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Updated: Apr 5, 2026

Advances in Human Induced Pluripotent Stem Cell-Derived Chimeric Antigen Receptor-Expressing Natural Killer Cells
Published on: February 14, 2025
Recent advances in CAR T and CAR NK cell therapy for AML
Makiko Suga1, Naoki Hosen2,3,4,5
1Department of Hematology and Oncology, The University of Osaka Graduate School of Medicine, 2-2,Yamadaoka, Suita, Japan.
Abstract:
CAR T cell therapy has demonstrated remarkable efficacy in treating haematological malignancies, including B-cell lymphomas, B-cell leukaemias, and multiple myeloma. CAR T cell therapy for acute myeloid leukaemia (AML) is also urgently needed. One of the major challenges is identifying AML-specific antigens, since many potential candidates (e.g. CD33, CD123, CLL-1, CD70, TIM-3 and FLT3) are also expressed on normal haematopoietic progenitors. This can lead to 'on-target/off-tumour' toxicity and bone marrow aplasia. CAR NK cell therapy for AML shows promise as a lower-toxicity, off-the-shelf alternative. NK cells have a lower inherent risk of GVHD and may cause milder CRS/ICANS. In this review, we will describe the current status of CAR T/NK cell development for AML. We will also introduce a new CAR T-cell or NK-cell therapy that targets mismatched HLA-DRB1 in patients with AML who have relapsed following an allogeneic haematopoietic stem cell transplant.
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