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Clinical applications of induced pluripotent stem cell-mediated therapies in cancer: progress, challenges, and future directions.

Regenerative medicine 2026 Vol.21(1) p. 91-110

AlKhafaje Z, Malathi H, Dash A, Precilla SJ, Singh G, Sinha A, Suliman M, Smerat A

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Induced pluripotent stem cells (iPSCs) have emerged as a transformative platform for developing innovative therapies against hematological malignancies.

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APA AlKhafaje Z, Malathi H, et al. (2026). Clinical applications of induced pluripotent stem cell-mediated therapies in cancer: progress, challenges, and future directions.. Regenerative medicine, 21(1), 91-110. https://doi.org/10.1080/17460751.2026.2629846
MLA AlKhafaje Z, et al.. "Clinical applications of induced pluripotent stem cell-mediated therapies in cancer: progress, challenges, and future directions.." Regenerative medicine, vol. 21, no. 1, 2026, pp. 91-110.
PMID 41728817

Abstract

Induced pluripotent stem cells (iPSCs) have emerged as a transformative platform for developing innovative therapies against hematological malignancies. Their pluripotent nature allows differentiation into functional immune effector cells, enabling the generation of iPSC-derived chimeric antigen receptor (CAR)-T and CAR-natural killer (NK) cells as scalable "off-the-shelf" immunotherapies that overcome donor limitations and immune rejection. These engineered immune cells exhibit improved specificity, persistence, and cytotoxicity against leukemia and lymphoma. Moreover, iPSCs hold great promise in regenerative hematology, as they facilitate bone marrow recovery after chemotherapy or radiation-induced damage, thereby enhancing hematopoietic function in cancer survivors. Despite these advances, clinical translation remains challenged by the risks of teratoma formation, manufacturing complexity, and cost. Until these issues are mitigated, widespread clinical application will be thwarted. Yet ongoing research holds the key to unlocking their potential to transform personalized cancer therapy by improving the effectiveness, safety, and availability of innovative immunotherapies.

MeSH Terms

Humans; Induced Pluripotent Stem Cells; Neoplasms; Animals; Immunotherapy; Receptors, Chimeric Antigen