Sickle Cell Disease
Sickle-cell disease (SCD) is a blood disorder caused by a genetic mutation in hemoglobin. SCD causes refolding of human hemoglobin or “sickling” when oxygen levels are low in the blood. These irregularly shaped sickle cells obstruct blood flow in the vessels that lead to limbs and organs. This results in pain, and can lead to infection and organ damage. The gene mutation that causes SCD is most commonly found in people of African ancestry. In the United States, it is estimated that SCD affects 70,000–100,000 people. It is also prevalent in parts of the developing world, especially in West and Central Africa. There is no cure for sickle cell disease and improvements in treatment are greatly needed.
The Center for World Health and Medicine will focus on the two most critical mechanisms of SCD pathology, sickle cell formation and the subsequent blockages of blood vessels, or vaso-occlusions. The Center for World Health and Medicine will evaluate combination therapies that simultaneously address the complex manifestations of SCD by testing known chemical families and advanced drug candidates against these mechanisms.
Prevention of Vaso-Occlusions: When red blood cells become sickle, they are more rigid and adherent to surrounding blood vessels, which result in blockage of blood flow, especially in small capillaries. The Center for World Health and Medicine is evaluating both an existing drug and experimental compounds for their ability to inhibit the adherence of sickle cells to these surrounding blood vessels.