[PDF][PDF] Phase I/II trial of StemRegenin-1 expanded umbilical cord blood hematopoietic stem cells supports testing as a stand-alone graft

JE Wagner, CG Brunstein, AE Boitano, TE DeFor… - Cell stem cell, 2016 - cell.com
JE Wagner, CG Brunstein, AE Boitano, TE DeFor, D McKenna, D Sumstad, BR Blazar
Cell stem cell, 2016cell.com
Clinical application of umbilical cord blood (UCB) as a source of hematopoietic stem cells for
transplantation is limited by low CD34+ cell dose, increased risk of graft failure, and slow
hematopoietic recovery. While the cell dose limitation is partially mitigated by using two UCB
units, larger-dosed single units would be preferable. We have evaluated the feasibility and
safety of StemRegenin-1 (SR-1), an aryl hydrocarbon receptor antagonist that expands
CD34+ cells, by placing one of the two units in expansion culture. SR-1 produced a 330-fold …
Summary
Clinical application of umbilical cord blood (UCB) as a source of hematopoietic stem cells for transplantation is limited by low CD34+ cell dose, increased risk of graft failure, and slow hematopoietic recovery. While the cell dose limitation is partially mitigated by using two UCB units, larger-dosed single units would be preferable. We have evaluated the feasibility and safety of StemRegenin-1 (SR-1), an aryl hydrocarbon receptor antagonist that expands CD34+ cells, by placing one of the two units in expansion culture. SR-1 produced a 330-fold increase in CD34+ cells and led to engraftment in 17/17 patients at a median of 15 days for neutrophils and 49 days for platelets, significantly faster than in patients treated with unmanipulated UCB. Taken together, the marked expansion, absence of graft failure, and enhanced hematopoietic recovery support testing of SR-1 expansion as a stand-alone graft and suggest it may ameliorate a limitation of UCB transplant.
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