ICL670A: Preclinical Profile

Man is unable to actively eliminate iron from the body, once it has been acquired. Toxic and eventually lethal levels of iron accumulate as a result of repeated transfusions, e.g. in s-thalassemia major, or due to excessive dietary iron uptake in anemias and hereditary hemochromatosis. Excess iron is deposited in the form of hemosiderins (insoluble “iron cores” of ferritin) mainly in the liver, spleen, many endocrine organs and in the myocardium. The exact mechanism of iron damage to these tissues is unknown, but it is established that organ failure correlates with iron burden in these tissues. Except for infectious diseases, cardiac complications are the major cause of death in s-thalassemia major patients.