Iron overload in Cooley’s anemia

The use of chelating or complexing agents to treat metabolic dysfunction. The classical example is the use of D-penicillamine to treat Wilson s disease, which is caused by an inability of the body to metabolize copper in the normal way. Another example is the use of desferrioxamine for iron overload in Cooley s anemia, which is caused by a fault in hemoglobin synthesis. 

Design of Polymeric Iron Chelators for Treating Iron Overload in Cooley s Anemia... 

Several powerful iron chelators, some recently synthesized specifically for the Cooley s Anemia program, are being tested to ascertain their potential as drugs for use in iron chelation therapy for treating iron overload in Cooley s Anemia. Some of the more promising ones include ... 

In primates, iron, metabolism is highly efficient 13,14, 15,16,17) Little of the metal is absorbed no specific mechanism exists for its elimination. Because it cannot be effectively cleared, the introduction of excess iron 18, 19, 20) into this closed metabolic loop leads to chronic overload and ultimately to peroxidative tissue damage. For example, patients with severe refractory anemia, such as Cooley s anemia ( -thalassemia major), require chronic transfusions, which increase their body iron by 200-250 mg with each unit of blood administered. Unless these individuals receive chelation therapy, they frequently die in their second or third decade from complications associated with iron overload. 

Cooley s anemia (P-thalassemia major), with chronic transfusional iron overload, probably best illustrates the hurdles which must be overcome in chelator design. The lifelong treatment necessary in this disorder dictates that both the long-term toxicity of the drug and the efficiency wiA which the ligand... 

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