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@ARTICLE{Kse:305545,
author = {H. Köse and C. Schneeweis and P. Putze and C. T. Contreras
and L. Ferreiro and L. Witte and I. Deidda and F. Herzberg
and S. Ebert and J. Jakubik$^*$ and L. Moldaner$^*$ and J.
Todorovic and I. Träger and C. Zang and U. M. Demel and E.
Hessmann and M. Kirchner and S. Rhein and J. Hoffmann and Z.
Tatarova$^*$ and M. Ghadimi and D. Saur$^*$ and K. Kappert
and P. Mertins and G. Schneider and U. Keller and M.
Wirth$^*$},
title = {{T}argeting {M}utual {D}ependence of
{P}hosphatidylinositol-3-{K}inase α/δ and {S}mall
{U}biquitin-{L}ike {M}odifier {S}ignaling in {P}ancreatic
{C}ancer.},
journal = {Gastroenterology},
volume = {nn},
issn = {0016-5085},
address = {New York, NY},
publisher = {Elsevier},
reportid = {DKFZ-2025-02219},
pages = {nn},
year = {2025},
note = {epub},
abstract = {Pancreatic ductal adenocarcinoma (PDAC) is a highly
aggressive and lethal cancer, with a 5-year survival rate of
$<13\%.$ Despite advances in diagnostics and treatments, the
standard of care for PDAC remains inadequate, and most
patients develop resistance to therapy. Targeted approaches,
such as Kirsten rat sarcoma (KRAS) inhibition, have shown
promise in preclinical models, although clinical application
remains challenged by the rapid development of resistance.
The phosphatidylinositol-3-kinase (PI3K) signaling pathway
is critical for PDAC development and maintenance, yet
pharmacologic targeting has failed to yield significant
clinical benefits.To investigate the relationship between
the PI3K and small ubiquitin-like modifier (SUMO) pathways
in PDAC, we used a comprehensive approach that included
unbiased genome-wide clustered regularly interspaced short
palindromic repeats/clustered regularly interspaced short
palindromic repeats-associated protein 9 resistance screens,
pharmacologic screens, transcriptomics, proteomics, and
phosphoproteomics experiments. Genetic knockout models were
applied to validate our findings. A novel molecularly
targeted combination therapy was tested in preclinical mouse
models.Using genetic and pharmacologic screenings, we
discovered a mutual and targetable codependence between the
PI3K and the SUMO pathways. Simultaneous inhibition of
PIK3α and PIK3δ, combined with SUMO-activating E1
targeting, triggered synthetic lethality and cell death. In
syngeneic orthotopic immune-competent PDAC models, this
combination therapy reduced tumor growth and promoted immune
cell infiltration and activity.Our study introduces a novel
rational combination therapy in PDAC. Dual targeting of
PI3Kα/δ and SUMO signaling bears potential for clinical
translation.},
keywords = {Combination Therapy (Other) / PI3K (Other) / Pancreatic
Cancer (Other) / Regulated Cell Death (Other) / SUMOylation
(Other)},
cin = {FM01 / MU01 / BE01},
ddc = {610},
cid = {I:(DE-He78)FM01-20160331 / I:(DE-He78)MU01-20160331 /
I:(DE-He78)BE01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41143762},
doi = {10.1053/j.gastro.2025.08.018},
url = {https://inrepo02.dkfz.de/record/305545},
}