The results from an eight-year study conducted by researchers from the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the National Heart, Lung, and Blood Institute shed light on a potential cure for sickle cell anemia in adults. The study was conducted at the National Institutes of Health’s Clinical Center in Bethesda, MD, where thirty patients aged 16 to 65 received modified blood stem cell transplants using a combination of the their own stem cells and those of a sibling. Oftentimes introducing donor marrow leads to rejection by the patient’s immune system, so they must take immunosuppressants in order to achieve a successful transplantation. In this case, sickle cell disease was reversed in 87 percent of the patients and 15 patients stopped taking immunosuppressants one year following the treatment; many did not experience rejection for an average of 3.4 years.
“That the patients who discontinued this medication were able to do so safely points to the stability of the partial transplant regimen,” noted Dr. Matthew Hsieh, lead author on the paper. While the study was for the most part successful, four of the subjects did not see any change in their symptoms and one died of a disease-related complication.
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