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@ARTICLE{Moser:298948,
author = {L. M. Moser$^*$ and C. Heim and S. E. Koschade$^*$ and P.
Wendel$^*$ and S. Bozkurt and S. Harenkamp and H. Kreyenberg
and M. Merker and C. Münch and E. Gradhand and M.
Vogler$^*$ and E. Ullrich$^*$ and H. Bönig and J.-H.
Klusmann$^*$ and P. Bader$^*$ and W. S. Wels$^*$ and E.
Rettinger$^*$},
title = {{CAR}-{CIK} vs. {CAR}-{T}: benchmarking novel
cytokine-induced killer cells as solid tumor immunotherapy
in {E}rb{B}2+ rhabdomyosarcoma.},
journal = {Frontiers in immunology},
volume = {16},
issn = {1664-3224},
address = {Lausanne},
publisher = {Frontiers Media},
reportid = {DKFZ-2025-00382},
pages = {1485817},
year = {2025},
abstract = {CAR-T cell therapy, though successful in hematologic
malignancies, faces challenges in solid tumors due to
limitations of autologous T cells. Cytokine-induced killer
(CIK) cells can be given safely across allogeneic barriers
and constitute alternative effector cells generated from
healthy donors. CIK cells are a heterogenous population of
predominantly T cells with a mixed natural killer (NK)
phenotype and combine non-MHC-restricted cytotoxicity with
potent anti-tumor capacity of the adaptive immune system.
Here, we characterize and compare efficacy, phenotypic
subpopulations and modes of action of CAR-CIK cells and
conventional CAR-T cells from same-donor samples in ErbB2+
rhabdomyosarcoma (RMS).To benchmark CAR-CIK against
conventional CAR-T cells, effector cells were generated from
same-donor samples and lentivirally transduced with a second
generation CD28-CD3ζ CAR. Effector subpopulations and their
dynamics upon target cell exposure were phenotypically
characterized by flow cytometry. Efficacy was assessed in
human ErbB2+ RMS cancer cell lines and primary patient
samples in vitro and ex vivo using cytotoxicity and spheroid
co-incubation assays. Modes of action were assessed by
comparing cytokine secretion profiles using bead-based
multiplexed flow cytometry and by liquid chromatography mass
spectrometry whole cell proteomics. Finally, we used an in
vivo model of RMS mimicking minimal metastatic residual
disease to compare anti-tumor potency of CAR-CIK vs. CAR-T
cells and to assess their target organ infiltration.In vitro
assays demonstrated superior cytotoxicity of CAR-CIK cells
against RMS cell lines and primary tumor samples. Long-term
co-incubation with tumor spheroids led to expansion of
CAR-CIK cells and enrichment of CD3+CD56+ TNK cells. CAR-CIK
cell cytokine signature showed significantly increased
secretion of effector molecules like interferon-γ, perforin
and granulysin, and lower secretion of Th2 cytokines IL-2,
IL-4 and IL-10. Whole cell proteomics showed corresponding
upregulation of chemokine signaling and NK-cytotoxicity
pathways in CAR-CIK cells. In NSG mice xenografted with
ErbB2+ RMS, a single injection of either CAR-effector cells
strongly impeded metastatic tumor development and
significantly improved survival.Our results demonstrate that
CAR-CIK cells are at least equipotent to CAR-T cells.
Combined with their favorable safety profile and allogeneic
applicability, these findings position CAR-CIK cells as
promising immune effectors for solid tumors.},
keywords = {Humans / Rhabdomyosarcoma: therapy / Rhabdomyosarcoma:
immunology / Cytokine-Induced Killer Cells: immunology /
Immunotherapy, Adoptive: methods / Animals / Receptor,
ErbB-2: immunology / Receptors, Chimeric Antigen: immunology
/ Receptors, Chimeric Antigen: genetics / Receptors,
Chimeric Antigen: metabolism / Mice / Cell Line, Tumor /
Xenograft Model Antitumor Assays / Cytokines: metabolism /
Cytotoxicity, Immunologic / Benchmarking / CAR-T (Other) /
ERBB2 (Other) / cytokine-induced killer cells (CIK) (Other)
/ rhabdomyosarcoma (Other) / solid tumors (Other) /
Receptor, ErbB-2 (NLM Chemicals) / Receptors, Chimeric
Antigen (NLM Chemicals) / ERBB2 protein, human (NLM
Chemicals) / Cytokines (NLM Chemicals)},
cin = {FM01},
ddc = {610},
cid = {I:(DE-He78)FM01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39963129},
pmc = {pmc:PMC11831232},
doi = {10.3389/fimmu.2025.1485817},
url = {https://inrepo02.dkfz.de/record/298948},
}