A novel approach for new treatments for iron overload diseases

Iron-overload disorders, such as hereditary hemochromatosis (HH) and beta-thalassemia, develops when the body absorbs excessive iron over years. They are quite common with a prevalence of 1/200 in population of northern-European ancestry and 1/500 worldwide respectively. Without treatment, iron-overload can lead to diabetes, heart disease or cirrhosis. Main treatment for HH is phlebotomy and patients need frequent visits in clinic. Pr Leduc’s team in collaboration with GSK is validating the hypothesis that an inhibitor to an enzyme associated with iron homeostasis will efficiently lower iron-overload and act as a new drug. Hit compounds have already been discovered. Pr Leduc’s laboratory has expertise with the therapeutic target and a strong experience in enzyme characterization and inhibition. Combined with GSK’ expertise in drug discovery, this research project focuses on the optimization of identified compounds in term of potency and specificity and also the characterization of their effects on iron regulation

François Béliveau
Faculty Supervisor: 
Richard Leduc
Project Year: