Transplant Reverses Sickle Cell Disease

A specialized bone marrowA spongy tissue that contains blood-producing stem cells, which give rise to many types of blood cells. transplant successfully reversed severe sickle cell disease in adults, in some cases without the need for extensive immune-suppressing drugs. Follow-up testing will be needed to further assess this experimental therapy.

Sickle cell disease affects more than 90,000 Americans, mostly of African descent. It causes red blood cells to become stiff, sticky, and sickle-shaped. The deformed cells can block blood flow, leading to pain, organ damage, and stroke.

Some affected children have been successfully treated with bone marrow transplants—an approach thought to be too toxic for adults. With this method, high doses of chemotherapy destroy all of a child’s bone marrow, which is then replaced with healthy marrow from a donor. Transplant recipients often require harsh immunosuppressants for years.

In the new study, a research team at the NIH Clinical Center tested a modified transplant procedure in 30 adults with severe sickle cell disease. A