Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy

Background Targeted thorium-227 conjugates (TTCs) are an emerging class of targeted alpha therapies (TATs). Their unique mode of action (MoA) is the induction of difficult-to-repair clustered DNA double-strand breaks. However, thus far, their effects on the immune system are largely unknown. Here, w...

Full description

Saved in:
Bibliographic Details
Main Authors: Pascale Lejeune, Véronique Cruciani, Axel Berg-Larsen, Andreas Schlicker, Anne Mobergslien, Lisa Bartnitzky, Sandra Berndt, Sabine Zitzmann-Kolbe, Claudia Kamfenkel, Stefan Stargard, Stefanie Hammer, Jennifer S Jørgensen, Malene Jackerott, Carsten H Nielsen, Christoph A Schatz, Hartwig Hennekes, Jenny Karlsson, Alan S Cuthbertson, Dominik Mumberg, Urs B Hagemann
Format: Article
Language:English
Published: BMJ Publishing Group 2021-10-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/9/10/e002387.full
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849733253119868928
author Pascale Lejeune
Véronique Cruciani
Axel Berg-Larsen
Andreas Schlicker
Anne Mobergslien
Lisa Bartnitzky
Sandra Berndt
Sabine Zitzmann-Kolbe
Claudia Kamfenkel
Stefan Stargard
Stefanie Hammer
Jennifer S Jørgensen
Malene Jackerott
Carsten H Nielsen
Christoph A Schatz
Hartwig Hennekes
Jenny Karlsson
Alan S Cuthbertson
Dominik Mumberg
Urs B Hagemann
author_facet Pascale Lejeune
Véronique Cruciani
Axel Berg-Larsen
Andreas Schlicker
Anne Mobergslien
Lisa Bartnitzky
Sandra Berndt
Sabine Zitzmann-Kolbe
Claudia Kamfenkel
Stefan Stargard
Stefanie Hammer
Jennifer S Jørgensen
Malene Jackerott
Carsten H Nielsen
Christoph A Schatz
Hartwig Hennekes
Jenny Karlsson
Alan S Cuthbertson
Dominik Mumberg
Urs B Hagemann
author_sort Pascale Lejeune
collection DOAJ
description Background Targeted thorium-227 conjugates (TTCs) are an emerging class of targeted alpha therapies (TATs). Their unique mode of action (MoA) is the induction of difficult-to-repair clustered DNA double-strand breaks. However, thus far, their effects on the immune system are largely unknown. Here, we investigated the immunostimulatory effects of the mesothelin-targeted thorium-227 conjugate (MSLN-TTC) in vitro and in vivo in monotherapy and in combination with an inhibitor of the immune checkpoint programmed death receptor ligand 1 (PD-L1) in immunocompetent mice.Methods The murine cell line MC38 was transfected with the human gene encoding for MSLN (hMSLN) to enable binding of the non-cross-reactive MSLN-TTC. The immunostimulatory effects of MSLN-TTC were studied in vitro on human cancer cell lines and MC38-hMSLN cells. The efficacy and MoA of MSLN-TTC were studied in vivo as monotherapy or in combination with anti-PD-L1 in MC38-hMSLN tumor-bearing immunocompetent C57BL/6 mice. Experiments were supported by RNA sequencing, flow cytometry, immunohistochemistry, mesoscale, and TaqMan PCR analyses to study the underlying immunostimulatory effects. In vivo depletion of CD8+ T cells and studies with Rag2/Il2Rg double knockout C57BL/6 mice were conducted to investigate the importance of immune cells to the efficacy of MSLN-TTC.Results MSLN-TTC treatment induced upregulation of DNA sensing pathway transcripts (IL-6, CCL20, CXCL10, and stimulator of interferon genes (STING)-related genes) in vitro as determined by RNASeq analysis. The results, including phospho-STING activation, were confirmed on the protein level. Danger-associated molecular pattern molecules were upregulated in parallel, leading to dendritic cell (DC) activation in vitro. MSLN-TTC showed strong antitumor activity (T:C 0.38, p<0.05) as a single agent in human MSLN-expressing MC38 tumor-bearing immunocompetent mice. Combining MSLN-TTC with anti-PD-L1 further enhanced the efficacy (T:C 0.08, p<0.001) as evidenced by the increased number of tumor-free surviving animals. MSLN-TTC monotherapy caused migration of CD103+ cDC1 DCs and infiltration of CD8+ T cells into tumors, which was enhanced on combination with anti-PD-L1. Intriguingly, CD8+ T-cell depletion decreased antitumor efficacy.Conclusions These in vitro and in vivo data on MSLN-TTC demonstrate that the MoA of TTCs involves activation of the immune system. The findings are of relevance for other targeted radiotherapies and may guide clinical combination strategies.
format Article
id doaj-art-05e7344e8ce54a129a43af0ebbd6ec6f
institution DOAJ
issn 2051-1426
language English
publishDate 2021-10-01
publisher BMJ Publishing Group
record_format Article
series Journal for ImmunoTherapy of Cancer
spelling doaj-art-05e7344e8ce54a129a43af0ebbd6ec6f2025-08-20T03:08:06ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262021-10-0191010.1136/jitc-2021-002387Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapyPascale Lejeune0Véronique Cruciani1Axel Berg-Larsen2Andreas Schlicker3Anne Mobergslien4Lisa Bartnitzky5Sandra Berndt6Sabine Zitzmann-Kolbe7Claudia Kamfenkel8Stefan Stargard9Stefanie Hammer10Jennifer S Jørgensen11Malene Jackerott12Carsten H Nielsen13Christoph A Schatz14Hartwig Hennekes15Jenny Karlsson16Alan S Cuthbertson17Dominik Mumberg18Urs B Hagemann19Bayer AG, Berlin, GermanyBayer AS, Oslo, NorwayBayer AS, Oslo, NorwayBayer AG, Berlin, GermanyBayer AS, Oslo, NorwayBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyMinerva Imaging ApS, Copenhagen, DenmarkMinerva Imaging ApS, Copenhagen, DenmarkMinerva Imaging ApS, Copenhagen, DenmarkBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBayer AS, Oslo, NorwayBayer AS, Oslo, NorwayBayer AG, Berlin, GermanyBayer AG, Berlin, GermanyBackground Targeted thorium-227 conjugates (TTCs) are an emerging class of targeted alpha therapies (TATs). Their unique mode of action (MoA) is the induction of difficult-to-repair clustered DNA double-strand breaks. However, thus far, their effects on the immune system are largely unknown. Here, we investigated the immunostimulatory effects of the mesothelin-targeted thorium-227 conjugate (MSLN-TTC) in vitro and in vivo in monotherapy and in combination with an inhibitor of the immune checkpoint programmed death receptor ligand 1 (PD-L1) in immunocompetent mice.Methods The murine cell line MC38 was transfected with the human gene encoding for MSLN (hMSLN) to enable binding of the non-cross-reactive MSLN-TTC. The immunostimulatory effects of MSLN-TTC were studied in vitro on human cancer cell lines and MC38-hMSLN cells. The efficacy and MoA of MSLN-TTC were studied in vivo as monotherapy or in combination with anti-PD-L1 in MC38-hMSLN tumor-bearing immunocompetent C57BL/6 mice. Experiments were supported by RNA sequencing, flow cytometry, immunohistochemistry, mesoscale, and TaqMan PCR analyses to study the underlying immunostimulatory effects. In vivo depletion of CD8+ T cells and studies with Rag2/Il2Rg double knockout C57BL/6 mice were conducted to investigate the importance of immune cells to the efficacy of MSLN-TTC.Results MSLN-TTC treatment induced upregulation of DNA sensing pathway transcripts (IL-6, CCL20, CXCL10, and stimulator of interferon genes (STING)-related genes) in vitro as determined by RNASeq analysis. The results, including phospho-STING activation, were confirmed on the protein level. Danger-associated molecular pattern molecules were upregulated in parallel, leading to dendritic cell (DC) activation in vitro. MSLN-TTC showed strong antitumor activity (T:C 0.38, p<0.05) as a single agent in human MSLN-expressing MC38 tumor-bearing immunocompetent mice. Combining MSLN-TTC with anti-PD-L1 further enhanced the efficacy (T:C 0.08, p<0.001) as evidenced by the increased number of tumor-free surviving animals. MSLN-TTC monotherapy caused migration of CD103+ cDC1 DCs and infiltration of CD8+ T cells into tumors, which was enhanced on combination with anti-PD-L1. Intriguingly, CD8+ T-cell depletion decreased antitumor efficacy.Conclusions These in vitro and in vivo data on MSLN-TTC demonstrate that the MoA of TTCs involves activation of the immune system. The findings are of relevance for other targeted radiotherapies and may guide clinical combination strategies.https://jitc.bmj.com/content/9/10/e002387.full
spellingShingle Pascale Lejeune
Véronique Cruciani
Axel Berg-Larsen
Andreas Schlicker
Anne Mobergslien
Lisa Bartnitzky
Sandra Berndt
Sabine Zitzmann-Kolbe
Claudia Kamfenkel
Stefan Stargard
Stefanie Hammer
Jennifer S Jørgensen
Malene Jackerott
Carsten H Nielsen
Christoph A Schatz
Hartwig Hennekes
Jenny Karlsson
Alan S Cuthbertson
Dominik Mumberg
Urs B Hagemann
Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
Journal for ImmunoTherapy of Cancer
title Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
title_full Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
title_fullStr Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
title_full_unstemmed Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
title_short Immunostimulatory effects of targeted thorium-227 conjugates as single agent and in combination with anti-PD-L1 therapy
title_sort immunostimulatory effects of targeted thorium 227 conjugates as single agent and in combination with anti pd l1 therapy
url https://jitc.bmj.com/content/9/10/e002387.full
work_keys_str_mv AT pascalelejeune immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT veroniquecruciani immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT axelberglarsen immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT andreasschlicker immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT annemobergslien immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT lisabartnitzky immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT sandraberndt immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT sabinezitzmannkolbe immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT claudiakamfenkel immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT stefanstargard immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT stefaniehammer immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT jennifersjørgensen immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT malenejackerott immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT carstenhnielsen immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT christophaschatz immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT hartwighennekes immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT jennykarlsson immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT alanscuthbertson immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT dominikmumberg immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy
AT ursbhagemann immunostimulatoryeffectsoftargetedthorium227conjugatesassingleagentandincombinationwithantipdl1therapy