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@ARTICLE{Goodwin:186664,
author = {C. M. Goodwin and A. M. Waters and J. E. Klomp and S.
Javaid and K. L. Bryant and C. A. Stalnecker and K.
Drizyte-Miller and B. Papke$^*$ and R. Yang and A. M. Amparo
and I. Ozkan-Dagliyan and E. Baldelli and V. Calvert and M.
Pierobon and J. A. Sorrentino and A. P. Beelen and N.
Bublitz$^*$ and M. Lüthen$^*$ and K. C. Wood and E. F.
Petricoin and C. Sers$^*$ and A. J. McRee and A. D. Cox and
C. J. Der},
title = {{C}ombination {T}herapies with {CDK}4/6 {I}nhibitors to
{T}reat {KRAS}-{M}utant {P}ancreatic {C}ancer.},
journal = {Cancer research},
volume = {83},
number = {1},
issn = {0099-7013},
address = {Philadelphia, Pa.},
publisher = {AACR},
reportid = {DKFZ-2023-00033},
pages = {141 - 157},
year = {2023},
abstract = {Mutational loss of CDKN2A (encoding p16INK4A)
tumor-suppressor function is a key genetic step that
complements activation of KRAS in promoting the development
and malignant growth of pancreatic ductal adenocarcinoma
(PDAC). However, pharmacologic restoration of p16INK4A
function with inhibitors of CDK4 and CDK6 (CDK4/6) has shown
limited clinical efficacy in PDAC. Here, we found that
concurrent treatment with both a CDK4/6 inhibitor (CDK4/6i)
and an ERK-MAPK inhibitor (ERKi) synergistically suppresses
the growth of PDAC cell lines and organoids by cooperatively
blocking CDK4/6i-induced compensatory upregulation of ERK,
PI3K, antiapoptotic signaling, and MYC expression. On the
basis of these findings, a Phase I clinical trial was
initiated to evaluate the ERKi ulixertinib in combination
with the CDK4/6i palbociclib in patients with advanced PDAC
(NCT03454035). As inhibition of other proteins might also
counter CDK4/6i-mediated signaling changes to increase
cellular CDK4/6i sensitivity, a CRISPR-Cas9 loss-of-function
screen was conducted that revealed a spectrum of
functionally diverse genes whose loss enhanced CDK4/6i
growth inhibitory activity. These genes were enriched around
diverse signaling nodes, including cell-cycle regulatory
proteins centered on CDK2 activation, PI3K-AKT-mTOR
signaling, SRC family kinases, HDAC proteins,
autophagy-activating pathways, chromosome regulation and
maintenance, and DNA damage and repair pathways. Novel
therapeutic combinations were validated using siRNA and
small-molecule inhibitor-based approaches. In addition,
genes whose loss imparts a survival advantage were
identified (e.g., RB1, PTEN, FBXW7), suggesting possible
resistance mechanisms to CDK4/6 inhibition. In summary, this
study has identified novel combinations with CDK4/6i that
may have clinical benefit to patients with PDAC.CRISPR-Cas9
screening and protein activity mapping reveal combinations
that increase potency of CDK4/6 inhibitors and overcome
drug-induced compensations in pancreatic cancer.},
keywords = {Humans / Carcinoma, Pancreatic Ductal: drug therapy /
Carcinoma, Pancreatic Ductal: genetics / Cell Line, Tumor /
Cyclin-Dependent Kinase 4 / Cyclin-Dependent Kinase 6 /
Pancreatic Neoplasms: drug therapy / Pancreatic Neoplasms:
genetics / Pancreatic Neoplasms: metabolism /
Phosphatidylinositol 3-Kinases: metabolism / Protein Kinase
Inhibitors: pharmacology / Protein Kinase Inhibitors:
therapeutic use / Proto-Oncogene Proteins p21(ras): genetics
/ Proto-Oncogene Proteins p21(ras): metabolism / CDK4
protein, human (NLM Chemicals) / Cyclin-Dependent Kinase 4
(NLM Chemicals) / Cyclin-Dependent Kinase 6 (NLM Chemicals)
/ KRAS protein, human (NLM Chemicals) / Phosphatidylinositol
3-Kinases (NLM Chemicals) / Protein Kinase Inhibitors (NLM
Chemicals) / Proto-Oncogene Proteins p21(ras) (NLM
Chemicals)},
cin = {BE01},
ddc = {610},
cid = {I:(DE-He78)BE01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:36346366},
pmc = {pmc:PMC9812941},
doi = {10.1158/0008-5472.CAN-22-0391},
url = {https://inrepo02.dkfz.de/record/186664},
}