Study Examines Hepcidin and Iron Overload

November 30, 2010 – A hormone made by the body may be a potential therapeutic tool for the treatment of beta-thalassemia (or “β-thalassemia”) according to scientists involved in a study (“Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice”) which is published in the Journal of Clinical Investigation and which was supported with funding from CAF.
Hepcidin, a hormone found naturally in the bloodstream and acting at the level of the digestive tract, has been known to be at low-levels in patients with β -thalessemia. The authors of the current study believe that boosting levels of hepcidin may actually have a direct effect in relieving anemic patients of some of their body’s iron overload.


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“The major consequence of iron overload is that the lifespan of a red blood cell is half that of a normal red blood cell. These blood cells are not properly formed, are not as healthy as normal blood cells, and therefore cannot properly function,” explains Dr. Stefano Rivella, associate professor of genetic medicine in the Department of Pediatrics at Weill Cornell Medical College.
In the Journal of Clinical Investigation study published online on Nov. 22, Dr. Rivella and his colleagues report that breeding mice that overproduce hepcidin with other mice suffering from β-thalassemia led to offspring that were almost as healthy as normal mice. However, when they crossed the hepcidin-expressing mice with normal mice, hepcidin levels were too high, leading to too much iron removal and an inability to produce healthy red blood cells.
“We see from this evidence that there is a balance in the body – not too much iron and not too little iron – that must be maintained to keep iron levels normal in order to produce normal blood cells,” says Dr. Rivella.
He explains that under normal conditions, hepcidin recognizes when there are not enough red blood cells. In turn, the body will then produce the correct amount of hepcidin, which regulates the amount of iron needed.
“In patients with β-thalassemia, this mechanism isn’t working.  It’s as if the raw materials – the iron – are being sent into a factory, but since no products – blood cells – are being made, more and more iron is being sent in and stored in the body’s organs,” explains Dr. Rivella.
Recently, Dr. Rivella and collaborators at UCLA were awarded a $4 million grant from the National Institutes of Health to test a drug that mimics hepcidin in people with beta-thalassemia and hemochromatosis. They hope to show that boosting hepcidin in the body helps to better treat their iron overload and anemia.
First author of the study is Dr. Sara Gardenghi of Weill Cornell Medical College. Dr. Gadenghi is a recipient of a CAF Medical Research Fellowship; Dr. Rivella is a past recipient of CAF research funding, as is Dr. laura Breda, another author of the study.  Co-authors include Pedro Ramos, Maria Franca Marongiu, Luca Melchiori, Laura Breda, Ella Guy, Kristen Muirhead, Niva Rao, Patricia Giardina and Robert Grady, all from Weill Cornell Medical College; Cindy Roy, from Johns Hopkins University School of Medicine, Baltimore, Md.; Nancy Andrews, from Duke University School of Medicine, Durham, N.C.; Elizabeta Nemeth, from the David Geffen School of Medicine, University of California, Los Angeles, Calif.; Antonia Follenzi, from Albert Einstein College of Medicine, Bronx, N.Y.; Xiuli An and Narla Mohandas, from the Red Cell Physiology Laboratory, New York Blood Center, New York, N.Y.; Yelena Ginzburg, from the Erythropoiesis Laboratory, New York Blood Center, New York, N.Y.; and Eliezer A. Rachmilewitz, from the Hematology Department, Edith Wolfson Medical Center, Holon, Israel.
Research conducted in the study is supported by funding from the National Institutes of Health, the Cooley’s Anemia Foundation, the Associazione Veneta per la Lotta alla Talassemia, the Carlo and Micol Schejola Foundation, the Children’s Cancer and Blood Foundation, and the American Portuguese Biomedical Research Fund.

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