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@ARTICLE{Enssle:301318,
author = {S. Enssle and A. Sax and P. May and N. El Khawanky and N.
Soliman and M. Perl and J. C. Enssle and K. Krey and J.
Ruland$^*$ and A. Pichlmair and F. Bassermann$^*$ and H.
Poeck and S. Heidegger},
title = {{G}asdermin {E} links tumor cell-intrinsic nucleic acid
signaling to proinflammatory cell death for successful
checkpoint inhibitor cancer immunotherapy.},
journal = {OncoImmunology},
volume = {14},
number = {1},
issn = {2162-4011},
address = {Abingdon},
publisher = {Taylor $\&$ Franics},
reportid = {DKFZ-2025-00990},
pages = {2504244},
year = {2025},
abstract = {Durable clinical responses to immune checkpoint inhibitors
(ICI) are limited to a minority of patients, and molecular
pathways that modulate their efficacy remain incompletely
defined. We have recently shown that activation of the
innate RNA-sensing receptor RIG-I and associated apoptotic
tumor cell death can facilitate tumor immunosurveillance and
-therapy, but the mechanism that drives its immunogenicity
remained unclear. We here show that intratumoral activity of
the pore-forming protein gasdermin E (GSDME) links active
RIG-I signaling and apoptotic cell death in tumor cells to
inflammatory pyroptosis. Activation of tumor-intrinsic
RIG‑I triggered cleavage of GSDME, pore formation, loss of
cell membrane integrity and leakage of cytosolic components
from dying tumor cells. Tumor antigen cross-presentation by
dendritic cells and subsequent expansion of cytotoxic T
cells strongly relied on tumor-intrinsic GSDME activity. In
preclinical murine cancer models, defective GSDME signaling
rendered tumors resistant to ICI therapy. Epigenetic
reprogramming with upregulation of Gdsme enhanced the
susceptibility of tumor cells to inflammatory cell death and
immunotherapy. In humans, transcriptome analysis of melanoma
samples showed strong correlation between genetic activity
of the RIG-I and pyroptosis pathways. In melanoma patients,
high transcriptional activity of a pyroptosis gene set was
associated with prolonged survival and beneficial response
to ICI therapy. In summary, our data show that GSDME links
RIG-I and apoptotic signaling to inflammatory cell death,
thereby driving its immunogenicity and responsiveness to
ICI. A deeper understanding of these pathways may allow for
the development of novel combined modality approaches to
improve ICI treatment responses in cancer patients.},
keywords = {Humans / Animals / Mice / Signal Transduction / Pyroptosis:
immunology / Immune Checkpoint Inhibitors: pharmacology /
Immune Checkpoint Inhibitors: therapeutic use /
Immunotherapy: methods / DEAD Box Protein 58: metabolism /
Cell Line, Tumor / Phosphate-Binding Proteins: metabolism /
Melanoma: immunology / Melanoma: pathology / Melanoma:
genetics / Melanoma: drug therapy / Female / Gasdermins /
Receptors, Immunologic / Cancer immunotherapy (Other) /
Gasdermin E (Other) / RIG-I (Other) / apoptosis (Other) /
cancer resistance mechanism (Other) / immune checkpoint
inhibitors (Other) / immunogenic cell death (Other) /
programmed cell death (Other) / pryroptosis (Other) / Immune
Checkpoint Inhibitors (NLM Chemicals) / GSDME protein, human
(NLM Chemicals) / DEAD Box Protein 58 (NLM Chemicals) /
Phosphate-Binding Proteins (NLM Chemicals) / RIGI protein,
human (NLM Chemicals) / Gasdermins (NLM Chemicals) /
Receptors, Immunologic (NLM Chemicals)},
cin = {MU01},
ddc = {610},
cid = {I:(DE-He78)MU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40366863},
doi = {10.1080/2162402X.2025.2504244},
url = {https://inrepo02.dkfz.de/record/301318},
}