The transfusion of platelets is a major component of the treatment of cancer patients undergoing chemotherapy and/or bone marrow transplants, trauma patients, sepsis, and battlefield injuries. However, due to a complete reliance upon donors as the only current approved source of platelets, and the strict, short shelf-life and storage conditions of donor- derived platelets, current supplies are not sufficient to meet increased needs as the population ages and may not be readily available in remote locations. We propose to develop a process for manufacturing human platelets under strict FDA guidelines from non-donor sources so that they may be administered to patients in the clinic. Our initial development work will utilize umbilical cord blood as the initial stem cell source for platelet production with the goal of developing methods that can be translated to the use of induce pluripotent stem cells as a renewable cell source that does not require continual donations. We will screen the cells to isolate those most capable of producing the best and largest quantities of platelets while we develop methods that result in the efficient, cost-effective production of large supplies of platelets sufficient for transfusions, 3-5 x 1011. The production of platelets will include the development of a bioreactor system that meets FDA guidelines and requirements for safety, quality, identity, purity, and potency and is capable of producing at least one transfusable unit per disposable bioreactor while limiting the cost of the final product. To ensure the accurate production of a clinically- relevant platelet product, we will test the safety and function of the platelets in a validated animal model and subject our manufactured platelets to clinical platelet functional tests utilized for donor-derived platelet testing. Finally, we will examine the feasibility of different storage options for our manufactured platelet products as they will be slightly different in composition from donor-derived platelets and thereby may not require the storage restrictions set for donor- derived platelets. This could greatly advance both the ready supply and the ability to access platelets in remote locations. KIYATEC?s platelets will reduce morbidity and mortality in multiple patient populations who suffer from bleeding related to platelet quantity or quality defects, including patients with cancer, trauma, sepsis and battlefield injuries.
The transfusion of platelets is a major component of the treatment of cancer patients undergoing chemotherapy and/or bone marrow transplants, trauma patients, sepsis, and battlefield injuries. However, due to a complete reliance upon donors as the only current approved source of platelets, and the strict, short shelf-life and storage conditions of donor- derived platelets, current supplies are not sufficient to meet increased needs as the population ages and may not be readily available in remote locations. We are proposing to overcome the challenges of supply and demand by manufacturing a ready source of platelets from a renewable source of cells that eliminate the need for donors.
