The Blood Stem Cell Holy Grail?

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Science  10 Sep 2010:
Vol. 329, Issue 5997, pp. 1291-1292
DOI: 10.1126/science.1195173

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Hematopoietic stem cell (HSC) transplantation is one of the major medical discoveries of the 20th century. A recent worldwide survey indicated that in 2006 more than 50,000 HSC transplants were performed (1), saving tens of thousands of lives every year. Unfortunately, many patients in need of HSC transplantation are deprived of this life-saving procedure because of an insufficient number of stem cells in the graft, potentially leading to graft failure, a complication associated with a high mortality rate. This is a frequent problem in patients undergoing autologous (stem cells from one's own marrow) transplantation and in patients receiving an allogeneic (stem cells from a donor) transplant with HSCs derived from human cord blood. Recent clinical studies with cord blood grafts from different donors indicate that even a modest (two- to threefold) ex vivo expansion of HSCs would have a profound clinical impact. On page 1345 of this issue, Boitano et al. (2) report a major stride toward the goal of ex vivo expansion of human HSCs with their exciting discovery of a small molecule called StemRegenin1 (SR1) (see the figure).