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Blood Stem-Cell Transplant Regimen Reverses Sickle Cell Disease In Adults BETHESDA, Md -- December 10, 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 a study published in the December 10 issue of the New England Journal of Medicine. “This trial represents a major milestone in developing a therapy aimed at curing sickle cell disease,” said coauthor Griffin P. Rodgers MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Bethesda, Maryland. “Our modified transplant regimen changes the equation for treating adult patients with severe disease in a safer, more effective way.” 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, the current adult trial sought to reduce toxicity by only partially replacing the bone marrow. The much longer lifespan of normal red blood cells, compared with sickle red blood cells, allows the healthy cells to outlast and completely replace the disease-causing cells. To achieve this goal, the investigators used a low dose of radiation to the whole body and alemtuzumab and sirolimus, to suppress the immune system. The radiation favourably conditions the bone marrow, where donor stem cells move in and begin producing new, healthy red blood cells. After a median of 2.5 years of follow-up, all 10 recipients were alive and sickle cell disease was eliminated in 9. “Our patients have had a remarkable change in their lives,” said principal investigator John F. Tisdale, MD, Molecular and Clinical Hematology Branch, National Instititues of Health, Bethesda, Maryland. “They are no longer being admitted to the hospital for frequent pain crises, they have been able to stop chronic pain medications, go back to school and work, get married and have children. Given these results, our regimen will likely have broad application to other nonmalignant diseases and can be performed at most transplant centres.” The investigators performed human leukocyte antigen (HLA) typing on 112 people with severe sickle cell disease and 169 healthy siblings. Of these, 10 patient-sibling identical matches were found. Blood stem cells collected from the blood of healthy donors were then infused into their siblings, aged 16 to 45 years. This relatively low toxicity regimen allowed patients to become tolerant to the donor immune cells and to achieve stable mixed donor chimerism. This mixture of host and donor cells was sufficient to reverse sickle cell disease. In most patients the donor’s red blood cells completely replaced the recipient’s. “Remarkably, the treatment did not result in graft-versus-host disease for any of the participants,” noted Susan B. Shurin, MD, National Heart, Lung and Blood Institute, Bethesda, Maryland. “This is a very important study because it lessens the toxicity of a therapy known to be highly effective.” One of the main obstacles in treating a larger number of African-Americans with sickle cell disease is the relative lack of an available HLA-matched donor. Dr. Tisdale explained, “Most white Americans can easily find a matched donor in the unrelated bone marrow or cord blood registries; yet when we screened a number of the people in our trial who were without an HLA-matched sibling donor, we could not find a compatible unrelated donor.” However, there may be a way beyond this health care disparity, Tisdale indicated. If participants in the current trial continue to do well with their transplants it may be possible to move to what he calls ‘haplo-transplantation,’ or a half-match from a sibling, parent or child. “This would allow most people with sickle cell disease to be treated and enjoy a better quality of life,” he said. SOURCE: National Institutes of Health E-mail this page to a friend or colleague! To print, use this version