients with iron overload disorders that will contribute to the design of a potential new treatment at the 18th Congress of the European Hematology Association in Stockholm.
Iron overload commonly leads to organ damage and even death. Currently available treatments for iron overload are burdensome or cause side effects. The cause of iron overload in diseases such as hereditary hemochromatosis and thalassemia is the deficiency of the hormone hepcidin, which regulates dietary iron absorption and mobilization of iron from stores. Hepcidin deficiency results in excessive iron absorption from the diet and iron loading of vital organs. Hepcidin replacement offers a potential new treatment for iron overload disorders. Natural hepcidin is difficult to synthesize and has unfavorable pharmacological properties. By defining the minimal structure of hepcidin that still retained the hormone activity, we developed minihepcidins, peptide mimics of the hormone, and engineered them to improve their bioavailability and to decrease the cost of production. Using hepcidin knockout mice as a model of the severe form of hereditary hemochromatosis, we demonstrated that minihepcidins completely prevented iron loading of mouse organs. In a mouse model of thalassemia, which is characterized by both anemia and iron overload, minihepcidin not only prevented iron loading, but also improved anemia. Minihepcidins may prove useful for the treatment of human iron overload conditions caused by hepcidin deficiency.
Presenter : Elizabeta Nemeth
Affiliation : University of California, Los Angeles, USA
Topic: POTENTIAL USE OF MINIHEPCIDINS AS THERAPEUTICS (abstract number S1171)
About the EHA Annual Congress
Hematology is a specialty that covers everything to do with blood: its origin in the bone marrow, diseases of blood and their treatments. The latest data on research and development within the spectrum of hematology will be presented. The Congress is aimed at health professionals in the field of hematology. The scientific topics range from stem cell physiology and development, to leukemia, lymphoma, myeloma - diagnosis and treatment; red blood cells -, white blood cells- and platelet disorders; thrombosis and bleeding disorders such as hemophilia and blood transfusion and stem cell transplantation.
Share this article