[Clinical applications of iPS cell-derived platelets].

Area of Science:

• Regenerative Medicine
• Transfusion Medicine
• Immunology

Background:

• Managing alloimmune platelet transfusion refractoriness (allo-PTR) presents significant challenges in transfusion medicine.
• The COVID-19 pandemic has further complicated traditional blood donation systems.

Purpose of the Study:

• To evaluate the safety and efficacy of induced pluripotent stem cell-derived platelet products (iPSC-PLTs) for patients with allo-PTR.
• To establish a foundation for clinical applications of iPSC-PLTs in transfusion medicine.

Main Methods:

• Establishment of a master cell bank (MCB) of expandable megakaryocyte lines (imMKCLs) from induced pluripotent stem cells (iPSCs).
• Manufacturing of iPSC-PLTs using a novel turbulent-type bioreactor and various compounds.
• Preclinical studies to confirm the quality, safety, and efficacy of iPSC-PLTs.

Main Results:

• The iPLAT1 clinical trial confirmed the safety of autologous iPSC-PLTs in a patient with allo-PTR due to anti-HPA-1a antibodies.
• Extensive preclinical studies demonstrated the quality, safety, and efficacy of iPSC-PLTs.
• Ongoing clinical trials are evaluating allogeneic transfusion of HLA homozygous iPSC-PLTs, with development of universal iPSC-PLTs underway.

Conclusions:

• iPSC-PLTs offer a potential solution for alloimmune platelet transfusion refractoriness.
• The development of iPSC-PLTs is expected to significantly advance transfusion medicine and address critical unmet needs.