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@ARTICLE{Endersby:167180,
author = {R. Endersby and J. Whitehouse and A. Pribnow and M.
Kuchibhotla and H. Hii and B. Carline and S. Gande and J.
Stripay and M. Ancliffe and M. Howlett and T. Schoep and C.
George and C. Andradas and P. Dyer and M. Schluck and B.
Patterson and S. K. Tacheva-Gigorova and M. N. Cooper and G.
Robinson and C. Stewart and S. M. Pfister$^*$ and M.
Kool$^*$ and T. Milde$^*$ and A. Gajjar and T. Johns and R.
J. Wechsler-Reya and M. F. Roussel and N. G. Gottardo},
title = {{S}mall-molecule screen reveals synergy of cell cycle
checkpoint kinase inhibitors with {DNA}-damaging
chemotherapies in medulloblastoma.},
journal = {Science translational medicine},
volume = {13},
number = {577},
issn = {1946-6242},
address = {Washington, DC},
publisher = {AAAS},
reportid = {DKFZ-2021-00171},
pages = {eaba7401 -},
year = {2021},
abstract = {Medulloblastoma (MB) consists of four core molecular
subgroups with distinct clinical features and prognoses.
Treatment consists of surgery, followed by radiotherapy and
cytotoxic chemotherapy. Despite this intensive approach,
outcome remains dismal for patients with certain subtypes of
MB, namely, MYC-amplified Group 3 and TP53-mutated SHH.
Using high-throughput assays, six human MB cell lines were
screened against a library of 3208 unique compounds. We
identified 45 effective compounds from the screen and found
that cell cycle checkpoint kinase (CHK1/2) inhibition
synergistically enhanced the cytotoxic activity of
clinically used chemotherapeutics cyclophosphamide,
cisplatin, and gemcitabine. To identify the best-in-class
inhibitor, multiple CHK1/2 inhibitors were assessed in mice
bearing intracranial MB. When combined with DNA-damaging
chemotherapeutics, CHK1/2 inhibition reduced tumor burden
and increased survival of animals with high-risk MB, across
multiple different models. In total, we tested 14 different
models, representing distinct MB subgroups, and data were
validated in three independent laboratories.
Pharmacodynamics studies confirmed central nervous system
penetration. In mice, combination treatment significantly
increased DNA damage and apoptosis compared to chemotherapy
alone, and studies with cultured cells showed that CHK
inhibition disrupted chemotherapy-induced cell cycle arrest.
Our findings indicated CHK1/2 inhibition, specifically with
LY2606368 (prexasertib), has strong chemosensitizing
activity in MB that warrants further clinical investigation.
Moreover, these data demonstrated that we developed a robust
and collaborative preclinical assessment platform that can
be used to identify potentially effective new therapies for
clinical evaluation for pediatric MB.},
cin = {B062},
ddc = {500},
cid = {I:(DE-He78)B062-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:33472956},
doi = {10.1126/scitranslmed.aba7401},
url = {https://inrepo02.dkfz.de/record/167180},
}