Goal 3: Advance Translational Research

Next generation of cellular blood products for clinical use

What advancements in our understanding of hematopoietic stem cell differentiation can be combined with practical developments in novel processing and storage technologies to develop the next generation of transfused blood therapeutics?

 

There is a dearth of tools to evaluate the efficacy of transfused blood products, although this is the most commonly employed therapeutic procedure in the US. There is also no financial incentive to develop the next generation of transfused blood products. This may be in part due to a lack of appreciation in the clinical setting for the reduced safety and efficacy profiles of the current products --as compared to what could potentially be provided. Another hurdle relates to the potential difficulty in retaining a safe base of returning blood donors as demand for current blood products is decreasing in the face of the potential adverse effects on morbidity/mortality that appear to be associated with liberal use of current blood therapeutics.

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

The management of blood transfusions in the clinical setting is undergoing drastic changes in the US with the realization that “more” is not better and that transfusion of red blood cells while life-saving in hemorrhagic settings may be associated with increased morbidity and mortality in more stable patients. Questions have also been raised as to the benefit of prophylactic (most transfusions) versus therapeutic platelet transfusions. Additionally, while red blood cells are being transfused to bring oxygen to tissues in need, and platelets are transfused to prevent/reduce further bleeding, their effectiveness in doing so is not known since often not measurable- hemoglobin levels or platelet corrected count increments are non-sensitive and inadequate effectiveness surrogates. Personalized blood cellular products derived from hematopoietic precursors or resulting from the implementation of novel processing and storage technologies could result in next generation cellular therapeutics that could potentially be used in smaller quantities; deliver specific therapeutics where needed; be effective (e.g., oxygenate hypoxemic tissues); be pathogen-free, less immunogenic, and free of potentially harmful plasticizers; and be available especially in times of emergencies.

Feasibility and challenges of addressing this CQ or CC :

Progress has been made in the last decade in producing blood products from stem cells, including gene-engineered cells. It is now possible to produce red blood cells and platelets from stem cells although the efficiency is low and the safety and efficacy of such products need assessment. New processing (e.g., pathogen-reduction) and storage (e.g. cold storage for platelets, new storage plastic bags) technologies are being considered although progress has been hampered by a lack of financial incentives and regulatory hurdles.

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Idea No. 92