Medicine Grand Rounds, Kenneth Ataga, “Pharmacological Therapies for Sickle Cell Disease: Beyond Hydroxyurea”

Dr. Ataga is the director of the UNC Comprehensive Sickle Cell Program. His clinical research is focused on the vasculopathy of sickle cell disease, with an emphasis on pulmonary hypertension and renal complications, and coagulation activation in sickle cell disease.

In addition, he has a strong research interest in the development of new therapies for sickle cell disease. His clinical trials in sickle cell disease are funded by the National Institutes of Health and the pharmaceutical industry. Results from an international, multicenter phase 2 clinical trial led by Dr. Ataga were published online by the New England Journal of Medicine last year, and were presented at the annual meeting of the American Society of Hematology in San Diego. The trial found that an investigational compound called SeIG1 dramatically reduces sickle cell pain crises.

Sickle cell disease is an inherited, blood disorder that affects one out of every 500 African-Americans. There are approximately 80,000 people in the United States with sickle cell disease. It is also one of the most common genetic diseases, affecting millions of people worldwide. About half of the world’s sickle cell disease population lives in three countries: Nigeria, India and the Democratic Republic of Congo.

Patients with sickle cell disease suffer anemia and complications in which sickle-shaped red cells and white blood cells adhere to small vessels and block blood flow to downstream organs. This vaso-occlusive process results in intense pain and repeated hospitalizations. It also leads to progressive multi-organ dysfunction and premature death.