BH3 mimetics targeting BCL-XL have efficacy in solid tumors with RB1 loss and replication stress

A Andreas Varkaris K Keshan Wang M Mannan Nouri N Nina Kozlova (Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School) D Daniel R. Schmidt (National Institute of Standards and Technology) A Anastasia Stavridi S Seiji Arai N Nicholas Ambrosio L Larysa Poluben J Juan M. Jiménez-Vacas D Daniel Westaby J Juliet Carmichael F Fang Xie (CAS Center for Excellence in Molecular Plant Sciences, Shanghai Institute of Plant Physiology and Ecology, Chinese Academy of Sciences) I Ines Figueiredo L Lorenzo Buroni A Antje Neeb B Bora Gurel N Nicholas Chevalier L Lisha Brown O Olga Voznesensky S Shao-Yong Chen J Joshua W. Russo X Xin Yuan D Dejan Juric (Mass General Cancer Center, Department of Medicine, Harvard Medical School, Boston) H Himisha Beltran J Johann S. de Bono M Matthew G. Vander Heiden D David J. Einstein T Taru Muranen (Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School) E Eva Corey (Department of Urology, University of Washington) A Adam Sharp S Steven P. Balk (Department of Medicine, Beth Israel Deaconess Medical Center)

Abstract

Abstract BH3 mimetic drugs that inhibit BCL-2, BCL-XL, or MCL-1 have limited activity in solid tumors. Through assessment of xenograft-derived 3D prostate cancer models and cell lines we find that tumors with RB1 loss are sensitive to BCL-XL inhibition. In parallel, drug screening demonstrates that disruption of nucleotide pools by agents including thymidylate synthase inhibitors sensitizes to BCL-XL inhibition, together indicating that replication stress increases dependence on BCL-XL. Mechanistically we establish that replication stress sensitizes to BCL-XL inhibition through TP53/CDKN1A-dependent suppression of BIRC5 expression. Therapy with a BCL-2/BCL-XL inhibitor (navitoclax) in combination with thymidylate synthase inhibitors (raltitrexed or capecitabine) causes marked and prolonged tumor regression in prostate and breast cancer xenograft models. These findings indicate that BCL-XL inhibitors may be effective as single agents in a subset of solid tumors with RB1 loss, and that pharmacological induction of replication stress may be a broadly applicable approach for sensitizing to BCL-XL inhibitors.

Article Details

Volume / Issue Vol. 16, Issue 1
Published May 28, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (32)

A

Andreas Varkaris

K

Keshan Wang

M

Mannan Nouri

N

Nina Kozlova

Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School

D

Daniel R. Schmidt

National Institute of Standards and Technology

A

Anastasia Stavridi

S

Seiji Arai

N

Nicholas Ambrosio

L

Larysa Poluben

J

Juan M. Jiménez-Vacas

D

Daniel Westaby

J

Juliet Carmichael

F

Fang Xie

CAS Center for Excellence in Molecular Plant Sciences, Shanghai Institute of Plant Physiology and Ecology, Chinese Academy of Sciences

I

Ines Figueiredo

L

Lorenzo Buroni

A

Antje Neeb

B

Bora Gurel

N

Nicholas Chevalier

L

Lisha Brown

O

Olga Voznesensky

S

Shao-Yong Chen

J

Joshua W. Russo

X

Xin Yuan

D

Dejan Juric

Mass General Cancer Center, Department of Medicine, Harvard Medical School, Boston

H

Himisha Beltran

J

Johann S. de Bono

M

Matthew G. Vander Heiden

D

David J. Einstein

T

Taru Muranen

Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School

E

Eva Corey

Department of Urology, University of Washington

A

Adam Sharp

S

Steven P. Balk

Department of Medicine, Beth Israel Deaconess Medical Center