The world’s first clinical trial for an aplastic anemia patient with thrombocytopenia administering platelets generated from autologous iPS cells
On September 21, 2018, the Japanese Ministry of Health, Labor and Welfare announced it had approved a plan for the first-ever use of an experimental therapy on a patient with aplastic anemia and thrombocytopenia, which will involve the infusion of platelets generated from an autologous induced pluripotent stem cells (iPSCs). The patient had a history of rejection reactions to allogeneic platelets.
Nature Editorial previously questioned Japan’s efforts to hasten development and clinical application of iPSC-derived products on both scientific and ethical grounds , especially with respect to the implementation of ‘fast-track’ approval leading to the first-in-human trials of allogeneic iPS stock cells. However, we applaud this trial of iPSC-derived platelets. It stands as a beacon, modeling both ethical and scientific integrity. Future clinical research with iPSC-derived products can look here for guidance.
The current trial was arrived at by a circuitous route. As originally conceived,...
AA: conceptualization, writing—original draft, writing—review and editing. EN: conceptualization, formal analysis, writing—review and editing. NJ: conceptualization, writing—review and editing.
Compliance with ethical standards
Conflict of interest
AA is President of the Japan Association for Bioethics; this paper reflects the author’s personal academic analyses and opinions and does not represent JAB’s official position. EN and NSJ have no conflict of interest to declare.
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