"This clinical research study constitutes a major advance in the potential curative treatment for sickle cell disease, which affects one out of 400 African-Americans in the United States," says Myron L. Weisfeldt, M.D., who nominated Brodsky's research for the award and who is the William Osler Professor of Medicine at Johns Hopkins and Director of Medicine for Johns Hopkins Medicine.
"Sickle cell patients have overwhelming symptoms and physical impairments due to strokes, myocardial infarction, kidney failure, and a wide variety of other chronic disabilities," Weisfeldt adds. "The clinical improvement of successful patients in this study has been truly remarkable."
Sickle cell disease is a hereditary illness in which a genetic defect in hemoglobin causes misshapen red blood cells and other defects that ultimately restrict oxygen-rich blood flow into various organs. The condition often results in life-long debilitating pain, organ damage, and a shortened life span.
Until recently, bone marrow transplants were successful in curing some cases of sickle cell disease, but were rarely done because fully matched, eligible donors are exceedingly rare and, even when a match is found, the transplants pose risks.
To overcome the lack of matched donors, Brodsky and colleagues developed techniques that enable sickle cell patients to receive transplants that are only half-identical, or "haploidentical," to their tissue type. The half-matched bone marrow, extracted by needle from a donor's hip bone, can be obtained from a patient's parents, children, and most siblings.
Normally, patients' bodies would reject bone marrow that was not identical to their own. But t
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Johns Hopkins Medicine