iPLAT1: the first-in-human clinical trial of iPSC-derived platelets as a phase 1 autologous transfusion study

Author:

Sugimoto Naoshi1ORCID,Kanda Junya2ORCID,Nakamura Sou1ORCID,Kitano Toshiyuki2,Hishizawa Masakatsu2,Kondo Tadakazu2ORCID,Shimizu Shin1,Shigemasa Akiko1,Hirai Hideyo3,Arai Yasuyuki3ORCID,Minami Manabu4ORCID,Tada Harue4,Momose Dai5,Koh Ki-Ryang5,Nogawa Masayuki6ORCID,Watanabe Naohide7,Okamoto Shinichiro7ORCID,Handa Makoto8,Sawaguchi Akira9,Matsuyama Nobuki10,Tanaka Mitsunobu10ORCID,Hayashi Tomoya10ORCID,Fuchizaki Akihiro10ORCID,Tani Yoshihiko10,Takaori-Kondo Akifumi2ORCID,Eto Koji1ORCID

Affiliation:

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

2. 2Department of Hematology, Kyoto University Hospital, Kyoto, Japan

3. 3Department of Clinical Laboratory Medicine, Kyoto University Hospital, Kyoto, Japan

4. 4Institute for Advancement of Clinical and Translational Science (iACT), Kyoto University Hospital, Kyoto, Japan

5. 5Department of Hematology, Osaka General Hospital of West Japan Railway Company, Osaka, Japan

6. 6Central Blood Institute, Blood Service Headquarters, Japanese Red Cross Society, Tokyo, Japan

7. 7Division of Hematology, Keio University School of Medicine, Tokyo, Japan

8. 8Center for Transfusion Medicine & Cell Therapy, Keio University School of Medicine, Tokyo, Japan

9. 9Department of Anatomy, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan

10. 10Japanese Red Cross Kinki Block Blood Center, Osaka, Japan

Abstract

Refractoriness to platelet transfusion is a major problem in a small group of patients, and large-scale manufacturing of clinical grade functional platelets ex vivo has remained an elusive goal. Sugimoto et al report on the results of the first clinical trial of an autologous transfusion of induced pluripotent stem cell (iPSC)-derived platelets in a patient who had severe aplastic anemia but no compatible platelet donor. Using methodology described in a complementary article in Blood Advances, the results provide proof-of-principle and illustrate the challenges to be faced in taking this approach further.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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