CAR T cell product performance in haematological malignancies before and after marketing authorisation
Open accessAbstract
Chimeric antigen receptor (CAR) T cells represent a potent new approach to treat haematological malignancies. Two CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel, have been approved in Europe and the USA, as well as several other countries, for the treatment of leukaemia and lymphoma. These approvals marked a major milestone in the field of cell and gene therapies. However, the clinical development and regulatory evaluation of these innovative therapies faced several challenges that are considered important lessons learned for future similar products. Here, we examine the products' non-clinical and clinical data packages to outline the challenges encountered during the regulatory evaluation process in Europe, and to provide an update on their performance after authorisation.
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Five CAR T cell products are approved for commercial use and available on the U.S. market: three for B-cell leukemia and lymphoma, one for mantle cell lymphoma, and one for multiple myeloma.
“Currently, five CAR T cell products are approved for commercial use and available on the U.S. market, three for B-cell leukemia and lymphoma (tisagenlecleucel, axicabtagene ciloleucel, lisocabtagene maraleucel), one for mantle cell lymphoma (brexucabtagene autoleucel), and one for multiple myeloma (idecabtagene vicleucel) (2-8).”
From Scalable Manufacturing of CAR T Cells for Cancer Immunotherapy (2021) · cites this paper as [2]
The Introduction identifies tisagenlecleucel and axicabtagene ciloleucel as approved in the USA for B-cell leukaemia or lymphoma indications. The Conclusion calls them the two approved products discussed in this 2020 paper. Our copy does not support the claim that five products were available on the U.S. market, or the stated approvals for the other three products. (LLM, conf=0.90)
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Nanobody-armed T cells endow CAR-T cells with cytotoxicity against lymphoma cells
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CAR T cells outperform CAR NK cells in CAR-mediated effector functions in head-to-head comparison
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A Novel Treatment for Ewing’s Sarcoma: Chimeric Antigen Receptor-T Cell Therapy
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Evidence generation and reproducibility in cell and gene therapy research: A call to action
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Senolytic CAR T Cells in Solid Tumors and Age-Related Pathologies
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Self-citationChimeric Antigen Receptor T-Cell Therapy: The Light of Day for Osteosarcoma
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A Mathematical Model for On-Target Off-Tumor Effect of CAR-T Cells on Gliomas
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An evidence map of randomised controlled trials evaluating genetic therapies
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The mRNA-Based Innovative Strategy: Progress and Challenges
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The Value of CAR-T-cell Immunotherapy in Cancer
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Self-citationUsing real-world evidence in haematology
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Distinct functions of CAR-T cells possessing a dectin-1 intracellular signaling domain
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Toxicity of Chimeric Antigen Receptor T Cells and its Management
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Self-citationCase Report: ITP Treatment After CAR-T Cell Therapy in Patients With Multiple Myeloma
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Global Perspective on the Development of Genetically Modified Immune Cells for Cancer Therapy
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CAR-T Cells for the Treatment of Central Nervous System Tumours: Known and Emerging Neurotoxicities
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Chimeric antigen receptor T-cell lymphoma immunotherapy: the next questions
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Targeting CD10 on B-Cell Leukemia Using the Universal CAR T-Cell Platform (UniCAR)
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Myeloid-derived suppressor cells: Implication in myeloid malignancies and immunotherapy
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Revolutionizing the treatment of cancer using CAR T Cells and Bispecific Antibodies
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Auch natürliche Killerzellen mit CAR ausrüstbar?
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Clinical application of cell‐based therapies opportunities and challenges
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Data from Scalable Manufacturing of CAR T Cells for Cancer Immunotherapy
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The Immune System in Nephrotoxicity
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- Authors
- Elsallab, M., Levine, B. L., Wayne, A. S., & Abou-El-Enein, M.
- Affiliations
- Berlin Institute of Health at Charité - Universitätsmedizin Berlin (DE)
- University of Pennsylvania (US)
- University of Southern California (US)
- Venue
- The Lancet Oncology
- Publisher
- Elsevier BV
- Published
- 2020-02
- Volume
- 21
- Issue
- 2
- Pages
- e104-e116
- ISSN
- 1470-2045 (ISSN-L)
- PMC
- PMC7841982
- OpenAlex
- W3003764567
- Cited by
- 81
- References
- 54
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 14 · 2023: 10 · 2022: 14 · 2021: 22 · 2020: 13
- FWCI
- 1.58
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- The Lancet Oncology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Cancer Institute
- Arab-German Young Academy of Sciences and Humanities
- Bundesministerium für Bildung und Forschung (P30CA014089)
- Topics
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Life Sciences › Immunology and Microbiology › Immunology › Immunotherapy and Immune Responses
- Life Sciences › Immunology and Microbiology › Immunology › T-cell and B-cell Immunology
- Keywords
- CAR T-cell therapy, tisagenlecleucel, axicabtagene ciloleucel, hematologic malignancies, drug approval, regulatory evaluation, clinical data, leukemia, lymphoma, real-world performance
- MeSH terms
- Biological Products, Europe, Humans, Legislation, Drug, Receptors, Antigen, T-Cell, Immunotherapy, Adoptive, Antigens, CD19, Hematologic Neoplasms, Marketing
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