The mitochondrial protease ClpP is a metabolic vulnerability and an immunogenic trigger against multiple myeloma

T Tommaso Perini (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) P Paola Zordan R Rossella Del Pizzo M Massimo Resnati (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) L Lisa Viviani (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) D Davide Stefanoni (2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy) L Laura Cassina (2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy) U Ugo Orfanelli (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) M Matteo Trudu (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) L Laura Oliva (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) D Daniel Lacidogna (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) M Mehmet K. Samur (Dana-Farber Cancer Institute and Harvard School of Public Health, Boston, Massachusetts, United States) M Maria Materozzi (1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy) D Denise Drago (7Proteomics and Metabolomics Facility, Center for Omics Sciences, IRCCS Ospedale San Raffaele, Milan, Italy) A Annapaola Andolfo (7Proteomics and Metabolomics Facility, Center for Omics Sciences, IRCCS Ospedale San Raffaele, Milan, Italy) M Marco Patrone (Biocrystallography Unit, Division of Immunology, Transplantation, and Infectious Diseases, Istituto Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute) M Massimo Degano A Alessandra Boletta (2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy) E Enrico Milan (1Istituto di Ricovero e Cura a Carattere Scientifico Ospedale San Raffaele) F Fabio Ciceri (Vita-Salute San Raffaele University, Milan) N Nikhil C. Munshi M Matteo Bellone S Simone Cenci

Abstract

Abstract Orchestrating key homeostatic functions, mitochondria likely entail cancer vulnerabilities. Moreover, because of their bacterial ancestry, they can release potent immunogenic signals. In this study, we showed that the mitochondrial protease caseinolytic peptidase P (ClpP) is both a cell-intrinsic metabolic vulnerability and an actionable immunogenic trigger in multiple myeloma (MM). We found that ClpP messenger RNA is higher in bone marrow (BM)-purified malignant plasma cells than in normal or premalignant counterparts and that MM lines rank first in ClpP expression among human cancers. Moreover, we demonstrated that human MM cells are highly vulnerable to ClpP inhibition in vitro and in vivo. Surprisingly, MM cell dependence on ClpP was not accounted for by its acknowledged oxidative phosphorylation surveillance activity. Proteomic discovery of proteolytic targets, metabolomics, and metabolic tracing identified a critical control exerted by ClpP on ornithine aminotransferase abundance to sustain cytosolic biosynthesis of polyamines, which are essential for MM cells. Transcriptomics and targeted validation also revealed the activation of a cyclic GMP-AMP synthase (cGAS)–dependent type I interferon (IFN) response in ClpP-silenced MM cells, whose supernatants boosted dendritic cell activation and ability to stimulate IFN-γ production by T cells. In vivo, ClpP silencing reshaped the BM immune environment in immunocompetent mice by significantly expanding IFN-γ–producing CD4+ and CD8+ T cells and CD4+ T memory cells, while containing exhausted CD4+ T cells and myeloid-derived suppressor cells. Thus, ClpP is a newly identified addiction of MM cells whose inhibition not only exerts cell-intrinsic toxicity but also triggers otherwise indolent antitumoral immunity. Our findings yield a novel immunogenic chemotherapeutic framework with potential relevance to myeloma.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 11
Published September 11, 2025
Pages 1286-1299
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (23)

T

Tommaso Perini

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

P

Paola Zordan

R

Rossella Del Pizzo

M

Massimo Resnati

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

L

Lisa Viviani

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

D

Davide Stefanoni

2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy

L

Laura Cassina

2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy

U

Ugo Orfanelli

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

M

Matteo Trudu

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

L

Laura Oliva

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

D

Daniel Lacidogna

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

M

Mehmet K. Samur

Dana-Farber Cancer Institute and Harvard School of Public Health, Boston, Massachusetts, United States

M

Maria Materozzi

1Age Related Diseases Unit, Division of Genetics and Cell Biology, IRCCS Ospedale San Raffaele, Milan, Italy

D

Denise Drago

7Proteomics and Metabolomics Facility, Center for Omics Sciences, IRCCS Ospedale San Raffaele, Milan, Italy

A

Annapaola Andolfo

7Proteomics and Metabolomics Facility, Center for Omics Sciences, IRCCS Ospedale San Raffaele, Milan, Italy

M

Marco Patrone

Biocrystallography Unit, Division of Immunology, Transplantation, and Infectious Diseases, Istituto Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute

M

Massimo Degano

A

Alessandra Boletta

2Division of Genetics and Cell Biology, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy

E

Enrico Milan

1Istituto di Ricovero e Cura a Carattere Scientifico Ospedale San Raffaele

F

Fabio Ciceri

Vita-Salute San Raffaele University, Milan

N

Nikhil C. Munshi

M

Matteo Bellone

S

Simone Cenci