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@ARTICLE{Rudat:141140,
author = {S. Rudat$^*$ and A. Pfaus$^*$ and Y. Y. Cheng$^*$ and J.
Holtmann$^*$ and J. M. Ellegast and C. Bühler and D. D.
Marcantonio and E. Martinez and S. Göllner and C.
Wickenhauser and C. Müller-Tidow and C. Lutz and L.
Bullinger and M. Milsom$^*$ and S. M. Sykes and S.
Fröhling$^*$ and C. Scholl$^*$},
title = {{RET}-mediated autophagy suppression as targetable
co-dependence in acute myeloid leukemia.},
journal = {Leukemia},
volume = {32},
number = {10},
issn = {1476-5551},
address = {London},
publisher = {Springer Nature},
reportid = {DKFZ-2018-01671},
pages = {2189 - 2202},
year = {2018},
abstract = {Many cases of AML are associated with mutational activation
of receptor tyrosine kinases (RTKs) such as FLT3. However,
RTK inhibitors have limited clinical efficacy as single
agents, indicating that AML is driven by concomitant
activation of different signaling molecules. We used a
functional genomic approach to identify RET, encoding an
RTK, as an essential gene in multiple subtypes of AML, and
observed that AML cells show activation of RET signaling via
ARTN/GFRA3 and NRTN/GFRA2 ligand/co-receptor complexes.
Interrogation of downstream pathways identified
mTORC1-mediated suppression of autophagy and subsequent
stabilization of leukemogenic drivers such as mutant FLT3 as
important RET effectors. Accordingly, genetic or
pharmacologic RET inhibition impaired the growth of
FLT3-dependent AML cell lines and was accompanied by
upregulation of autophagy and FLT3 depletion. RET dependence
was also evident in mouse models of AML and primary AML
patient samples, and transcriptome and immunohistochemistry
analyses identified elevated RET mRNA levels and
co-expression of RET and FLT3 proteins in a substantial
proportion of AML patients. Our results indicate that
RET-mTORC1 signaling promotes AML through autophagy
suppression, suggesting that targeting RET or, more broadly,
depletion of leukemogenic drivers via autophagy induction
provides a therapeutic opportunity in a relevant subset of
AML patients.},
cin = {G100 / A012 / L101 / G102},
ddc = {610},
cid = {I:(DE-He78)G100-20160331 / I:(DE-He78)A012-20160331 /
I:(DE-He78)L101-20160331 / I:(DE-He78)G102-20160331},
pnm = {317 - Translational cancer research (POF3-317)},
pid = {G:(DE-HGF)POF3-317},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:29654265},
doi = {10.1038/s41375-018-0102-4},
url = {https://inrepo02.dkfz.de/record/141140},
}