Results of a preliminary study by scientists at the National Institutes of Health and Johns Hopkins show that “mini” stem cell transplantation may safely reverse severe sickle cell disease in adults.
The phase I/II study to establish safety of the procedure, published December 10 in the New England Journal of Medicine, describes 10 patients with severe sickle cell disease who received intravenous transplants of blood-forming stem cells. The transplanted stem cells came from the peripheral blood of healthy related donors matched to the patients’ tissue types.
Using this procedure, nine of 10 patients treated have normal red blood cells and reversal of organ damage caused by the disease.
This is a new type of transplant, a little different from the other one. In this study, the patient’s bone marrow is not completely annihilated, but smaller portions of it are killed off, to make room for the newly transplanted cells. The sickled cells co-exist with newly implanted ‘normal’ cells, but the normal cells are in a higher concentration, thereby limiting sickle cell pain and complications.
The study is still in early research phases, but here is another bold step towards finding a solution to sickle cell disease.
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Source: Johns Hopkins Medical Institutions (2009, December 10). ‘Mini’ transplant may reverse severe sickle cell disease. ScienceDaily.