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Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders

Dutan Polit, Lucia ; Eidhof, Ilse ; McNeill, Rhiannon V. ; Warre-Cornish, Katherine M. ; Yde Ohki, Cristine Marie ; Walter, Natalie Monet ; Sala, Carlo ; Verpelli, Chiara ; Radtke, Franziska and Galderisi, Silvana , et al. (2023) In Neuroscience Applied 2.
Abstract

The number of individuals suffering from neuropsychiatric disorders (NPDs) has increased worldwide, with 3 million disability-adjusted life-years calculated in 2019. Though research using various approaches including genetics, imaging, clinical and animal models has advanced our knowledge regarding NPDs, we still lack basic knowledge regarding the underlying pathophysiological mechanisms. Moreover, there is an urgent need for highly effective therapeutics for NPDs. Human induced pluripotent stem cells (hiPSCs) generated from somatic cells enabled scientists to create brain cells in a patient-specific manner. However, there are challenges to the use of hiPSCs that need to be addressed. In the current paper, consideration of best... (More)

The number of individuals suffering from neuropsychiatric disorders (NPDs) has increased worldwide, with 3 million disability-adjusted life-years calculated in 2019. Though research using various approaches including genetics, imaging, clinical and animal models has advanced our knowledge regarding NPDs, we still lack basic knowledge regarding the underlying pathophysiological mechanisms. Moreover, there is an urgent need for highly effective therapeutics for NPDs. Human induced pluripotent stem cells (hiPSCs) generated from somatic cells enabled scientists to create brain cells in a patient-specific manner. However, there are challenges to the use of hiPSCs that need to be addressed. In the current paper, consideration of best practices for neuropharmacological and neuropsychiatric research using hiPSCs will be discussed. Specifically, we provide recommendations for best practice in patient recruitment, including collecting demographic, clinical, medical (before and after treatment and response), diagnostic (including scales) and genetic data from the donors. We highlight considerations regarding donor genetics and sex, in addition to discussing biological and technical replicates. Furthermore, we present our views on selecting control groups/lines, experimental designs, and considerations for conducting neuropharmacological studies using hiPSC-based models in the context of NPDs. In doing so, we explore key issues in the field concerning reproducibility, statistical analysis, and how to translate in vitro studies into clinically relevant observations. The aim of this article is to provide a key resource for hiPSC researchers to perform robust and reproducible neuropharmacological studies, with the ultimate aim of improving identification and clinical translation of novel therapeutic drugs for NPDs.

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type
Contribution to journal
publication status
published
subject
keywords
Cellular models, Drug discovery, Neurodevelopmental disorders, Neuropsychiatry, Neuropsychopharmacology, Sex as a biological variable (SABV)
in
Neuroscience Applied
volume
2
article number
101125
publisher
Elsevier
external identifiers
  • scopus:85209586967
DOI
10.1016/j.nsa.2023.101125
language
English
LU publication?
yes
id
67501b96-32a1-4020-8d93-7afdd87dc37a
date added to LUP
2025-03-12 14:24:59
date last changed
2025-04-04 14:07:14
@article{67501b96-32a1-4020-8d93-7afdd87dc37a,
  abstract     = {{<p>The number of individuals suffering from neuropsychiatric disorders (NPDs) has increased worldwide, with 3 million disability-adjusted life-years calculated in 2019. Though research using various approaches including genetics, imaging, clinical and animal models has advanced our knowledge regarding NPDs, we still lack basic knowledge regarding the underlying pathophysiological mechanisms. Moreover, there is an urgent need for highly effective therapeutics for NPDs. Human induced pluripotent stem cells (hiPSCs) generated from somatic cells enabled scientists to create brain cells in a patient-specific manner. However, there are challenges to the use of hiPSCs that need to be addressed. In the current paper, consideration of best practices for neuropharmacological and neuropsychiatric research using hiPSCs will be discussed. Specifically, we provide recommendations for best practice in patient recruitment, including collecting demographic, clinical, medical (before and after treatment and response), diagnostic (including scales) and genetic data from the donors. We highlight considerations regarding donor genetics and sex, in addition to discussing biological and technical replicates. Furthermore, we present our views on selecting control groups/lines, experimental designs, and considerations for conducting neuropharmacological studies using hiPSC-based models in the context of NPDs. In doing so, we explore key issues in the field concerning reproducibility, statistical analysis, and how to translate in vitro studies into clinically relevant observations. The aim of this article is to provide a key resource for hiPSC researchers to perform robust and reproducible neuropharmacological studies, with the ultimate aim of improving identification and clinical translation of novel therapeutic drugs for NPDs.</p>}},
  author       = {{Dutan Polit, Lucia and Eidhof, Ilse and McNeill, Rhiannon V. and Warre-Cornish, Katherine M. and Yde Ohki, Cristine Marie and Walter, Natalie Monet and Sala, Carlo and Verpelli, Chiara and Radtke, Franziska and Galderisi, Silvana and Mucci, Armida and Collo, Ginetta and Edenhofer, Frank and Castrén, Maija L. and Réthelyi, János M. and Ejlersen, Morten and Hohmann, Sonja Simone and Ilieva, Mirolyuba S. and Lukjanska, Renate and Matuleviciute, Rugile and Michel, Tanja Maria and de Vrij, Femke M.S. and Kushner, Steven A. and Lendemeijer, Bas and Kittel-Schneider, Sarah and Ziegler, Georg C. and Gruber-Schoffnegger, Doris and Pasterkamp, R. Jeroen and Kasri, Amal and Potier, Marie Claude and Knoblich, Jürgen A. and Brüstle, Oliver and Peitz, Michael and Pich, Emilio Merlo and Harwood, Adrian J. and Abranches, Elsa and Falk, Anna and Vernon, Anthony C. and Grünblatt, Edna and Srivastava, Deepak P.}},
  keywords     = {{Cellular models; Drug discovery; Neurodevelopmental disorders; Neuropsychiatry; Neuropsychopharmacology; Sex as a biological variable (SABV)}},
  language     = {{eng}},
  publisher    = {{Elsevier}},
  series       = {{Neuroscience Applied}},
  title        = {{Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders}},
  url          = {{http://dx.doi.org/10.1016/j.nsa.2023.101125}},
  doi          = {{10.1016/j.nsa.2023.101125}},
  volume       = {{2}},
  year         = {{2023}},
}