Sickle cell anemia: the impact of discovery, politics, and business

J Health Care Poor Underserved. 2013 Nov;24(4 Suppl):147-58. doi: 10.1353/hpu.2014.0008.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Sickle Cell / drug therapy*
  • Anemia, Sickle Cell / economics
  • Animals
  • Cost Savings
  • Deferoxamine / administration & dosage*
  • Deferoxamine / pharmacokinetics
  • Disease Models, Animal
  • Drug Delivery Systems / methods*
  • Feasibility Studies
  • Half-Life
  • Humans
  • Iron / analysis
  • Iron Chelating Agents / administration & dosage*
  • Iron Chelating Agents / pharmacokinetics
  • Iron Overload / prevention & control*
  • Liposomes
  • Liver / chemistry
  • Liver / diagnostic imaging
  • Politics
  • Public Health
  • Rats
  • Rats, Sprague-Dawley
  • Tomography, Emission-Computed, Single-Photon
  • United States

Substances

  • Iron Chelating Agents
  • Liposomes
  • Iron
  • Deferoxamine