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Updated: Aug 22, 2025

Megakaryocyte Differentiation and Platelet Formation from Human Cord Blood-derived CD34+ Cells
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[Clinical applications of iPS cell-derived platelets]

Naoshi Sugimoto1, Koji Eto1,2

  • 1Department of Clinical Application, Center for iPS Cell Research and Application (CiRA), Kyoto University.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|November 9, 2022
PubMed

Insights

Induced pluripotent stem cell-derived platelet products (iPSC-PLTs) show promise for treating alloimmune platelet transfusion refractoriness (allo-PTR). A clinical trial confirmed the safety of autologous iPSC-PLTs, paving the way for future advancements.

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.

Keywords:
Novel drugsPlatelet transfusion refractorinessTurbulent-type bioreactorimMKCL

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