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Published on: September 13, 2019
Tumorigenicity assessment of cell therapy products: The need for global consensus and points to consider
Y Sato1, H Bando2, M Di Piazza3
1Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Kawasaki, Japan.
Abstract:
Pluripotent stem cells offer the potential for an unlimited source for cell therapy products. However, there is concern regarding the tumorigenicity of these products in humans, mainly due to the possible unintended contamination of undifferentiated cells or transformed cells. Because of the complex nature of these new therapies and the lack of a globally accepted consensus on the strategy for tumorigenicity evaluation, a case-by-case approach is recommended for the risk assessment of each cell therapy product. In general, therapeutic products need to be qualified using available technologies, which ideally should be fully validated. In such circumstances, the developers of cell therapy products may have conducted various tumorigenicity tests and consulted with regulators in respective countries. Here, we critically review currently available in vivo and in vitro testing methods for tumorigenicity evaluation against expectations in international regulatory guidelines. We discuss the value of those approaches, in particular the limitations of in vivo methods, and comment on challenges and future directions. In addition, we note the need for an internationally harmonized procedure for tumorigenicity assessment of cell therapy products from both regulatory and technological perspectives.
Insights
Pluripotent stem cells offer unlimited cell therapy potential but raise tumorigenicity concerns. A harmonized international approach is needed for robust risk assessment and evaluation of these advanced therapies.
Area of Science:
- Regenerative Medicine
- Cell Therapy
- Biotechnology
Background:
- Pluripotent stem cells (PSCs) are a promising source for cell therapies.
- Tumorigenicity, arising from undifferentiated or transformed cells, is a key safety concern.
- A lack of global consensus necessitates a case-by-case risk assessment strategy.
Purpose of the Study:
- To critically review in vivo and in vitro tumorigenicity testing methods for PSC-derived products.
- To compare current methods against international regulatory expectations.
- To identify challenges and propose future directions for harmonized assessment.
Main Methods:
- Review of existing scientific literature on tumorigenicity assays.
- Analysis of in vivo and in vitro testing methodologies.
- Comparison with international regulatory guidelines for cell therapy products.
Main Results:
- Current in vivo and in vitro methods for tumorigenicity evaluation have limitations.
- Existing technologies require validation against regulatory standards.
- A case-by-case approach is currently recommended for risk assessment.
Conclusions:
- There is a need for internationally harmonized procedures for tumorigenicity assessment.
- Addressing limitations in current testing methods is crucial for safe cell therapy development.
- Collaboration between regulators and developers is essential for advancing cell therapy safety.
