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Updated: Apr 1, 2026

Using Human Induced Pluripotent Stem Cells for the Generation of Tumor Antigen-specific T Cells
Published on: October 24, 2019
Tumorigenicity as a clinical hurdle for pluripotent stem cell therapies
Andrew S Lee1,2,3, Chad Tang1,4, Mahendra S Rao5
1Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, California 94305, USA.
Abstract:
Human pluripotent stem cells (PSCs) are a leading candidate for cell-based therapies because of their capacity for unlimited self renewal and pluripotent differentiation. These advances have recently culminated in the first-in-human PSC clinical trials by Geron, Advanced Cell Technology and the Kobe Center for Developmental Biology for the treatment of spinal cord injury and macular degeneration. Despite their therapeutic promise, a crucial hurdle for the clinical implementation of human PSCs is their potential to form tumors in vivo. In this Perspective, we present an overview of the mechanisms underlying the tumorigenic risk of human PSC-based therapies and discuss current advances in addressing these challenges.
Insights
Human pluripotent stem cells (PSCs) offer therapeutic potential but carry tumor risks. This perspective reviews PSC tumorigenic mechanisms and strategies to overcome these challenges for safe cell therapies.
Area of Science:
- Stem Cell Biology
- Regenerative Medicine
- Cancer Biology
Background:
- Human pluripotent stem cells (PSCs) possess self-renewal and differentiation capabilities, making them promising for cell-based therapies.
- Clinical trials using PSCs are underway for conditions like spinal cord injury and macular degeneration.
- Tumorigenicity remains a significant barrier to the widespread clinical application of PSCs.
Purpose of the Study:
- To provide an overview of the mechanisms driving tumor formation in human PSCs.
- To discuss current strategies for mitigating the tumorigenic risks associated with PSC therapies.
- To highlight advances in ensuring the safety of PSC-based treatments.
Main Methods:
- Review of existing literature on PSC biology and tumorigenesis.
- Analysis of mechanisms underlying PSC-induced tumor formation.
- Synthesis of current research on safety strategies for PSC therapies.
Main Results:
- Identified key molecular pathways and cellular processes contributing to PSC tumorigenicity.
- Highlighted the importance of understanding differentiation status and genetic stability.
- Summarized various approaches to control or eliminate tumorigenic potential.
Conclusions:
- Addressing the tumorigenic risk is critical for realizing the clinical potential of human PSCs.
- Continued research into safety mechanisms will facilitate the translation of PSC therapies.
- Overcoming tumorigenicity will enable the development of effective and safe cell-based treatments.
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