Healthy red blood cells are small and flexible, and they have ability to flow throughout capillaries, the smallest veins in the body. In patients with sickle cell disease, red blood cells (RBC) that express the mutant, sickle form, of hemoglobin, sickle under low oxygen conditions in the peripheral capillaries.
Sickle cell disease damages many organs in patients including the lungs, kidneys, brain, liver, spleen and results in daily pain for patients and progressively, frequent hospitalizations.
Limited Treatment Options
Very little has changed in the treatment of this disease over the last several decades. The only approved drug for its treatment is a re-purposed chemotherapy agent called hydroxyurea. While hydroxyurea has been shown to reduce the signs and symptoms of sickle cell disease, achieving correct dosing of this drug is complex and requires patients endure life-long monitoring on a monthly basis, as a hydroxyurea regimen has the potential to cause neutropenia. Furthermore, concerns continue to exist about its potential impact on fertility with sickle cell disease, potential genetic toxicity to fetuses and potential cancer causing effects.