wagner, John, brunstein, claudio, Boitano, Anthony, DeFor, Todd, McKenna, David, Sumstad, Darin, Blazar, Bruce, Tolar, Jakub, Le, Chap, Jones, Julie, Cooke, Michael and Bleul, Conrad (2015) Safety and Efficacy of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Cells after Myeloablative Conditioning. Cell Stem Cell.

Abstract

Umbilical cord blood (UCB) transplantation is limited by low CD34+ cell dose, increased risk of graft failure and slow hematopoietic recovery. Therefore, we evaluated the feasibility, safety and potential efficacy of StemRegenin-1 (SR-1), an aryl hydrocarbon receptor antagonist, previously shown to enable marked expansion of human CD34+ cells. In a first-in-human clinical trial, SR-1 resulted in a 330-fold (68-609) increase in CD34+ cells and led to engraftment in 17/17 patients at a median of 15 (6-30) days for neutrophils and 49 days (28-136) for platelets. Furthermore, a comparison to 111 control patients treated with an identical conditioning regimen and immunoprophylaxis followed by transplantation of unmanipulated UCB demonstrated significantly faster neutrophil and platelet recovery (p<0.001 and p=0.001, respectively) in recipients of SR-1 expanded UCB. Marked expansion, absence of graft failure and enhanced hematopoietic recovery together suggest that SR-1 may well have ameliorated one of the principal obstacles of UCB transplantation.

Item Type:	Article
Date Deposited:	31 May 2016 23:45
Last Modified:	31 May 2016 23:45
URI:	https://oak.novartis.com/id/eprint/26403