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@ARTICLE{Keller:275260,
author = {K. M. Keller and J. Koetsier and L. Schild and V. Amo-Addae
and S. Eising and K. van den Handel and K. Ober and B.
Koopmans and A. Essing and M. L. van den Boogaard and K. P.
S. Langenberg and N. Jäger$^*$ and M. Kool$^*$ and S.
Pfister$^*$ and M. E. M. Dolman and J. J. Molenaar and S. R.
van Hooff},
title = {{T}he potential of {PARP} as a therapeutic target across
pediatric solid malignancies.},
journal = {BMC cancer},
volume = {23},
number = {1},
issn = {1471-2407},
address = {Heidelberg},
publisher = {Springer},
reportid = {DKFZ-2023-00707},
pages = {310},
year = {2023},
abstract = {Pediatric cancer is the leading cause of disease-related
death in children and the need for better therapeutic
options remains urgent. Due to the limited number of
patients, target and drug development for pediatrics is
often supplemented by data from studies focused on adult
cancers. Recent evidence shows that pediatric cancers
possess different vulnerabilities that should be explored
independently from adult cancers.Using the publicly
available Genomics of Drug Sensitivity in Cancer database,
we explore therapeutic targets and biomarkers specific to
the pediatric solid malignancies Ewing sarcoma,
medulloblastoma, neuroblastoma, osteosarcoma, and
rhabdomyosarcoma. Results are validated using cell viability
assays and high-throughput drug screens are used to identify
synergistic combinations.Using published drug screening
data, PARP is identified as a drug target of interest across
multiple different pediatric malignancies. We validate these
findings, and we show that efficacy can be improved when
combined with conventional chemotherapeutics, namely
topoisomerase inhibitors. Additionally, using gene set
enrichment analysis, we identify ribosome biogenesis as a
potential biomarker for PARP inhibition in pediatric cancer
cell lines.Collectively, our results provide evidence to
support the further development of PARP inhibition and the
combination with TOP1 inhibition as a therapeutic approach
in solid pediatric malignancies. Additionally, we propose
ribosome biogenesis as a component to PARP inhibitor
sensitivity that should be further investigated to help
maximize the potential utility of PARP inhibition and
combinations across pediatric solid malignancies.},
keywords = {DNA damage (Other) / PARP (Other) / Pediatric cancer
(Other) / Replication stress (Other) / Ribosomes (Other) /
Synergy (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:37020198},
doi = {10.1186/s12885-022-10319-7},
url = {https://inrepo02.dkfz.de/record/275260},
}