Researchers from the NIH Clinical Center and three NIH institutes, including the NHLBI, have used stem cell transplants to reverse sickle cell disease in severely affected adults. Eighty thousand Americans and millions of people worldwide currently suffer from sickle cell disease, where deformed and oxygen-poor red blood cells can cause anemia, severe pain, organ damage, stroke, and sudden death.
Bone marrow transplants have successfully treated sickle cell disease in children. Very promising trials in children have shown that harsh treatments to allow the transplanted cells to completely replace the bone marrow may not be necessary to achieve great improvement. The harshest transplantation treatment has generally been too toxic for adults, whose organs have accumulated more damage than those of children. The new therapy works by giving milder treatments, only partially replacing adults' bone marrow and immune cells with matched donor stem cells. Nine of the 10 transplant recipients in the study were cured and all were alive after about two and a half years of follow-up. The results are published in the December 10 issue of The New England Journal of Medicine.
This is a remarkable finding that promises to open up curative treatments to adult sickle cell patients who have bone marrow donors and return them to health. The NHLBI continues to support research dedicated to exploring improved treatments with fewer side effects for sickle cell disease and other blood disorders.