CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.

T cell acute lymphoblastic leukemia (T-ALL) is an aggressive malignancy characterized by high rates of induction failure and relapse, and effective targeted immunotherapies are lacking. Despite promising clinical progress with genome-edited CD7-directed CAR-T cells, which present significant logisti...

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Autores: Tirado, Néstor, Fidyt, Klaudyna, Mansilla, María José, Garcia-Perez, Alba, Martínez-Moreno, Alba, Vinyoles, Meritxell, Alcain, Juan, García-Peydró, Marina, Roca-Ho, Heleia, Fernandez-Fuentes, Narcís, Guerrero-Murillo, Mercedes, Falgàs, Aïda, Velasco-Hernandez, Talia, Bueno, Clara, Panelli, Patrizio, Mulens-Arias, Vladimir, Apostolov, Apostol, Engel Rocamora, Pablo, González Navarro, E. Azucena, Vick, Binje, Jeremias, Irmela, Caye-Eude, Aurélie, Baruchel, André, Cavé, Hélène, Genescà, Eulàlia, Ribera, Jordi, Díaz Beyà, Marina, Martínez-Sánchez, María Victoria, Fuster, José Luis, Escudero López, Adela, Minguillón, Jordi, Pérez-Martínez, Antonio, Ramírez-Orellana, Manuel, Torrebadell, Montserrat, Díaz, Víctor M., Toribio, María L., Sánchez-Martínez, Diego, Menéndez Buján, Pablo
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:2445/227381
Acceso en línea:https://hdl.handle.net/2445/227381
Access Level:acceso abierto
Palabra clave:Leucèmia
Oncologia
Leukemia
Oncology
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spelling CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.Tirado, NéstorFidyt, KlaudynaMansilla, María JoséGarcia-Perez, AlbaMartínez-Moreno, AlbaVinyoles, MeritxellAlcain, JuanGarcía-Peydró, MarinaRoca-Ho, HeleiaFernandez-Fuentes, NarcísGuerrero-Murillo, MercedesFalgàs, AïdaVelasco-Hernandez, TaliaBueno, ClaraPanelli, PatrizioMulens-Arias, VladimirApostolov, ApostolEngel Rocamora, PabloGonzález Navarro, E. AzucenaVick, BinjeJeremias, IrmelaCaye-Eude, AurélieBaruchel, AndréCavé, HélèneGenescà, EulàliaRibera, JordiDíaz Beyà, MarinaMartínez-Sánchez, María VictoriaFuster, José LuisEscudero López, AdelaMinguillón, JordiPérez-Martínez, AntonioRamírez-Orellana, ManuelTorrebadell, MontserratDíaz, Víctor M.Toribio, María L.Sánchez-Martínez, DiegoMenéndez Buján, PabloLeucèmiaOncologiaLeukemiaOncologyT cell acute lymphoblastic leukemia (T-ALL) is an aggressive malignancy characterized by high rates of induction failure and relapse, and effective targeted immunotherapies are lacking. Despite promising clinical progress with genome-edited CD7-directed CAR-T cells, which present significant logistical and regulatory issues, CAR-T cell therapy in T-ALL remains challenging due to the shared antigen expression between malignant and healthy T cells. This can result in CAR-T cell fratricide, T cell aplasia, and the potential for blast contamination during CAR-T cell manufacturing. Recently described CAR-T cells target non-pan-T antigens, absent on healthy T cells but expressed on specific T-ALL subsets. These antigens include CD1a (NCT05679895), which is expressed in cortical T-ALL, and CCR9. We show that CCR9 is expressed on >70% of T-ALL patients (132/180) and is maintained at relapse, with a safe expression profile in healthy hematopoietic and non-hematopoietic tissues. Further analyses showed that dual targeting of CCR9 and CD1a could benefit T-ALL patients with a greater blast coverage than single CAR-T cell treatments. We therefore developed, characterized, and preclinically validated a novel humanized CCR9-specific CAR with robust and specific antileukemic activity as a monotherapy in vitro and in vivo against cell lines, primary T-ALL samples, and patientderived xenografts. Importantly, CCR9/CD1a dual-targeting CAR-T cells showed higher efficacy than single-targeting CAR-T cells, particularly in T-ALL cases with phenotypically heterogeneous leukemic populations. Dual CD1a/CCR9 CAR-T therapy may prevent T cell aplasia and obviate the need for allogeneic transplantation and regulatory-challenging genome engineering approaches in T-ALL.BioMed Central2026202620252026info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion16 p.application/pdfhttps://hdl.handle.net/2445/227381Articles publicats en revistes (Biomedicina)reponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésReproducció del document publicat a: https://doi.org/10.1186/s13045-025-01715-0Journal of Hematology & Oncology, 2025, vol. 18, num.1https://doi.org/10.1186/s13045-025-01715-0cc-by-nc-nd (c) Tirado, Néstor et al., 2025https://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:recercat.cat:2445/2273812026-05-29T05:05:01Z
dc.title.none.fl_str_mv CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
title CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
spellingShingle CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
Tirado, Néstor
Leucèmia
Oncologia
Leukemia
Oncology
title_short CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
title_full CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
title_fullStr CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
title_full_unstemmed CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
title_sort CAR-T cells targeting CCR9 and CD1a for the treatment of T cell acute lymphoblastic leukemia.
dc.creator.none.fl_str_mv Tirado, Néstor
Fidyt, Klaudyna
Mansilla, María José
Garcia-Perez, Alba
Martínez-Moreno, Alba
Vinyoles, Meritxell
Alcain, Juan
García-Peydró, Marina
Roca-Ho, Heleia
Fernandez-Fuentes, Narcís
Guerrero-Murillo, Mercedes
Falgàs, Aïda
Velasco-Hernandez, Talia
Bueno, Clara
Panelli, Patrizio
Mulens-Arias, Vladimir
Apostolov, Apostol
Engel Rocamora, Pablo
González Navarro, E. Azucena
Vick, Binje
Jeremias, Irmela
Caye-Eude, Aurélie
Baruchel, André
Cavé, Hélène
Genescà, Eulàlia
Ribera, Jordi
Díaz Beyà, Marina
Martínez-Sánchez, María Victoria
Fuster, José Luis
Escudero López, Adela
Minguillón, Jordi
Pérez-Martínez, Antonio
Ramírez-Orellana, Manuel
Torrebadell, Montserrat
Díaz, Víctor M.
Toribio, María L.
Sánchez-Martínez, Diego
Menéndez Buján, Pablo
author Tirado, Néstor
author_facet Tirado, Néstor
Fidyt, Klaudyna
Mansilla, María José
Garcia-Perez, Alba
Martínez-Moreno, Alba
Vinyoles, Meritxell
Alcain, Juan
García-Peydró, Marina
Roca-Ho, Heleia
Fernandez-Fuentes, Narcís
Guerrero-Murillo, Mercedes
Falgàs, Aïda
Velasco-Hernandez, Talia
Bueno, Clara
Panelli, Patrizio
Mulens-Arias, Vladimir
Apostolov, Apostol
Engel Rocamora, Pablo
González Navarro, E. Azucena
Vick, Binje
Jeremias, Irmela
Caye-Eude, Aurélie
Baruchel, André
Cavé, Hélène
Genescà, Eulàlia
Ribera, Jordi
Díaz Beyà, Marina
Martínez-Sánchez, María Victoria
Fuster, José Luis
Escudero López, Adela
Minguillón, Jordi
Pérez-Martínez, Antonio
Ramírez-Orellana, Manuel
Torrebadell, Montserrat
Díaz, Víctor M.
Toribio, María L.
Sánchez-Martínez, Diego
Menéndez Buján, Pablo
author_role author
author2 Fidyt, Klaudyna
Mansilla, María José
Garcia-Perez, Alba
Martínez-Moreno, Alba
Vinyoles, Meritxell
Alcain, Juan
García-Peydró, Marina
Roca-Ho, Heleia
Fernandez-Fuentes, Narcís
Guerrero-Murillo, Mercedes
Falgàs, Aïda
Velasco-Hernandez, Talia
Bueno, Clara
Panelli, Patrizio
Mulens-Arias, Vladimir
Apostolov, Apostol
Engel Rocamora, Pablo
González Navarro, E. Azucena
Vick, Binje
Jeremias, Irmela
Caye-Eude, Aurélie
Baruchel, André
Cavé, Hélène
Genescà, Eulàlia
Ribera, Jordi
Díaz Beyà, Marina
Martínez-Sánchez, María Victoria
Fuster, José Luis
Escudero López, Adela
Minguillón, Jordi
Pérez-Martínez, Antonio
Ramírez-Orellana, Manuel
Torrebadell, Montserrat
Díaz, Víctor M.
Toribio, María L.
Sánchez-Martínez, Diego
Menéndez Buján, Pablo
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Leucèmia
Oncologia
Leukemia
Oncology
topic Leucèmia
Oncologia
Leukemia
Oncology
description T cell acute lymphoblastic leukemia (T-ALL) is an aggressive malignancy characterized by high rates of induction failure and relapse, and effective targeted immunotherapies are lacking. Despite promising clinical progress with genome-edited CD7-directed CAR-T cells, which present significant logistical and regulatory issues, CAR-T cell therapy in T-ALL remains challenging due to the shared antigen expression between malignant and healthy T cells. This can result in CAR-T cell fratricide, T cell aplasia, and the potential for blast contamination during CAR-T cell manufacturing. Recently described CAR-T cells target non-pan-T antigens, absent on healthy T cells but expressed on specific T-ALL subsets. These antigens include CD1a (NCT05679895), which is expressed in cortical T-ALL, and CCR9. We show that CCR9 is expressed on >70% of T-ALL patients (132/180) and is maintained at relapse, with a safe expression profile in healthy hematopoietic and non-hematopoietic tissues. Further analyses showed that dual targeting of CCR9 and CD1a could benefit T-ALL patients with a greater blast coverage than single CAR-T cell treatments. We therefore developed, characterized, and preclinically validated a novel humanized CCR9-specific CAR with robust and specific antileukemic activity as a monotherapy in vitro and in vivo against cell lines, primary T-ALL samples, and patientderived xenografts. Importantly, CCR9/CD1a dual-targeting CAR-T cells showed higher efficacy than single-targeting CAR-T cells, particularly in T-ALL cases with phenotypically heterogeneous leukemic populations. Dual CD1a/CCR9 CAR-T therapy may prevent T cell aplasia and obviate the need for allogeneic transplantation and regulatory-challenging genome engineering approaches in T-ALL.
publishDate 2025
dc.date.none.fl_str_mv 2025
2026
2026
2026
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/2445/227381
url https://hdl.handle.net/2445/227381
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Reproducció del document publicat a: https://doi.org/10.1186/s13045-025-01715-0
Journal of Hematology & Oncology, 2025, vol. 18, num.1
https://doi.org/10.1186/s13045-025-01715-0
dc.rights.none.fl_str_mv cc-by-nc-nd (c) Tirado, Néstor et al., 2025
https://creativecommons.org/licenses/by-nc-nd/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc-by-nc-nd (c) Tirado, Néstor et al., 2025
https://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 16 p.
application/pdf
dc.publisher.none.fl_str_mv BioMed Central
publisher.none.fl_str_mv BioMed Central
dc.source.none.fl_str_mv Articles publicats en revistes (Biomedicina)
reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
repository.name.fl_str_mv
repository.mail.fl_str_mv
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