Current treatments and prospective therapies to manage sickle cell disease

Abstract

Sickle cell disease (SCD) occurs as a result of a genetic mutation that produces hemoglobin S (HbS), an abnormal type of hemoglobin (Hb). The HbS forms sickle-shaped red blood cells that adhere to vessel walls, causing bouts of pain commonly called vaso-occlusive crisis. Fluid intake, ibuprofen, and heating pads treat acute pain effectively. Patients experiencing severe pain require opioids, hydroxyurea, or blood transfusions. Currently, SCD has no known permanent cure; however, patients who undergo hematopoietic stemcell transplantation (HSCT) have shown an elimination of disease. Transplantation replaces sickle stem cells with normal stem cells. Sibling-matched HSCT has the highest chance of engraftment. In a recent discovery, using induced pluripotent stem cells (iPSCs) derived from adult somatic cells corrected the inherited genetic mutation that causes SCD. Gene therapy using iPSCs will become an alternative to HSCT if proven successful in human clinical trials.