Repurposed anti-cholesterol drug could improve treatment-resistant anemias

Monday, May 11, 2015 - 14:00 in Health & Medicine

Diamond Blackfan anemia (DBA), a rare inherited bone marrow failure syndrome, is usually diagnosed during childhood and is typically treated with glucocorticoids that cause a host of unwanted, often dangerous side effects. Using a mouse model, a research team has now determined that combining the cholesterol-lowering drug fenofibrate with glucocorticoids could allow for dramatically lower steroid doses in the treatment of DBA and other erythropoietin-resistant anemias. These promising results are the foundation for a clinical trial that will begin soon.

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