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@ARTICLE{Jungwirth:310296,
author = {G. Jungwirth$^*$ and J. Cao and Y. Pan and R. Warta and C.
Lotsch and M. Moustafa$^*$ and M. Knoll$^*$ and T. Yu and V.
Braun and L. Jassowicz and P. D. Trong and Z. Wang and A.
Younsi and M. Scherer and M. Bendszus and S. M. Krieg and A.
von Deimling$^*$ and J. Debus$^*$ and A. Abdollahi$^*$ and
A. Unterberg and F. Sahm$^*$ and C. Herold-Mende},
title = {{D}rug screening on tumor organoids exposes therapeutic
vulnerabilities of meningiomas to {HDAC}1/2i panobinostat.},
journal = {Science translational medicine},
volume = {18},
number = {839},
issn = {1946-6234},
address = {Washington, DC},
publisher = {AAAS},
reportid = {DKFZ-2026-00519},
pages = {eaea3115},
year = {2026},
abstract = {Managing aggressive meningiomas remains challenging because
of limited treatment options besides surgical tumor removal
and radiotherapy. To increase the repertoire of promising
therapies for aggressive meningiomas, we established a
multistep drug screening workflow, focusing on targetable
genes obtained from transcriptome data of highly aggressive
grade 3 meningiomas. In vitro screening of 107 targeted
drugs identified nine effective inhibitors. To study these
drugs in a more natural environment, we established a
standardized patient-derived tumor organoid (TO) model
preserving accurately the original tissue's genotype and
phenotype. Individual drug responses were assessed in TOs
from 60 meningioma cases characterized at the molecular
level. In particular, the US Food and Drug
Administration-approved epigenetic drug panobinostat
demonstrated high antimeningioma efficacy in $70\%$ of TOs,
mediated through histone deacetylase 1 and 2 (HDAC1/2)
inhibition. In addition, treatment in an orthotopic in vivo
model revealed improved survival. In search of the molecular
mechanism underlying a potentially intrinsic panobinostat
resistance, we identified up-regulation of the
HDAC8-transforming growth factor-β
(TGFβ)-epithelial-to-mesenchymal transition (EMT) axis in
the TO model, whereas subsequent HDAC8 depletion increased
the sensitivity to panobinostat. These data highlight the
utility of personalized drug screenings on TOs to identify
suitable drug targets and inhibitors for more effective
treatment of clinically aggressive meningiomas and to help
advance our understanding of counteracting resistance
mechanisms.},
cin = {HD01 / E210 / B300 / E050},
ddc = {500},
cid = {I:(DE-He78)HD01-20160331 / I:(DE-He78)E210-20160331 /
I:(DE-He78)B300-20160331 / I:(DE-He78)E050-20160331},
pnm = {319H - Addenda (POF4-319H)},
pid = {G:(DE-HGF)POF4-319H},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41779868},
doi = {10.1126/scitranslmed.aea3115},
url = {https://inrepo02.dkfz.de/record/310296},
}