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@ARTICLE{MontielEquihua:309872,
author = {C. Montiel Equihua and J. J. Molenaar and I. Areso and J.
A. Biegel and P. Blanc and S. N. Chi and S. Daems and L.
Danielson and J. Drost and N. E. Franke and M. C. Frühwald
and A. Gajjar and J. I. Geller and A. Huang and P. D. Johann
and P. Kearns and K. Nysom and S. O'Connor and M. V. Ortiz
and J. Parker and S. Patel and S. Patel and C. W. Roberts
and D. Williamson and J. S. Yi and A. D. Pearson and D.
Jenkinson and M. Kool$^*$ and F. Bourdeaut},
collaboration = {LifeArc and I. T. f. C. w. Cancer and C. R. UK and C. G. C.
P. team},
title = {{P}aediatric {T}herapeutic {D}evelopment {W}orkshop on
rhabdoid tumours.},
journal = {British journal of cancer},
volume = {nn},
issn = {0007-0920},
address = {Edinburgh},
publisher = {Nature Publ. Group},
reportid = {DKFZ-2026-00357},
pages = {nn},
year = {2026},
note = {#DKTKZFB26# / epub},
abstract = {Rhabdoid tumours (RT) are malignancies of the central
nervous system, kidneys, liver and soft tissues that most
commonly affect very young children with survival rates
below $30\%$ in high-risk cohorts. Treatment entails
surgery, intensive chemotherapy and radiotherapy, associated
with substantial short- and long-term toxicities. There is
an unmet need to develop targeted therapies for RT to
improve patient outcomes and mitigate the toxicities of
current therapy. Detailed research followed by a workshop
had the objective of enabling the development of targeted
therapeutics for RT. Given the inherent commonality of their
biology (i.e. biallelic inactivation of SMARCB1 or more
rarely SMARCA4) the therapeutic approach should be similar
for intra-cranial and extra-cranial tumours.
DDB1-CUL4-associated factor 5 is a promising target, and the
development of small molecule binders/degraders is a
priority. Enhancer of zeste 2 polycomb repressive complex 2
subunit (EZH2) degraders may have greater therapeutic
potential than inhibitors. Fibroblast growth factor receptor
and platelet-derived growth factor receptor inhibitors may
have value in subgroups. Mouse double minute 2 homologue
(MDM2) is a priority target for novel therapeutic
development and combination trials. Combinations of EZH2,
MDM2 inhibitors and selective inhibitors of nuclear export
should be evaluated robustly preclinically and drive early
clinical studies.},
subtyp = {Review Article},
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:41680284},
doi = {10.1038/s41416-026-03348-7},
url = {https://inrepo02.dkfz.de/record/309872},
}