Presented in collaboration with Be The Match BioTherapies®
The increased understanding of the biological events underlying the generation of platelets from megakaryocytes led to the development of novel strategies that might eventually supplement or replace donor-derived platelet transfusions. Ex vivo manufacturing of platelets from primary hematopoietic stem cells or from induced pluripotent stem cells has been pursued and near-clinical doses of platelets have been achieved by utilizing highly optimized ex vivo cultures and/or state-of-the-art bioreactors. In addition, megakaryocyte-biased cellular products that have the ability to naturally release platelets in the patient’s own body after infusion have been proposed to mitigate repeated platelet transfusions. Clinical implementation of these approaches has the potential to overcome the limitations associated with donor-dependent platelet transfusions such as risk of bacterial contamination, alloimmunization and refractoriness. Furthermore, these novel strategies have the potential to mitigate the increasing demand for donor platelet transfusions which is compounded by dependency on volunteer donors, short storage time and lifespan after infusion as well as shortages in supply due to inclement weather and holidays. This session will review three different approaches to the standard platelet transfusions. Each presenter will highlight the biological basis, process and description of product development as well as manufacturing and regulatory strategies to advance these cellular products to the clinic. Attendees will require a general understanding of human thrombocytopoiesis, i.e. platelet production in vivo and ex vivo, and development of cellular products as therapeutics along with the knowledge of the clinical indications for platelet transfusions and associated risks.
Recognize the process of megakaryocytes and platelets generation from hematopoietic stem cells.
Distinguish between the new megakaryocyte or platelet-based cellular products and the traditional platelet transfusion products.
Appreciate that despite the advances and potential clinical impact of the new cellular products, manufacturing, safety and regulatory features require critical considerations prior to administering such products to patients.