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

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.

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Publication Details

Journal
Regenerative Medicine
Published
2026-01-02
DOI
https://doi.org/10.1080/17460751.2026.2629846
Primary Topic
CAR-T cell therapy research
Type
article
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Clinical applications of induced pluripotent stem cell-mediated therapies in cancer: progress, challenges, and future directions

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

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article en

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.

Regenerative MedicineVol. 21(1)
Chandigarh University (IN), Al-Ahliyya Amman University (JO), Jain University (IN), Siksha O Anusandhan University (IN), Iraqi University (IQ), Uttaranchal University (IN), Islamic University (BD), Sathyabama Institute of Science and Technology (IN), King Khalid University (SA)
Openalex Percentile: Top 44%
CAR-T cell therapy research
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Clinical applications of induced pluripotent stem cell-mediated therapies in cancer: progress, challenges, and future directions — Zahraa AlKhafaje, Muath Suliman, et al. · Regenerative Medicine (2026) | TGRS Research Map | TGRS