Acute resistance to BET inhibitors remodels compensatory transcriptional programs via p300 coactivation

V Viral Shah (Global Modeling and Assimilation Office, NASA Goddard Space Flight Center) G George Giotopoulos H Hikari Osaki (4Cambridge Stem Cell Institute, Cambridge, United Kingdom) M Markus Meyerhöfer (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany) E Eshwar Meduri A Aaron Gallego-Crespo (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany) M Malte A. Behrendt (1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany) M Maria Saura-Panella (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany) A Aarti Tarkar (6GlaxoSmithKline Research and Development, Collegeville, PA) B Benedict Schubert (1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany) H Haiyang Yun (4Cambridge Stem Cell Institute, Cambridge, United Kingdom) S Sarah J. Horton S Shuchi Agrawal-Singh P Patricia S. Haehnel (1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany) F Faisal Basheer (13Addenbrooke's Hospital, Department of Haematology, Cambridge, United Kingdom) D Dave Lugo (8Adaptive Immunity and Immuno-epigenetics Research Unit, GlaxoSmithKline Research and Development, Stevenage, United Kingdom) I Ioanna Eleftheriadou (6GlaxoSmithKline Research and Development, Collegeville, PA) O Olena Barbash (6GlaxoSmithKline Research and Development, Collegeville, PA) A Arindam Dhar (6GlaxoSmithKline Research and Development, Collegeville, PA) M Michael W. M. Kühn (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany) B Borhane Guezguez M Matthias Theobald T Thomas Kindler P Paolo Gallipoli P Paul Yeh (5Department of Medicine, School of Clinical Sciences at Monash Health, Monash University, Clayton, VIC, Australia) M Mark A. Dawson (12Department of Clinical Haematology, Peter MacCallum Cancer Centre, Melbourne, Australia) R Rab K. Prinjha (8Adaptive Immunity and Immuno-epigenetics Research Unit, GlaxoSmithKline Research and Development, Stevenage, United Kingdom) B Brian J. P. Huntly D Daniel Sasca (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany)

Abstract

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.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 7
Published February 13, 2025
Pages 748-764
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (29)

V

Viral Shah

Global Modeling and Assimilation Office, NASA Goddard Space Flight Center

G

George Giotopoulos

H

Hikari Osaki

4Cambridge Stem Cell Institute, Cambridge, United Kingdom

M

Markus Meyerhöfer

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

E

Eshwar Meduri

A

Aaron Gallego-Crespo

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

M

Malte A. Behrendt

1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany

M

Maria Saura-Panella

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

A

Aarti Tarkar

6GlaxoSmithKline Research and Development, Collegeville, PA

B

Benedict Schubert

1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany

H

Haiyang Yun

4Cambridge Stem Cell Institute, Cambridge, United Kingdom

S

Sarah J. Horton

S

Shuchi Agrawal-Singh

P

Patricia S. Haehnel

1Department of Hematology and Oncology, University Medical Center of the Johannes Gutenberg University, Mainz, Germany

F

Faisal Basheer

13Addenbrooke's Hospital, Department of Haematology, Cambridge, United Kingdom

D

Dave Lugo

8Adaptive Immunity and Immuno-epigenetics Research Unit, GlaxoSmithKline Research and Development, Stevenage, United Kingdom

I

Ioanna Eleftheriadou

6GlaxoSmithKline Research and Development, Collegeville, PA

O

Olena Barbash

6GlaxoSmithKline Research and Development, Collegeville, PA

A

Arindam Dhar

6GlaxoSmithKline Research and Development, Collegeville, PA

M

Michael W. M. Kühn

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

B

Borhane Guezguez

M

Matthias Theobald

T

Thomas Kindler

P

Paolo Gallipoli

P

Paul Yeh

5Department of Medicine, School of Clinical Sciences at Monash Health, Monash University, Clayton, VIC, Australia

M

Mark A. Dawson

12Department of Clinical Haematology, Peter MacCallum Cancer Centre, Melbourne, Australia

R

Rab K. Prinjha

8Adaptive Immunity and Immuno-epigenetics Research Unit, GlaxoSmithKline Research and Development, Stevenage, United Kingdom

B

Brian J. P. Huntly

D

Daniel Sasca

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany