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@ARTICLE{Gorantla:301577,
author = {S. P. Gorantla and M. Rassner and K. A. Crossley and T. A.
Müller and T. Poggio and S. Khaja Saleem and H. Kleinfelder
and S. M. M. Gambheer and C. Endres and S. Schaberg and D.
Schmidt and G. Prince and I. Gonzalez-Menendez and D.
Bentrop and R. Trittler and S. Rylova and D. Pfeifer and G.
Andrieux$^*$ and L. Quintanilla-Martinez and A. L.
Illert$^*$ and N. von Bubnoff and R. Zeiser and J.
Duyster$^*$},
title = {{E}fficacy of {JAK}1/2 inhibition in murine
myeloproliferative neoplasms is not mediated by targeting
oncogenic signaling.},
journal = {Nature Communications},
volume = {16},
number = {1},
issn = {2041-1723},
address = {[London]},
publisher = {Springer Nature},
reportid = {DKFZ-2025-01085},
pages = {4833},
year = {2025},
abstract = {Ruxolitinib is a potent JAK1/JAK2 inhibitor, approved for
the treatment of primary myelofibrosis (PMF) patients based
on the concept of inhibition of oncogenic signaling.
However, the effect of ruxolitinib on JAK2-V617F allelic
burden is modest, suggesting that inhibition of JAK2-V617F
signaling-driven clone expansion is not the main mechanism
of action. We evaluate whether ruxolitinib mainly blocks the
proliferation of the malignant clone or exerts its effects
also by targeting non-malignant cells. Therefore, we develop
two JAK2-V617F-driven myeloproliferative neoplasm (MPN)
mouse models harboring ruxolitinib resistance mutations.
Mice carrying ruxolitinib-resistant JAK2-V617F-driven MPN
respond to ruxolitinib treatment similar to mice with
ruxolitinib-sensitive JAK2-V617F MPN with respect to
reduction of spleen size, leukocyte count and
pro-inflammatory cytokines in the serum. Ruxolitinib reduces
pro-inflammatory cytokines in both stromal cells and
non-malignant hematopoietic cells. Using a rigorous
ruxolitinib resistance mutation approach, we can prove that
ruxolitinib acts independent of oncogenic JAK2-V617F
signaling and reduces the main features of MPN disease such
as spleen size and leukocyte counts. Our findings
characterize the mechanism of action for ruxolitinib in
MPN.},
keywords = {Animals / Janus Kinase 2: antagonists $\&$ inhibitors /
Janus Kinase 2: genetics / Janus Kinase 2: metabolism /
Nitriles / Pyrazoles: pharmacology / Pyrazoles: therapeutic
use / Pyrimidines / Myeloproliferative Disorders: drug
therapy / Myeloproliferative Disorders: genetics /
Myeloproliferative Disorders: pathology / Myeloproliferative
Disorders: metabolism / Mice / Signal Transduction: drug
effects / Janus Kinase 1: antagonists $\&$ inhibitors /
Janus Kinase 1: genetics / Janus Kinase 1: metabolism /
Disease Models, Animal / Mutation / Protein Kinase
Inhibitors: pharmacology / Protein Kinase Inhibitors:
therapeutic use / Cytokines: metabolism / Cytokines: blood /
Drug Resistance, Neoplasm: genetics / Humans / Cell
Proliferation: drug effects / Mice, Inbred C57BL / Female /
Spleen: drug effects / Spleen: pathology / Janus Kinase 2
(NLM Chemicals) / ruxolitinib (NLM Chemicals) / Nitriles
(NLM Chemicals) / Pyrazoles (NLM Chemicals) / Pyrimidines
(NLM Chemicals) / Janus Kinase 1 (NLM Chemicals) / Jak2
protein, mouse (NLM Chemicals) / Protein Kinase Inhibitors
(NLM Chemicals) / Jak1 protein, mouse (NLM Chemicals) /
Cytokines (NLM Chemicals)},
cin = {FR01},
ddc = {500},
cid = {I:(DE-He78)FR01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40413183},
doi = {10.1038/s41467-025-60019-6},
url = {https://inrepo02.dkfz.de/record/301577},
}