Sickle Cell Treatment: What Does It Mean for Patients?

Sickle cell disease (SCD), which occurs when each parent passes a sickle cell trait to their child, is the most common genetic blood disorder. The disease causes red blood cells, which are normally round and smooth, to become banana-shaped (or “sickle”), hard and sticky when under stress. This shape and texture make it hard for them to flow through the blood vessels, causing the blood vessels to become clogged. Infection, pain and anemia can result, as well as breathing problems such as asthma and recurrent pneumonia.

Until recently the medications used to treat SCD reduced complications and pain, and increased hemoglobin. Because SCD affects a small number of Americans (about 100,000), not much attention has been paid to it historically.

Something exciting happened in December 2023, when the U.S. Food and Drug Administration approved a gene therapy for the treatment of SCD in adult and pediatric patients 12 years and older who require regular red blood cell transfusions. Nationwide Children’s Hospital is one of a handful of facilities in the United States that is certified to have the advanced facilities and specially trained staff which are needed for the administration of the therapy, which edits a patient’s own blood stem cells to produce high levels of the healthy, oxygen-carrying hemoglobin that is typically produced in utero. In partnership with Dayton Children’s Hospital, we recently began providing this long-awaited treatment.

Our new ability to provide this kind of personalized therapy and hope for the future to patients who have had long and difficult journeys managing SCD is thrilling for us.

Once given and as recovery goes on, the gene therapy infusion allows recipients to discontinue some of their medications and return to normal activities after increased fetal hemoglobin levels and energy.