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@ARTICLE{Raffel:131068,
author = {S. Raffel$^*$ and M. Falcone$^*$ and N. Kneisel$^*$ and J.
Hansson and W. Wang$^*$ and C. Lutz and L. Bullinger and G.
Poschet and Y. Nonnenmacher and A. Barnert$^*$ and C.
Bahr$^*$ and P. Zeisberger$^*$ and A. Przybylla$^*$ and M.
Sohn$^*$ and M. Tönjes and A. Erez and L. Adler and P.
Jensen$^*$ and C. Scholl$^*$ and S. Fröhling$^*$ and S.
Cocciardi and P. Wuchter and C. Thiede and A. Flörcken and
J. Westermann and G. Ehninger and P. Lichter$^*$ and K.
Hiller and R. Hell and C. Herrmann$^*$ and A. D. Ho and J.
Krijgsveld$^*$ and B. Radlwimmer$^*$ and A. Trumpp$^*$},
title = {{BCAT}1 restricts α{KG} levels in {AML} stem cells leading
to {IDH}mut-like {DNA} hypermethylation.},
journal = {Nature},
volume = {551},
number = {7680},
issn = {1476-4687},
address = {London [u.a.]},
publisher = {Nature Publ. Group},
reportid = {DKFZ-2017-06135},
pages = {384 - 388},
year = {2017},
abstract = {The branched-chain amino acid (BCAA) pathway and high
levels of BCAA transaminase 1 (BCAT1) have recently been
associated with aggressiveness in several cancer entities.
However, the mechanistic role of BCAT1 in this process
remains largely uncertain. Here, by performing
high-resolution proteomic analysis of human acute myeloid
leukaemia (AML) stem-cell and non-stem-cell populations, we
find the BCAA pathway enriched and BCAT1 protein and
transcripts overexpressed in leukaemia stem cells. We show
that BCAT1, which transfers α-amino groups from BCAAs to
α-ketoglutarate (αKG), is a critical regulator of
intracellular αKG homeostasis. Further to its role in the
tricarboxylic acid cycle, αKG is an essential cofactor for
αKG-dependent dioxygenases such as Egl-9 family hypoxia
inducible factor 1 (EGLN1) and the ten-eleven translocation
(TET) family of DNA demethylases. Knockdown of BCAT1 in
leukaemia cells caused accumulation of αKG, leading to
EGLN1-mediated HIF1α protein degradation. This resulted in
a growth and survival defect and abrogated
leukaemia-initiating potential. By contrast, overexpression
of BCAT1 in leukaemia cells decreased intracellular αKG
levels and caused DNA hypermethylation through altered TET
activity. AML with high levels of BCAT1 (BCAT1high)
displayed a DNA hypermethylation phenotype similar to cases
carrying a mutant isocitrate dehydrogenase (IDHmut), in
which TET2 is inhibited by the oncometabolite
2-hydroxyglutarate. High levels of BCAT1 strongly correlate
with shorter overall survival in IDHWTTET2WT, but not IDHmut
or TET2mut AML. Gene sets characteristic for IDHmut AML were
enriched in samples from patients with an
IDHWTTET2WTBCAT1high status. BCAT1high AML showed robust
enrichment for leukaemia stem-cell signatures, and paired
sample analysis showed a significant increase in BCAT1
levels upon disease relapse. In summary, by limiting
intracellular αKG, BCAT1 links BCAA catabolism to HIF1α
stability and regulation of the epigenomic landscape,
mimicking the effects of IDH mutations. Our results suggest
the BCAA-BCAT1-αKG pathway as a therapeutic target to
compromise leukaemia stem-cell function in patients with
IDHWTTET2WT AML.},
cin = {G102 / A010 / B060 / B230 / B080 / G100 / V960 / A060 /
L101 / L301},
ddc = {070},
cid = {I:(DE-He78)G102-20160331 / I:(DE-He78)A010-20160331 /
I:(DE-He78)B060-20160331 / I:(DE-He78)B230-20160331 /
I:(DE-He78)B080-20160331 / I:(DE-He78)G100-20160331 /
I:(DE-He78)V960-20160331 / I:(DE-He78)A060-20160331 /
I:(DE-He78)L101-20160331 / I:(DE-He78)L301-20160331},
pnm = {311 - Signalling pathways, cell and tumor biology
(POF3-311)},
pid = {G:(DE-HGF)POF3-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:29144447},
doi = {10.1038/nature24294},
url = {https://inrepo02.dkfz.de/record/131068},
}