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@ARTICLE{Monnikhof:303120,
author = {M. Monnikhof and M. Y. Schakelaar and C. Meulenbroeks and
M. Quist and A. Perzolli and A. Selten and C. J. M. Koster
and D. S. C. Maassen and A. Montoro Canelo and M. Fredriks
and M. J. A. Koppers and K. Clevers and J. Klein and V.
Kaludjerovic and J. Meeldijk and E. W. Pijnappel and H. G.
Rebel and S. van Kempen and S. Crnko and T. Koorman and A.
Federico and F. Valzano and P. Wesseling and F. G. J.
Calkoen and J. van der Lugt and T. Ten Broeke and M.
Kool$^*$ and N. Bovenschen},
title = {{D}ual targeting of {CD}155/{TIGIT} and {PD}-{L}1/{PD}-1
immune checkpoints potentiates {NK} cell-mediated
cytotoxicity in medulloblastoma.},
journal = {Neuro-oncology advances},
volume = {7},
number = {1},
issn = {2632-2498},
address = {Oxford},
publisher = {Oxford University Press},
reportid = {DKFZ-2025-01539},
pages = {vdaf099},
year = {2025},
abstract = {Medulloblastoma (MB) is one of the most prevalent pediatric
brain malignancies and makes up approximately $20\%$ of all
primary brain tumors in children. Current treatment options
are not curative for approximately $30\%$ of patients and
leave survivors with an impaired quality of life. Immune
checkpoint inhibition can offer a novel targeted therapy but
largely remains understudied in MB. The aim of this study
was to determine whether dual immune checkpoint inhibition
can be used as a novel targeted therapy in MB.We utilized
single cell and single nuclei sequencing datasets of primary
MB tumors, established Group 3 and Sonic Hedgehog MB cell
lines and MB patient-derived xenograft (PDX) organoid
models, and primary patient-derived MB tissue of all
subtypes to study immune checkpoints and their blockade to
target MB.We identified the expression of immune checkpoint
protein CD155 on MB tumor cells and the expression of its
inhibitory binding partner TIGIT on immune cells of MB
patient-derived tissues, cell lines, and PDX MB organoids.
In addition, while MB shows weak, if any, PD-L1 protein
expression, we found that MB cells can upregulate PD-L1
expression upon stimulation by natural killer (NK) cells or
interferon-γ as a putative immune evasive strategy.
Subsequent immunotherapeutic interventions with FDA-approved
antibodies Tiragolumab (anti-TIGIT), Durvalumab (anti-PD-1),
and their combination potentiated primary NK cell activation
and killing of MB cell lines and PDX-derived MB
organoids.These data propose a translatable and novel
immunotherapeutic strategy for children diagnosed with
subgroups Sonic Hedgehog and Group 3 MB.},
keywords = {CD155/TIGIT (Other) / PD-L1/PD-1 (Other) / immune
checkpoints (Other) / medulloblastoma (Other) / organoids
(Other)},
cin = {B062 / HD01},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40703802},
pmc = {pmc:PMC12284645},
doi = {10.1093/noajnl/vdaf099},
url = {https://inrepo02.dkfz.de/record/303120},
}