Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics

Abstract The scarcity of effective biomarkers and therapeutic strategies for predicting disease onset and progression in neurodegenerative diseases such as Alzheimer’s disease (AD) is a major challenge. Conventional drug discovery approaches have been unsuccessful in providing efficient intervention...

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Main Authors: Carla Cuní-López, Romal Stewart, Satomi Okano, Garry L. Redlich, Mark W. Appleby, Anthony R. White, Hazel Quek
Format: Article
Language:English
Published: Nature Portfolio 2025-03-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-92593-6
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author Carla Cuní-López
Romal Stewart
Satomi Okano
Garry L. Redlich
Mark W. Appleby
Anthony R. White
Hazel Quek
author_facet Carla Cuní-López
Romal Stewart
Satomi Okano
Garry L. Redlich
Mark W. Appleby
Anthony R. White
Hazel Quek
author_sort Carla Cuní-López
collection DOAJ
description Abstract The scarcity of effective biomarkers and therapeutic strategies for predicting disease onset and progression in neurodegenerative diseases such as Alzheimer’s disease (AD) is a major challenge. Conventional drug discovery approaches have been unsuccessful in providing efficient interventions due to their ‘one-size-fits-all’ nature. As an alternative, personalised drug development holds promise to pre-select responders and identifying suitable indicators of drug efficacy. In this exploratory study, we have established a pipeline with the potential to guide patient stratification studies before clinical trials. This pipeline uses 2D and 3D in vitro models of monocyte-derived microglia-like cells (MDMi) from AD and mild cognitive impairment (MCI) patients, and matched healthy control (HC) individuals. By profiling cytokine responses in these models using multidimensional analyses, we have observed that the 3D model offers a more defined separation of profiles between individuals based on disease status. While this pilot study focuses on AD and MCI, future investigations incorporating other neurodegenerative disorders will be necessary to validate the pipeline’s findings and demonstrate its broader applicability.
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publishDate 2025-03-01
publisher Nature Portfolio
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spelling doaj-art-8512c06f8e7b4d7184c6d22e7a592ea82025-08-20T03:01:38ZengNature PortfolioScientific Reports2045-23222025-03-0115111210.1038/s41598-025-92593-6Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeuticsCarla Cuní-López0Romal Stewart1Satomi Okano2Garry L. Redlich3Mark W. Appleby4Anthony R. White5Hazel Quek6Brain and Mental Health, QIMR Berghofer Medical Research InstituteBrain and Mental Health, QIMR Berghofer Medical Research InstituteStatistics, QIMR Berghofer Medical Research InstituteImplicit BioscienceImplicit BioscienceBrain and Mental Health, QIMR Berghofer Medical Research InstituteBrain and Mental Health, QIMR Berghofer Medical Research InstituteAbstract The scarcity of effective biomarkers and therapeutic strategies for predicting disease onset and progression in neurodegenerative diseases such as Alzheimer’s disease (AD) is a major challenge. Conventional drug discovery approaches have been unsuccessful in providing efficient interventions due to their ‘one-size-fits-all’ nature. As an alternative, personalised drug development holds promise to pre-select responders and identifying suitable indicators of drug efficacy. In this exploratory study, we have established a pipeline with the potential to guide patient stratification studies before clinical trials. This pipeline uses 2D and 3D in vitro models of monocyte-derived microglia-like cells (MDMi) from AD and mild cognitive impairment (MCI) patients, and matched healthy control (HC) individuals. By profiling cytokine responses in these models using multidimensional analyses, we have observed that the 3D model offers a more defined separation of profiles between individuals based on disease status. While this pilot study focuses on AD and MCI, future investigations incorporating other neurodegenerative disorders will be necessary to validate the pipeline’s findings and demonstrate its broader applicability.https://doi.org/10.1038/s41598-025-92593-6Preclinical drug testingPreclinical pipeline for predictionMonocyte-derived microgliaMDMi2D and 3D in vitro modelsPatient stratification
spellingShingle Carla Cuní-López
Romal Stewart
Satomi Okano
Garry L. Redlich
Mark W. Appleby
Anthony R. White
Hazel Quek
Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
Scientific Reports
Preclinical drug testing
Preclinical pipeline for prediction
Monocyte-derived microglia
MDMi
2D and 3D in vitro models
Patient stratification
title Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
title_full Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
title_fullStr Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
title_full_unstemmed Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
title_short Exploring a patient-specific in vitro pipeline for stratification and drug response prediction of microglia-based therapeutics
title_sort exploring a patient specific in vitro pipeline for stratification and drug response prediction of microglia based therapeutics
topic Preclinical drug testing
Preclinical pipeline for prediction
Monocyte-derived microglia
MDMi
2D and 3D in vitro models
Patient stratification
url https://doi.org/10.1038/s41598-025-92593-6
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