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Blood stem-cell transplant regimen reverses sickle cell disease in adults
Date:12/11/2009

A modified blood adult stem-cell transplant regimen has effectively reversed sickle cell disease in 9 of 10 adults who had been severely affected by the disease, according to results of a National Institutes of Health study in the Dec. 10 issue of the New England Journal of Medicine. The trial was conducted at the NIH Clinical Center in Bethesda, Md., by NIH researchers at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases.

"This trial represents a major milestone in developing a therapy aimed at curing sickle cell disease," said NIDDK Director Griffin P. Rodgers M.D., a co-author of the paper. "Our modified transplant regimen changes the equation for treating adult patients with severe disease in a safer, more effective way."

Sickle cell disease is caused by an altered gene that produces abnormal hemoglobin, the protein in normal red blood cells that carries oxygen throughout the body. When affected red cells lose oxygen, they collapse into a sickle, or C, shape and become stiff and sticky. Clumps of these cells block blood flow and can cause severe pain, organ damage from lack of oxygen, and stroke. Anemia often develops in people with the disease because sickle cells die off quickly and bone marrow does not make new ones fast enough.

In trials by other investigators, nearly 200 children with severe sickle cell disease were cured with bone marrow transplants after undergoing a regimen in which their own marrow was completely destroyed with chemotherapy. That regimen, however, had proven too toxic for adults, who have years of accumulated organ damage from the disease and are less able to tolerate complete marrow transplantation.

In contrast to the established method in children, this adult trial sought to reduce toxicity by only partially replacing the bone marrow. The mu
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Contact: Arthur Stone
niddkmedia@mail.nih.gov
301-496-3583
NIH/National Institute of Diabetes and Digestive and Kidney Diseases
Source:Eurekalert

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