Biopharmaceutical company Fate Therapeutics has begun a Phase II multi-centre study of ProHema in adult patients undergoing double umbilical cord blood transplantation (dUCBT) for hematologic malignancy.
ProHema is an investigational hematopoietic stem cell therapy containing pharmacologically-modulated hematopoietic stem cells (HSCs).
Fate Therapeutics president and CEO Christian Weyer said; "ProHema is being developed with the intent to improve outcomes in patients undergoing cord blood transplantation by facilitating both accelerated engraftment and durable reconstitution using a simple, point-of-care, ex vivo modulation process."
The controlled study will enrol a minimum of 45 patients who will be randomised with ProHema plus an unmanipulated cord blood unit or two unmanipulated cord blood units in 2:1 ratio.
Neutrophil and platelet recovery, incidence of serious infections and graft-versus-host disease, 100-day mortality and relative dominance of ProHema over the unmanipulated cord in contributing to reconstitution will be assessed in the study.
Company anticipates reporting results in 2013.
The Ohio State University blood and marrow transplant programme director and professor of medicine, Phase II study principal investigator Dr Steven Devine said the preclinical data suggests that ProHema has the potential to address several of the unmet medical needs in the evolving field of hematopoietic stem cell transplantation.
"While further investigation is required, an intervention that enables early and durable reconstitution of the best-HLA-matched cord blood unit has the potential to improve patient outcomes and substantially enhance the therapeutic value proposition of cord blood transplant," Devine said.
The safety and efficacy profile of the ProHema was demonstrated in the Phase Ib single-centre study that also established early clinical proof-of-concept.