Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112

PT-112 is a novel small molecule exhibiting promising clinical activity in patients with solid tumors. PT-112 kills malignant cells by inhibiting ribosome biogenesis while promoting the emission of immunostimulatory signals. Accordingly, PT-112 is an authentic immunogenic cell death (ICD) inducer an...

Full description

Saved in:
Bibliographic Details
Main Authors: Ruth Soler-Agesta, Manuel Beltrán-Visiedo, Ai Sato, Takahiro Yamazaki, Emma Guilbaud, Christina Y. Yim, Maria T. Congenie, Tyler D. Ames, Alberto Anel, Lorenzo Galluzzi
Format: Article
Language:English
Published: Taylor & Francis Group 2025-12-01
Series:OncoImmunology
Subjects:
Online Access:https://www.tandfonline.com/doi/10.1080/2162402X.2025.2507245
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849309673316941824
author Ruth Soler-Agesta
Manuel Beltrán-Visiedo
Ai Sato
Takahiro Yamazaki
Emma Guilbaud
Christina Y. Yim
Maria T. Congenie
Tyler D. Ames
Alberto Anel
Lorenzo Galluzzi
author_facet Ruth Soler-Agesta
Manuel Beltrán-Visiedo
Ai Sato
Takahiro Yamazaki
Emma Guilbaud
Christina Y. Yim
Maria T. Congenie
Tyler D. Ames
Alberto Anel
Lorenzo Galluzzi
author_sort Ruth Soler-Agesta
collection DOAJ
description PT-112 is a novel small molecule exhibiting promising clinical activity in patients with solid tumors. PT-112 kills malignant cells by inhibiting ribosome biogenesis while promoting the emission of immunostimulatory signals. Accordingly, PT-112 is an authentic immunogenic cell death (ICD) inducer and synergizes with immune checkpoint inhibitors in preclinical models of mammary and colorectal carcinoma. Moreover, PT-112 monotherapy has led to durable clinical responses, some of which persisting after treatment discontinuation. Mitochondrial outer membrane permeabilization (MOMP) regulates the cytotoxicity and immunogenicity of various anticancer agents. Here, we harnessed mouse mammary carcinoma TS/A cells to test whether genetic alterations affecting MOMP influence PT-112 activity. As previously demonstrated, PT-112 elicited robust antiproliferative and cytotoxic effects against TS/A cells, which were preceded by the ICD-associated exposure of calreticulin (CALR) on the cell surface, and accompanied by the release of HMGB1 in the culture supernatant. TS/A cells responding to PT-112 also exhibited eIF2α phosphorylation and cytosolic mtDNA accumulation, secreted type I IFN, and exposed MHC Class I molecules as well as the co-inhibitory ligand PD-L1 on their surface. Acute cytotoxicity and HMGB1 release caused by PT-112 in TS/A cells were influenced by MOMP competence. Conversely, PT-112 retained antiproliferative effects and its capacity to drive type I IFN secretion as well as CALR, MHC Class I and PD-L1 exposure on the cell surface irrespective of MOMP defects. These data indicate a partial involvement of MOMP in the mechanisms of action of PT-112, suggesting that PT-112 is active across various tumor types, including malignancies with MOMP defects.
format Article
id doaj-art-6d1d8cf1bd6f4e868803f26cc499a080
institution Kabale University
issn 2162-402X
language English
publishDate 2025-12-01
publisher Taylor & Francis Group
record_format Article
series OncoImmunology
spelling doaj-art-6d1d8cf1bd6f4e868803f26cc499a0802025-08-20T03:54:01ZengTaylor & Francis GroupOncoImmunology2162-402X2025-12-0114110.1080/2162402X.2025.2507245Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112Ruth Soler-Agesta0Manuel Beltrán-Visiedo1Ai Sato2Takahiro Yamazaki3Emma Guilbaud4Christina Y. Yim5Maria T. Congenie6Tyler D. Ames7Alberto Anel8Lorenzo Galluzzi9Department of Radiation Oncology, Weill Cornell Medical College, New York, NY, USADepartment of Radiation Oncology, Weill Cornell Medical College, New York, NY, USADepartment of Radiation Oncology, Weill Cornell Medical College, New York, NY, USADepartment of Radiation Oncology, Weill Cornell Medical College, New York, NY, USADepartment of Radiation Oncology, Weill Cornell Medical College, New York, NY, USAPromontory Therapeutics, New York, NY, USAPromontory Therapeutics, New York, NY, USAPromontory Therapeutics, New York, NY, USABiochemistry and Molecular and Cell Biology, Aragón Health Research Institute (IIS-Aragón), University of Zaragoza, Zaragoza, SpainDepartment of Radiation Oncology, Weill Cornell Medical College, New York, NY, USAPT-112 is a novel small molecule exhibiting promising clinical activity in patients with solid tumors. PT-112 kills malignant cells by inhibiting ribosome biogenesis while promoting the emission of immunostimulatory signals. Accordingly, PT-112 is an authentic immunogenic cell death (ICD) inducer and synergizes with immune checkpoint inhibitors in preclinical models of mammary and colorectal carcinoma. Moreover, PT-112 monotherapy has led to durable clinical responses, some of which persisting after treatment discontinuation. Mitochondrial outer membrane permeabilization (MOMP) regulates the cytotoxicity and immunogenicity of various anticancer agents. Here, we harnessed mouse mammary carcinoma TS/A cells to test whether genetic alterations affecting MOMP influence PT-112 activity. As previously demonstrated, PT-112 elicited robust antiproliferative and cytotoxic effects against TS/A cells, which were preceded by the ICD-associated exposure of calreticulin (CALR) on the cell surface, and accompanied by the release of HMGB1 in the culture supernatant. TS/A cells responding to PT-112 also exhibited eIF2α phosphorylation and cytosolic mtDNA accumulation, secreted type I IFN, and exposed MHC Class I molecules as well as the co-inhibitory ligand PD-L1 on their surface. Acute cytotoxicity and HMGB1 release caused by PT-112 in TS/A cells were influenced by MOMP competence. Conversely, PT-112 retained antiproliferative effects and its capacity to drive type I IFN secretion as well as CALR, MHC Class I and PD-L1 exposure on the cell surface irrespective of MOMP defects. These data indicate a partial involvement of MOMP in the mechanisms of action of PT-112, suggesting that PT-112 is active across various tumor types, including malignancies with MOMP defects.https://www.tandfonline.com/doi/10.1080/2162402X.2025.2507245CGASCD8+ cytotoxic T lymphocytesdamage-associated molecular patternsimmune checkpoint inhibitorsmtDNAPD-1
spellingShingle Ruth Soler-Agesta
Manuel Beltrán-Visiedo
Ai Sato
Takahiro Yamazaki
Emma Guilbaud
Christina Y. Yim
Maria T. Congenie
Tyler D. Ames
Alberto Anel
Lorenzo Galluzzi
Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
OncoImmunology
CGAS
CD8+ cytotoxic T lymphocytes
damage-associated molecular patterns
immune checkpoint inhibitors
mtDNA
PD-1
title Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
title_full Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
title_fullStr Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
title_full_unstemmed Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
title_short Partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of PT-112
title_sort partial mitochondrial involvement in the antiproliferative and immunostimulatory effects of pt 112
topic CGAS
CD8+ cytotoxic T lymphocytes
damage-associated molecular patterns
immune checkpoint inhibitors
mtDNA
PD-1
url https://www.tandfonline.com/doi/10.1080/2162402X.2025.2507245
work_keys_str_mv AT ruthsoleragesta partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT manuelbeltranvisiedo partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT aisato partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT takahiroyamazaki partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT emmaguilbaud partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT christinayyim partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT mariatcongenie partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT tylerdames partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT albertoanel partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112
AT lorenzogalluzzi partialmitochondrialinvolvementintheantiproliferativeandimmunostimulatoryeffectsofpt112