Development of Minihepcidins for the Treatment of Beta Thalassemia

National Center For Advancing Translational Sciences

Investigators

Abstract

Beta thalassemia is a rare, inherited blood disorder that causes severe anemia and damage to organs. The only current treatment is blood transfusions, and patients often must have many of these procedures. Repeated transfusions can cause too much iron to build up in the body. Patients with beta thalassemia also have reduced levels of a hormone called hepcidin, which helps the body use iron properly. A buildup of iron from transfusions combined with low levels of hepcidin can cause iron overload, which can damage the heart, liver and other tissues. The goal of this project was to produce a treatment that increases levels of hepcidin and lowers the damaging effects of iron in patients with beta thalassemia. The collaboration was to include completion of the following studies: -Formulation development -Pharmacokinetic/absorption, distribution, metabolism and excretion (PK/ADME) studies -Investigational New Drug (IND)-directed toxicology The collaboration ended after Merganser attracted private funding for the agreed upon studies and no longer required NCATS expertise and resources.