Elucidating molecularly stratified single agent, and combination, therapeutic strategies targeting MCL1 for lethal prostate cancer

J Juan M. Jiménez-Vacas D Daniel Westaby I Ines Figueiredo A Alexis De Haven Brandon A Ana Padilha W Wei Yuan G George Seed D Denisa Bogdan B Bora Gurel C Claudia Bertan S Susana Miranda M Maryou Lambros A Antonio J. Montero-Hidalgo I Ilsa Coleman I Ivan Pak Lok Yu (Vancouver Prostate Centre, Mohseni Institute of Urologic Science, University of British Columbia, 2660 Oak St, Vancouver, BC V6H 3Z6, Canada) L Lorenzo Buroni W Wanting Zeng A Antje J. Neeb J Jon Welti J Jan Rekowski R Roberta Paravati F Florian Gabel N Nicole Pandell A Ana Ferreira M Mateus Crespo R Ruth Riisnaes S Souvik Das (State Key Laboratory of Critical Earth Material Cycling and Mineral Deposits, Nanjing University) J Joe Taylor N Nick Waldron E Emily Hobern M Melanie Valenti J Jian Ning I Ilona Bernett K Kate Liodaki T Thomas Persse P Patricia Galipeau S Scott Wilkinson S Shana Y. Trostel F Fatima Karzai C Cindy H. Chau E Erica L. Beatson X Xiaohu Zhang C Carleen Klumpp-Thomas (National Center for Advancing Translational Sciences) A Andreas Varkaris R Raul M. Luque A Amanda Swain F Florence Raynaud N Nathan A. Lack (Vancouver Prostate Centre, Mohseni Institute of Urologic Science, University of British Columbia, 2660 Oak St, Vancouver, BC V6H 3Z6, Canada) C Craig J. Thomas G Gavin Ha W William D. Figg M Marco Bezzi A Adam G. Sowalsky P Peter S. Nelson (Division of Hematology and Oncology, University of Washington, Fred Hutchinson Cancer Center) S Suzanne Carreira S Steven P. Balk (Department of Medicine, Beth Israel Deaconess Medical Center) J Johann S. de Bono A Adam Sharp

Abstract

Abstract Metastatic castration-resistant prostate cancer (mCRPC) is a lethal disease requiring additional therapeutic strategies. MCL1, an anti-apoptotic BCL2 family member, promotes cancer-cell survival, but its role in mCRPC remains poorly understood. Here, we characterise MCL1 in multiple mCRPC biopsy cohorts and patient-derived models, assessing responses to MCL1 inhibition. MCL1 copy number gain (14%–34%) correlates with increased MCL1 expression and worse outcomes. MCL1 inhibition exhibits anti-tumour effects in MCL1-gained mCRPC models. Co-inhibition of MCL1 and AKT induces cancer-specific cell death in PTEN-loss/PI3K-activated models in vitro and in vivo, modulating BAD-BCLXL and BIM-MCL1 interactions, with durable anti-tumour activity in models with AKT inhibitor acquired resistance. Finally, CDK9-mediated MCL1 downregulation combined with AKT inhibition recapitulates these findings, providing further opportunities for clinical translation. These data support early phase clinical trials targeting MCL1, both as monotherapy for MCL1-gained mCRPC, and in combination with AKT inhibition for PTEN-loss/PI3K-activated mCRPC.

Article Details

Volume / Issue Vol. 16, Issue 1
Published October 08, 2025
ISSN 2041-1723
Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (58)

J

Juan M. Jiménez-Vacas

D

Daniel Westaby

I

Ines Figueiredo

A

Alexis De Haven Brandon

A

Ana Padilha

W

Wei Yuan

G

George Seed

D

Denisa Bogdan

B

Bora Gurel

C

Claudia Bertan

S

Susana Miranda

M

Maryou Lambros

A

Antonio J. Montero-Hidalgo

I

Ilsa Coleman

I

Ivan Pak Lok Yu

Vancouver Prostate Centre, Mohseni Institute of Urologic Science, University of British Columbia, 2660 Oak St, Vancouver, BC V6H 3Z6, Canada

L

Lorenzo Buroni

W

Wanting Zeng

A

Antje J. Neeb

J

Jon Welti

J

Jan Rekowski

R

Roberta Paravati

F

Florian Gabel

N

Nicole Pandell

A

Ana Ferreira

M

Mateus Crespo

R

Ruth Riisnaes

S

Souvik Das

State Key Laboratory of Critical Earth Material Cycling and Mineral Deposits, Nanjing University

J

Joe Taylor

N

Nick Waldron

E

Emily Hobern

M

Melanie Valenti

J

Jian Ning

I

Ilona Bernett

K

Kate Liodaki

T

Thomas Persse

P

Patricia Galipeau

S

Scott Wilkinson

S

Shana Y. Trostel

F

Fatima Karzai

C

Cindy H. Chau

E

Erica L. Beatson

X

Xiaohu Zhang

C

Carleen Klumpp-Thomas

National Center for Advancing Translational Sciences

A

Andreas Varkaris

R

Raul M. Luque

A

Amanda Swain

F

Florence Raynaud

N

Nathan A. Lack

Vancouver Prostate Centre, Mohseni Institute of Urologic Science, University of British Columbia, 2660 Oak St, Vancouver, BC V6H 3Z6, Canada

C

Craig J. Thomas

G

Gavin Ha

W

William D. Figg

M

Marco Bezzi

A

Adam G. Sowalsky

P

Peter S. Nelson

Division of Hematology and Oncology, University of Washington, Fred Hutchinson Cancer Center

S

Suzanne Carreira

S

Steven P. Balk

Department of Medicine, Beth Israel Deaconess Medical Center

J

Johann S. de Bono

A

Adam Sharp