Sickle cell disease is an inherited condition in which some red blood cells take on a “sickle” shape that can clog up blood vessels, causing pain, organ failure and strokes. Symptoms can be treated with blood transfusions and drugs, but these have side effects and don’t always work.
The only curative treatment is a bone marrow transplant that replaces the blood stem cells, but this has only been performed in children, as clinicians thought adult patients’ bodies would have been too ravaged by the disease to survive a bone marrow transplant — which includes chemotherapy, radiation and drugs that suppress the immune system.
By taking the transplantation down a few notches — low-dose radiation, a different immunosuppressant drug and no chemotherapy — the researchers were able to pull off successful transplantations in 9 out of 10 patients, aged 16 to 45. The less intense procedure left some of the patients’ own faulty bone marrow intact, but there was enough room for donor cells to fill in that symptoms of the disease were gone in the 9 patients with successful grafts, at least for the duration of the 30-month study. All 10 patients are still alive and have not developed graft-versus-host disease, although some of them remain on immunosuppressants. (Reuters)