Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease

Svetlana Pasteuning-Vuhman; Rianne de Jongh; Annabel Timmers; R. Jeroen Pasterkamp

doi:10.1016/j.molmed.2020.09.013

Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease

Svetlana Pasteuning-Vuhman, Rianne de Jongh, Annabel Timmers, R. Jeroen Pasterkamp^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

Abstract

Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC modeling and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.

Original language	English
Pages (from-to)	263-279
Number of pages	17
Journal	Trends in molecular medicine
Volume	27
Issue number	3
DOIs	https://doi.org/10.1016/j.molmed.2020.09.013
Publication status	Published - Mar 2021

Keywords

brain organoids
drug development
iPSC
microfluidics
neurodegeneration
screening

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10.1016/j.molmed.2020.09.013

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title = "Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease",

abstract = "Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC modeling and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.",

keywords = "brain organoids, drug development, iPSC, microfluidics, neurodegeneration, screening",

author = "Svetlana Pasteuning-Vuhman and {de Jongh}, Rianne and Annabel Timmers and Pasterkamp, {R. Jeroen}",

note = "Funding Information: This work was supported by Stichting ALS Nederland (TOTALS, ALS on a Chip), Stichting Parkinson Fonds, the MAXOMOD and INTEGRALS consortia under the frame of E-Rare-3, the ERANet for Research on Rare Diseases, and by the EU Joint Program Neurodegenerative Diseases (JPND; TRIAGE) (to R.J.P.). Publisher Copyright: {\textcopyright} 2020 Elsevier Ltd Copyright: Copyright 2021 Elsevier B.V., All rights reserved.",

year = "2021",

month = mar,

doi = "10.1016/j.molmed.2020.09.013",

language = "English",

volume = "27",

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AU - Pasteuning-Vuhman, Svetlana

AU - de Jongh, Rianne

AU - Timmers, Annabel

AU - Pasterkamp, R. Jeroen

N1 - Funding Information: This work was supported by Stichting ALS Nederland (TOTALS, ALS on a Chip), Stichting Parkinson Fonds, the MAXOMOD and INTEGRALS consortia under the frame of E-Rare-3, the ERANet for Research on Rare Diseases, and by the EU Joint Program Neurodegenerative Diseases (JPND; TRIAGE) (to R.J.P.). Publisher Copyright: © 2020 Elsevier Ltd Copyright: Copyright 2021 Elsevier B.V., All rights reserved.

PY - 2021/3

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N2 - Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC modeling and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.

AB - Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC modeling and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.

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KW - iPSC

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JO - Trends in molecular medicine

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Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease

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Keywords

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