According to a story from The Oklahoman, Dr. Roger McEver, who is a scientist and the Vice President of Research at the Oklahoma Medical Research Foundation (OMRF), played a pioneering role in the creation of a new treatment for sickle cell disease by conducting critical research and development. The new drug, called Adakveo, was recently approved by the US Food and Drug Administration (FDA) as a treatment for a serious complication of the disease called sickle cell crisis or pain crisis.
About Sickle Cell Disease
Sickle cell disease is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead become take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. The disease most frequently affects people of African ancestry. Life expectancy is between 40 and 60. To learn more about sickle cell disease, click here.
Treating Sickle Cell Crisis
Previous studies have linked a protein called P-selectin to the development of sickle cell crisis. Dr. McEver founded a biotech company called Selexys in order to further develop an antibody treatment that he began working on in the lab. This antibody is effectively an inhibitor of P-selectin. The company Novartis would ultimately purchase Selexys and the experimental drug following impressive results in the clinical setting in which the drug was able to reduce pain crises by 45 percent. The trial included a total of 198 patients.
Dr. McEver says that Adakveo represents the final result of research that he first started in the 1980s. It is the third medicine that has been developed and approved based on research supported by the OMRF.