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@ARTICLE{Shah:298920,
author = {V. Shah$^*$ and G. Giotopoulos and H. Osaki and M.
Meyerhöfer$^*$ and E. Meduri and A. Gallego-Crespo$^*$ and
M. A. Behrendt$^*$ and M. Saura-Pañella$^*$ and A. Tarkar
and B. Schubert$^*$ and H. Yun and S. J. Horton and S.
Agrawal-Singh and P. S. Haehnel$^*$ and F. Basheer and D.
Lugo and I. Eleftheriadou and O. Barbash and A. Dhar and M.
W. M. Kühn$^*$ and B. Guezguez$^*$ and M. Theobald$^*$ and
T. Kindler$^*$ and P. Gallipoli and P. Yeh and M. A. Dawson
and R. K. Prinjha and B. J. P. Huntly and D. Sasca$^*$},
title = {{A}cute resistance to {BET} inhibitors remodels
compensatory transcriptional programs via p300
coactivation.},
journal = {Blood},
volume = {145},
number = {7},
issn = {0006-4971},
address = {Washington, DC},
publisher = {American Society of Hematology},
reportid = {DKFZ-2025-00356},
pages = {748 - 764},
year = {2025},
abstract = {Initial clinical trials with drugs targeting epigenetic
modulators, such as bromodomain and extraterminal protein
(BET) inhibitors, demonstrate modest results in acute
myeloid leukemia (AML). A major reason for this involves an
increased transcriptional plasticity within AML, which
allows the cells to escape therapeutic pressure. In this
study, we investigated the immediate epigenetic and
transcriptional responses after BET inhibition and
demonstrated that BET inhibitor-mediated release of
bromodomain-containing protein 4 from chromatin is
accompanied by acute compensatory feedback that attenuates
downregulation or even increases the expression of specific
transcriptional modules. This adaptation is marked at key
AML maintenance genes and is mediated by p300, suggesting a
rational therapeutic opportunity to improve outcomes by
combining BET and p300 inhibition. p300 activity is required
during all steps of resistance adaptation; however, the
specific transcriptional programs that p300 regulates to
induce resistance to BET inhibition differ, in part, between
AML subtypes. As a consequence, in some AMLs, the
requirement for p300 is highest during the earlier stages of
resistance to BET inhibition, when p300 regulates
transitional transcriptional patterns that allow
leukemia-homeostatic adjustments. In other AMLs, p300 shapes
a linear resistance to BET inhibition and remains critical
throughout all stages of the evolution of resistance.
Altogether, our study elucidates the mechanisms that
underlie an 'acute' state of resistance to BET inhibition,
achieved through p300 activity, and how these mechanisms
remodel to mediate 'chronic' resistance. Importantly, our
data also suggest that sequential treatment with BET and
p300 inhibition may prevent resistance development, thereby
improving outcomes.},
keywords = {Humans / Leukemia, Myeloid, Acute: drug therapy / Leukemia,
Myeloid, Acute: genetics / Leukemia, Myeloid, Acute:
metabolism / Leukemia, Myeloid, Acute: pathology / Drug
Resistance, Neoplasm: drug effects / Drug Resistance,
Neoplasm: genetics / E1A-Associated p300 Protein: metabolism
/ E1A-Associated p300 Protein: antagonists $\&$ inhibitors /
E1A-Associated p300 Protein: genetics / Transcription
Factors: genetics / Transcription Factors: antagonists $\&$
inhibitors / Transcription Factors: metabolism / Gene
Expression Regulation, Leukemic: drug effects /
Transcription, Genetic: drug effects / Cell Line, Tumor /
Animals / Mice / Nuclear Proteins: genetics / Nuclear
Proteins: metabolism / Nuclear Proteins: antagonists $\&$
inhibitors / p300-CBP Transcription Factors: metabolism /
p300-CBP Transcription Factors: antagonists $\&$ inhibitors
/ Bromodomain Containing Proteins / Cell Cycle Proteins /
E1A-Associated p300 Protein (NLM Chemicals) / EP300 protein,
human (NLM Chemicals) / Transcription Factors (NLM
Chemicals) / BRD4 protein, human (NLM Chemicals) / Nuclear
Proteins (NLM Chemicals) / p300-CBP Transcription Factors
(NLM Chemicals) / Bromodomain Containing Proteins (NLM
Chemicals) / Cell Cycle Proteins (NLM Chemicals)},
cin = {FM01},
ddc = {610},
cid = {I:(DE-He78)FM01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39651888},
doi = {10.1182/blood.2022019306},
url = {https://inrepo02.dkfz.de/record/298920},
}