A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis

Abstract We report a medium‐throughput drug‐screening platform (METPlatform) based on organotypic cultures that allows to evaluate inhibitors against metastases growing in situ. By applying this approach to the unmet clinical need of brain metastasis, we identified several vulnerabilities. Among the...

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Main Authors: Lucía Zhu, Diana Retana, Pedro García‐Gómez, Laura Álvaro‐Espinosa, Neibla Priego, Mariam Masmudi‐Martín, Natalia Yebra, Lauritz Miarka, Elena Hernández‐Encinas, Carmen Blanco‐Aparicio, Sonia Martínez, Cecilia Sobrino, Nuria Ajenjo, Maria‐Jesus Artiga, Eva Ortega‐Paino, Raúl Torres‐Ruiz, Sandra Rodríguez‐Perales, RENACER, Riccardo Soffietti, Luca Bertero, Paola Cassoni, Tobias Weiss, Javier Muñoz, Juan Manuel Sepúlveda, Pedro González‐León, Luis Jiménez‐Roldán, Luis Miguel Moreno, Olga Esteban, Ángel Pérez‐Núñez, Aurelio Hernández‐Laín, Oscar Toldos, Yolanda Ruano, Lucía Alcázar, Guillermo Blasco, José Fernández‐Alén, Eduardo Caleiras, Miguel Lafarga, Diego Megías, Osvaldo Graña‐Castro, Carolina Nör, Michael D Taylor, Leonie S Young, Damir Varešlija, Nicola Cosgrove, Fergus J Couch, Lorena Cussó, Manuel Desco, Silvana Mouron, Miguel Quintela‐Fandino, Michael Weller, Joaquín Pastor, Manuel Valiente, Adolfo de la Lama‐Zaragoza, Lourdes Calero‐Felix, Concepcion Fiaño‐Valverde, Pedro David Delgado‐López, Antonio Montalvo‐Afonso, Mar Pascual‐Llorente, Ángela Díaz‐Piqueras, SH Nam‐Cha, Cristina Barrena López, Gerard Plans Ahicart, Elena Martínez‐Saez, Santiago Ramón y Cajal, Pilar Nicolás
Format: Article
Language:English
Published: Springer Nature 2022-02-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.15252/emmm.202114552
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author Lucía Zhu
Diana Retana
Pedro García‐Gómez
Laura Álvaro‐Espinosa
Neibla Priego
Mariam Masmudi‐Martín
Natalia Yebra
Lauritz Miarka
Elena Hernández‐Encinas
Carmen Blanco‐Aparicio
Sonia Martínez
Cecilia Sobrino
Nuria Ajenjo
Maria‐Jesus Artiga
Eva Ortega‐Paino
Raúl Torres‐Ruiz
Sandra Rodríguez‐Perales
RENACER
Riccardo Soffietti
Luca Bertero
Paola Cassoni
Tobias Weiss
Javier Muñoz
Juan Manuel Sepúlveda
Pedro González‐León
Luis Jiménez‐Roldán
Luis Miguel Moreno
Olga Esteban
Ángel Pérez‐Núñez
Aurelio Hernández‐Laín
Oscar Toldos
Yolanda Ruano
Lucía Alcázar
Guillermo Blasco
José Fernández‐Alén
Eduardo Caleiras
Miguel Lafarga
Diego Megías
Osvaldo Graña‐Castro
Carolina Nör
Michael D Taylor
Leonie S Young
Damir Varešlija
Nicola Cosgrove
Fergus J Couch
Lorena Cussó
Manuel Desco
Silvana Mouron
Miguel Quintela‐Fandino
Michael Weller
Joaquín Pastor
Manuel Valiente
Adolfo de la Lama‐Zaragoza
Lourdes Calero‐Felix
Concepcion Fiaño‐Valverde
Pedro David Delgado‐López
Antonio Montalvo‐Afonso
Mar Pascual‐Llorente
Ángela Díaz‐Piqueras
SH Nam‐Cha
Cristina Barrena López
Gerard Plans Ahicart
Elena Martínez‐Saez
Santiago Ramón y Cajal
Pilar Nicolás
author_facet Lucía Zhu
Diana Retana
Pedro García‐Gómez
Laura Álvaro‐Espinosa
Neibla Priego
Mariam Masmudi‐Martín
Natalia Yebra
Lauritz Miarka
Elena Hernández‐Encinas
Carmen Blanco‐Aparicio
Sonia Martínez
Cecilia Sobrino
Nuria Ajenjo
Maria‐Jesus Artiga
Eva Ortega‐Paino
Raúl Torres‐Ruiz
Sandra Rodríguez‐Perales
RENACER
Riccardo Soffietti
Luca Bertero
Paola Cassoni
Tobias Weiss
Javier Muñoz
Juan Manuel Sepúlveda
Pedro González‐León
Luis Jiménez‐Roldán
Luis Miguel Moreno
Olga Esteban
Ángel Pérez‐Núñez
Aurelio Hernández‐Laín
Oscar Toldos
Yolanda Ruano
Lucía Alcázar
Guillermo Blasco
José Fernández‐Alén
Eduardo Caleiras
Miguel Lafarga
Diego Megías
Osvaldo Graña‐Castro
Carolina Nör
Michael D Taylor
Leonie S Young
Damir Varešlija
Nicola Cosgrove
Fergus J Couch
Lorena Cussó
Manuel Desco
Silvana Mouron
Miguel Quintela‐Fandino
Michael Weller
Joaquín Pastor
Manuel Valiente
Adolfo de la Lama‐Zaragoza
Lourdes Calero‐Felix
Concepcion Fiaño‐Valverde
Pedro David Delgado‐López
Antonio Montalvo‐Afonso
Mar Pascual‐Llorente
Ángela Díaz‐Piqueras
SH Nam‐Cha
Cristina Barrena López
Gerard Plans Ahicart
Elena Martínez‐Saez
Santiago Ramón y Cajal
Pilar Nicolás
author_sort Lucía Zhu
collection DOAJ
description Abstract We report a medium‐throughput drug‐screening platform (METPlatform) based on organotypic cultures that allows to evaluate inhibitors against metastases growing in situ. By applying this approach to the unmet clinical need of brain metastasis, we identified several vulnerabilities. Among them, a blood–brain barrier permeable HSP90 inhibitor showed high potency against mouse and human brain metastases at clinically relevant stages of the disease, including a novel model of local relapse after neurosurgery. Furthermore, in situ proteomic analysis applied to metastases treated with the chaperone inhibitor uncovered a novel molecular program in brain metastasis, which includes biomarkers of poor prognosis and actionable mechanisms of resistance. Our work validates METPlatform as a potent resource for metastasis research integrating drug‐screening and unbiased omic approaches that is compatible with human samples. Thus, this clinically relevant strategy is aimed to personalize the management of metastatic disease in the brain and elsewhere.
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publishDate 2022-02-01
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series EMBO Molecular Medicine
spelling doaj-art-192ccb44b2e3449a849f6353de0e50dd2025-08-20T03:43:14ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842022-02-0114312910.15252/emmm.202114552A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasisLucía Zhu0Diana Retana1Pedro García‐Gómez2Laura Álvaro‐Espinosa3Neibla Priego4Mariam Masmudi‐Martín5Natalia Yebra6Lauritz Miarka7Elena Hernández‐Encinas8Carmen Blanco‐Aparicio9Sonia Martínez10Cecilia Sobrino11Nuria Ajenjo12Maria‐Jesus Artiga13Eva Ortega‐Paino14Raúl Torres‐Ruiz15Sandra Rodríguez‐Perales16RENACERRiccardo Soffietti17Luca Bertero18Paola Cassoni19Tobias Weiss20Javier Muñoz21Juan Manuel Sepúlveda22Pedro González‐León23Luis Jiménez‐Roldán24Luis Miguel Moreno25Olga Esteban26Ángel Pérez‐Núñez27Aurelio Hernández‐Laín28Oscar Toldos29Yolanda Ruano30Lucía Alcázar31Guillermo Blasco32José Fernández‐Alén33Eduardo Caleiras34Miguel Lafarga35Diego Megías36Osvaldo Graña‐Castro37Carolina Nör38Michael D Taylor39Leonie S Young40Damir Varešlija41Nicola Cosgrove42Fergus J Couch43Lorena Cussó44Manuel Desco45Silvana Mouron46Miguel Quintela‐Fandino47Michael Weller48Joaquín Pastor49Manuel Valiente50Adolfo de la Lama‐ZaragozaLourdes Calero‐FelixConcepcion Fiaño‐ValverdePedro David Delgado‐LópezAntonio Montalvo‐AfonsoMar Pascual‐LlorenteÁngela Díaz‐PiquerasSH Nam‐ChaCristina Barrena LópezGerard Plans AhicartElena Martínez‐SaezSantiago Ramón y CajalPilar NicolásBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOBrain Metastasis Group, CNIOExperimental Therapeutics Programme, CNIOExperimental Therapeutics Programme, CNIOExperimental Therapeutics Programme, CNIOBiobank, CNIOBiobank, CNIOBiobank, CNIOBiobank, CNIOMolecular Cytogenetics Unit, CNIOMolecular Cytogenetics Unit, CNIODepartment of Neuro‐Oncology, University and City of Health and Science HospitalDepartment of Medical Sciences, University of TurinDepartment of Medical Sciences, University of TurinDepartment of Neurology, Clinical Neuroscience Center, University Hospital Zurich and University of ZurichProteomics Unit, ProteoRedISCIII, CNIONeuro‐Oncology Unit, Hospital Universitario 12 de OctubreNeurosurgery Unit, Hospital Universitario 12 de OctubreNeurosurgery Unit, Hospital Universitario 12 de OctubreNeurosurgery Unit, Hospital Universitario 12 de OctubreNeurosurgery Unit, Hospital Universitario 12 de OctubreNeurosurgery Unit, Hospital Universitario 12 de OctubreNeuropathology Unit, Instituto i+12, Hospital Universitario 12 de OctubreNeuropathology Unit, Instituto i+12, Hospital Universitario 12 de OctubrePathology Department, Instituto i+12, Hospital Universitario 12 de OctubreNeurosurgery Department, Hospital Universitario de La PrincesaNeurosurgery Department, Hospital Universitario de La PrincesaNeurosurgery Department, Hospital Universitario de La PrincesaHistopathology Unit, CNIODepartment of Anatomy and Cell Biology and Centro de Investigación Biomédica en Red sobre Enfermedades Neurodegenerativas (CIBERNED), University of Cantabria‐IDIVALConfocal Microscopy Unit, CNIOBioinformatics Unit, CNIODevelopmental and Stem Cell Biology Program and The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick ChildrenDevelopmental and Stem Cell Biology Program and The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick ChildrenEndocrine Oncology Research Group, Department of Surgery, RCSI University of Medicine and Health SciencesEndocrine Oncology Research Group, Department of Surgery, RCSI University of Medicine and Health SciencesEndocrine Oncology Research Group, Department of Surgery, RCSI University of Medicine and Health SciencesDepartment of Laboratory Medicine and Pathology, Mayo ClinicDepartamento de Bioingeniería e Ingeniería Aeroespacial, Universidad Carlos III de MadridDepartamento de Bioingeniería e Ingeniería Aeroespacial, Universidad Carlos III de MadridBreast Cancer Clinical Research Unit, CNIOBreast Cancer Clinical Research Unit, CNIODepartment of Neurology, Clinical Neuroscience Center, University Hospital Zurich and University of ZurichExperimental Therapeutics Programme, CNIOBrain Metastasis Group, CNIOAbstract We report a medium‐throughput drug‐screening platform (METPlatform) based on organotypic cultures that allows to evaluate inhibitors against metastases growing in situ. By applying this approach to the unmet clinical need of brain metastasis, we identified several vulnerabilities. Among them, a blood–brain barrier permeable HSP90 inhibitor showed high potency against mouse and human brain metastases at clinically relevant stages of the disease, including a novel model of local relapse after neurosurgery. Furthermore, in situ proteomic analysis applied to metastases treated with the chaperone inhibitor uncovered a novel molecular program in brain metastasis, which includes biomarkers of poor prognosis and actionable mechanisms of resistance. Our work validates METPlatform as a potent resource for metastasis research integrating drug‐screening and unbiased omic approaches that is compatible with human samples. Thus, this clinically relevant strategy is aimed to personalize the management of metastatic disease in the brain and elsewhere.https://doi.org/10.15252/emmm.202114552drug‐screenmetastasisorganotypic culturespatient‐derivedresistance
spellingShingle Lucía Zhu
Diana Retana
Pedro García‐Gómez
Laura Álvaro‐Espinosa
Neibla Priego
Mariam Masmudi‐Martín
Natalia Yebra
Lauritz Miarka
Elena Hernández‐Encinas
Carmen Blanco‐Aparicio
Sonia Martínez
Cecilia Sobrino
Nuria Ajenjo
Maria‐Jesus Artiga
Eva Ortega‐Paino
Raúl Torres‐Ruiz
Sandra Rodríguez‐Perales
RENACER
Riccardo Soffietti
Luca Bertero
Paola Cassoni
Tobias Weiss
Javier Muñoz
Juan Manuel Sepúlveda
Pedro González‐León
Luis Jiménez‐Roldán
Luis Miguel Moreno
Olga Esteban
Ángel Pérez‐Núñez
Aurelio Hernández‐Laín
Oscar Toldos
Yolanda Ruano
Lucía Alcázar
Guillermo Blasco
José Fernández‐Alén
Eduardo Caleiras
Miguel Lafarga
Diego Megías
Osvaldo Graña‐Castro
Carolina Nör
Michael D Taylor
Leonie S Young
Damir Varešlija
Nicola Cosgrove
Fergus J Couch
Lorena Cussó
Manuel Desco
Silvana Mouron
Miguel Quintela‐Fandino
Michael Weller
Joaquín Pastor
Manuel Valiente
Adolfo de la Lama‐Zaragoza
Lourdes Calero‐Felix
Concepcion Fiaño‐Valverde
Pedro David Delgado‐López
Antonio Montalvo‐Afonso
Mar Pascual‐Llorente
Ángela Díaz‐Piqueras
SH Nam‐Cha
Cristina Barrena López
Gerard Plans Ahicart
Elena Martínez‐Saez
Santiago Ramón y Cajal
Pilar Nicolás
A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
EMBO Molecular Medicine
drug‐screen
metastasis
organotypic cultures
patient‐derived
resistance
title A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
title_full A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
title_fullStr A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
title_full_unstemmed A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
title_short A clinically compatible drug‐screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
title_sort clinically compatible drug screening platform based on organotypic cultures identifies vulnerabilities to prevent and treat brain metastasis
topic drug‐screen
metastasis
organotypic cultures
patient‐derived
resistance
url https://doi.org/10.15252/emmm.202114552
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AT eduardocaleiras clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
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AT diegomegias clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
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AT michaeldtaylor clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
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AT damirvareslija clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT nicolacosgrove clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
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AT manueldesco clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT silvanamouron clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT miguelquintelafandino clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT michaelweller clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT joaquinpastor clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT manuelvaliente clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT adolfodelalamazaragoza clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT lourdescalerofelix clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT concepcionfianovalverde clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT pedrodaviddelgadolopez clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT antoniomontalvoafonso clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT marpascualllorente clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT angeladiazpiqueras clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT shnamcha clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT cristinabarrenalopez clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT gerardplansahicart clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT elenamartinezsaez clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT santiagoramonycajal clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis
AT pilarnicolas clinicallycompatibledrugscreeningplatformbasedonorganotypicculturesidentifiesvulnerabilitiestopreventandtreatbrainmetastasis