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Sugimoto, Naoshi; Nakamura, Sou; Shimizu, Shin; Shigemasa, Akiko; Kanda, Junya; Matsuyama, Nobuki; Tanaka, Mitsunobu; Hayashi, Tomoya; Fuchizaki, Akihiro; Nogawa, Masayuki; Watanabe, Naohide; Okamoto, Shinichiro; Handa, Makoto; Sawaguchi, Akira; Momose, Dai; Koh, Ki-Ryang; Tani, Yoshihiko; Takaori-Kondo, Akifumi; Eto, Koji
Blood advances, 12/2022, Volume: 6, Issue: 23Journal Article
•GMP-based iPSC-platelet production using imMKCL master cells from an aplastic anemia patient with alloimmune transfusion refractoriness.•Extensive nonclinical evaluation confirmed the safety, quality, and efficacy of the patient iPSC-platelet product. Display omitted Donor-derived platelets are used to treat or prevent hemorrhage in patients with thrombocytopenia. However, ∼5% or more of these patients are complicated with alloimmune platelet transfusion refractoriness (allo-PTR) due to alloantibodies against HLA-I or human platelet antigens (HPA). In these cases, platelets from compatible donors are necessary, but it is difficult to find such donors for patients with rare HLA-I or HPA. To produce platelet products for patients with aplastic anemia with allo-PTR due to rare HPA-1 mismatch in Japan, we developed an ex vivo good manufacturing process (GMP)–based production system for an induced pluripotent stem cell–derived platelet product (iPSC-PLTs). Immortalized megakaryocyte progenitor cell lines (imMKCLs) were established from patient iPSCs, and a competent imMKCL clone was selected for the master cell bank (MCB) and confirmed for safety, including negativity of pathogens. From this MCB, iPSC-PLTs were produced using turbulent flow bioreactors and new drugs. In extensive nonclinical studies, iPSC-PLTs were confirmed for quality, safety, and efficacy, including hemostasis in a rabbit model. This report presents a complete system for the GMP-based production of iPSC-PLTs and the required nonclinical studies and thus supports the iPLAT1 study, the first-in-human clinical trial of iPSC-PLTs in a patient with allo-PTR and no compatible donor using the autologous product. It also serves as a comprehensive reference for the development of widely applicable allogeneic iPSC-PLTs and other cell products that use iPSC-derived progenitor cells as MCB.
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