Sickle cell anemia: the impact of discovery, politics, and business.
Sickle cell anemia affects 100,000 African Americans. Frequent blood transfusions to prevent stroke lead to fatal iron-overload. Iron chelation with deferoxamine (DFO) requires expensive infusions. In the present study, we explore the feasibility of using a new delivery system for DFO, i.e., targeted liposome entrapped DFO (LDFO). Our results reveal that our novel formulation lowered the dosage requirements by 50%-75%, allowed for less frequent and shorter treatment durations, eliminating the need for a pump and the standard multi-night administration of DFO. In an iron-overloaded rat model, LDFO reduced iron in the liver, and also improved cardiac function. The lower dosage and improved safety profile makes our novel LDFO delivery system a highly desirable new therapy. Meanwhile, this system will also provide an ideal model for studying the mechanism of Fe overload-induced arrhythmias. The political and economic factors related to health care disparities are also discussed.
Lai-Hua
LH
Xie
Angelia A
AA
Doye
Eric
E
Conley
Judith K
JK
Gwathmey
D000755:Anemia, Sickle Cell / Q000188:drug therapy* / Q000191:economics; D000818:Animals; D017046:Cost Savings; D003676:Deferoxamine / Q000008:administration & dosage* / Q000493:pharmacokinetics; D004195:Disease Models, Animal; D016503:Drug Delivery Systems / Q000379:methods*; D005240:Feasibility Studies; D006207:Half-Life; D006801:Humans; D007501:Iron / Q000032:analysis; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000493:pharmacokinetics; D019190:Iron Overload / Q000517:prevention & control*; D008081:Liposomes; D008099:Liver / Q000737:chemistry / Q000000981:diagnostic imaging; D011057:Politics; D011634:Public Health; D051381:Rats; D017207:Rats, Sprague-Dawley; D015899:Tomography, Emission-Computed, Single-Photon; D014481:United States
D007502:Iron Chelating Agents; D008081:Liposomes; D007501:Iron; D003676:Deferoxamine