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@ARTICLE{Keller:178319,
author = {K. M. Keller and S. Krausert$^*$ and A. Gopisetty$^*$ and
D. Luedtke and J. Koster and N. A. Schubert and A.
Rodríguez and S. R. van Hooff and D. Stichel$^*$ and M. E.
M. Dolman and G. Vassal and S. M. Pfister$^*$ and H. N.
Caron and L. F. Stancato and J. J. Molenaar and N.
Jäger$^*$ and M. Kool$^*$},
title = {{T}arget {A}ctionability {R}eview: a systematic evaluation
of replication stress as a therapeutic target for paediatric
solid malignancies.},
journal = {European journal of cancer},
volume = {162},
issn = {0014-2964},
address = {Amsterdam [u.a.]},
publisher = {Elsevier},
reportid = {DKFZ-2021-03266},
pages = {107 - 117},
year = {2022},
note = {#EA:B062#LA:B062#},
abstract = {Owing to the high numbers of paediatric cancer-related
deaths, advances in therapeutic options for childhood cancer
is a heavily studied field, especially over the past decade.
Classical chemotherapy offers some therapeutic benefit but
has proven long-term complications in survivors, and there
is an urgent need to identify novel target-driven therapies.
Replication stress is a major cause of genomic instability
in cancer, triggering the stalling of the replication fork.
Failure of molecular response by DNA damage checkpoints, DNA
repair mechanisms and restarting the replication forks can
exacerbate replication stress and initiate cell death
pathways, thus presenting as a novel therapeutic target. To
bridge the gap between preclinical evidence and clinical
utility thereof, we apply the literature-driven systematic
target actionability review methodology to published
proof-of-concept (PoC) data related to the process of
replication stress.A meticulous PubMed literature search was
performed to gather replication stress-related articles
(published between 2014 and 2021) across 16 different
paediatric solid tumour types. Articles that fulfilled
inclusion criteria were uploaded into the R2 informatics
platform [r2.amc.nl] and assessed by critical appraisal. Key
evidence based on nine pre-established PoC modules was
summarised, and scores based on the quality and outcome of
each study were assigned by two separate reviewers. Articles
with discordant modules/scores were re-scored by a third
independent reviewer, and a final consensus score was agreed
upon by adjudication between all three reviewers. To
visualise the final scores, an interactive heatmap
summarising the evidence and scores associated with each PoC
module across all, including paediatric tumour types, were
generated.145 publications related to targeting replication
stress in paediatric tumours were systematically reviewed
with an emphasis on DNA repair pathways and cell cycle
checkpoint control. Although various targets in these
pathways have been studied in these diseases to different
extents, the results of this extensive literature search
show that ATR, CHK1, PARP or WEE1 are the most promising
targets using either single agents or in combination with
chemotherapy or radiotherapy in neuroblastoma, osteosarcoma,
high-grade glioma or medulloblastoma. Targeting these
pathways in other paediatric malignancies may work as well,
but here, the evidence was more limited. The evidence for
other targets (such as ATM and DNA-PK) was also limited but
showed promising results in some malignancies and requires
more studies in other tumour types. Overall, we have created
an extensive overview of targeting replication stress across
16 paediatric tumour types, which can be explored using the
interactive heatmap on the R2 target actionability review
platform
$[https://hgserver1.amc.nl/cgi-bin/r2/main.cgi?option=imi2_targetmap_v1].$},
subtyp = {Review Article},
keywords = {Cell cycle checkpoints (Other) / DNA repair (Other) /
Paediatric oncology (Other) / Preclinical research (Other) /
Replication stress (Other) / Systematic review (Other) /
Targeted drugs (Other)},
cin = {B062 / HD01 / B300},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)B300-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34963094},
doi = {10.1016/j.ejca.2021.11.030},
url = {https://inrepo02.dkfz.de/record/178319},
}