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

Lucia Dutan polit; Ilse Eidhof; Rhiannon V. Mcneill; Katherine M. Warre-Cornish; Cristine Marie Yde Ohki; Natalie Monet Walter; Carlo Sala; Chiara Verpelli; Franziska Radtke; Silvana Galderisi; Armida Mucci; Ginetta Collo; Frank Edenhofer; Maija L. Castrén; János M. Réthelyi; Morten Ejlersen; Sonja Simone Hohmann; Mirolyuba S. Ilieva; Renate Lukjanska; Rugile Matuleviciute; Tanja maria Michel; Femke m.s. De vrij; Steven a. Kushner; Bas Lendemeijer; Sarah Kittel-Schneider; Georg c. Ziegler; Doris Gruber-Schoffnegger; R. jeroen Pasterkamp; Amal Kasri; Marie-Claude Potier; Jürgen A. Knoblich; Oliver Brüstle; Michael Peitz; Emilio Merlo Pich; Adrian J. Harwood; Elsa Abranches; Anna Falk; Anthony C. Vernon; Edna Grünblatt; Deepak P. Srivastava

doi:10.1016/j.nsa.2023.101125

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

Lucia Dutan polit, Ilse Eidhof, Rhiannon V. Mcneill, Katherine M. Warre-Cornish, Cristine Marie Yde Ohki, Natalie Monet Walter, Carlo Sala, Chiara Verpelli, Franziska Radtke, Silvana Galderisi, Armida Mucci, Ginetta Collo, Frank Edenhofer, Maija L. Castrén, János M. Réthelyi, Morten Ejlersen, Sonja Simone Hohmann, Mirolyuba S. Ilieva, Renate Lukjanska, Rugile MatuleviciuteTanja maria Michel, Femke m.s. De vrij, Steven a. Kushner, Bas Lendemeijer, Sarah Kittel-Schneider, Georg c. Ziegler, Doris Gruber-Schoffnegger, R. jeroen Pasterkamp, Amal Kasri, Marie-Claude Potier, Jürgen A. Knoblich, Oliver Brüstle, Michael Peitz, Emilio Merlo Pich, Adrian J. Harwood, Elsa Abranches, Anna Falk, Anthony C. Vernon, Edna Grünblatt^*, Deepak P. Srivastava^*

^*Corresponding author for this work

Basic and Clinical Neuroscience

Research output: Contribution to journal › Article › peer-review

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 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.

Original language	English
Article number	101125
Journal	Neuroscience Applied
Volume	2
Early online date	20 Mar 2023
DOIs	https://doi.org/10.1016/j.nsa.2023.101125
Publication status	Published - 2023

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Dutan polit, L., Eidhof, I., Mcneill, R. V., Warre-Cornish, K. M., Yde Ohki, C. M., Walter, N. M., Sala, C., Verpelli, C., Radtke, F., Galderisi, S., Mucci, A., Collo, G., Edenhofer, F., Castrén, M. L., Réthelyi, J. M., Ejlersen, M., Hohmann, S. S., Ilieva, M. S., Lukjanska, R., ... Srivastava, D. P. (2023). Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders. Neuroscience Applied, 2, Article 101125. https://doi.org/10.1016/j.nsa.2023.101125

@article{1680284dbe014f5392ac4d35979bb1df,

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

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 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.",

author = "{Dutan polit}, Lucia and Ilse Eidhof and Mcneill, {Rhiannon V.} and Warre-Cornish, {Katherine M.} and {Yde Ohki}, {Cristine Marie} and Walter, {Natalie Monet} and Carlo Sala and Chiara Verpelli and Franziska Radtke and Silvana Galderisi and Armida Mucci and Ginetta Collo and Frank Edenhofer and Castr{\'e}n, {Maija L.} and R{\'e}thelyi, {J{\'a}nos M.} and Morten Ejlersen and Hohmann, {Sonja Simone} and Ilieva, {Mirolyuba S.} and Renate Lukjanska and Rugile Matuleviciute and Michel, {Tanja maria} and {De vrij}, {Femke m.s.} and Kushner, {Steven a.} and Bas Lendemeijer and Sarah Kittel-Schneider and Ziegler, {Georg c.} and Doris Gruber-Schoffnegger and Pasterkamp, {R. jeroen} and Amal Kasri and Marie-Claude Potier and Knoblich, {J{\"u}rgen A.} and Oliver Br{\"u}stle and Michael Peitz and Pich, {Emilio Merlo} and Harwood, {Adrian J.} and Elsa Abranches and Anna Falk and Vernon, {Anthony C.} and Edna Gr{\"u}nblatt and Srivastava, {Deepak P.}",

year = "2023",

doi = "10.1016/j.nsa.2023.101125",

language = "English",

volume = "2",

journal = "Neuroscience Applied",

issn = "2772-4085",

}

Dutan polit, L, Eidhof, I, Mcneill, RV, Warre-Cornish, KM, Yde Ohki, CM, Walter, NM, Sala, C, Verpelli, C, Radtke, F, Galderisi, S, Mucci, A, Collo, G, Edenhofer, F, Castrén, ML, Réthelyi, JM, Ejlersen, M, Hohmann, SS, Ilieva, MS, Lukjanska, R, Matuleviciute, R, Michel, TM, De vrij, FMS, Kushner, SA, Lendemeijer, B, Kittel-Schneider, S, Ziegler, GC, Gruber-Schoffnegger, D, Pasterkamp, RJ, Kasri, A, Potier, M-C, Knoblich, JA, Brüstle, O, Peitz, M, Pich, EM, Harwood, AJ, Abranches, E, Falk, A, Vernon, AC, Grünblatt, E & Srivastava, DP 2023, 'Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders', Neuroscience Applied, vol. 2, 101125. https://doi.org/10.1016/j.nsa.2023.101125

TY - JOUR

T1 - Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders

AU - Dutan polit, Lucia

AU - Eidhof, Ilse

AU - Mcneill, Rhiannon V.

AU - Warre-Cornish, Katherine M.

AU - Yde Ohki, Cristine Marie

AU - Walter, Natalie Monet

AU - Sala, Carlo

AU - Verpelli, Chiara

AU - Radtke, Franziska

AU - Galderisi, Silvana

AU - Mucci, Armida

AU - Collo, Ginetta

AU - Edenhofer, Frank

AU - Castrén, Maija L.

AU - Réthelyi, János M.

AU - Ejlersen, Morten

AU - Hohmann, Sonja Simone

AU - Ilieva, Mirolyuba S.

AU - Lukjanska, Renate

AU - Matuleviciute, Rugile

AU - Michel, Tanja maria

AU - De vrij, Femke m.s.

AU - Kushner, Steven a.

AU - Lendemeijer, Bas

AU - Kittel-Schneider, Sarah

AU - Ziegler, Georg c.

AU - Gruber-Schoffnegger, Doris

AU - Pasterkamp, R. jeroen

AU - Kasri, Amal

AU - Potier, Marie-Claude

AU - Knoblich, Jürgen A.

AU - Brüstle, Oliver

AU - Peitz, Michael

AU - Pich, Emilio Merlo

AU - Harwood, Adrian J.

AU - Abranches, Elsa

AU - Falk, Anna

AU - Vernon, Anthony C.

AU - Grünblatt, Edna

AU - Srivastava, Deepak P.

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

U2 - 10.1016/j.nsa.2023.101125

DO - 10.1016/j.nsa.2023.101125

M3 - Article

SN - 2772-4085

VL - 2

JO - Neuroscience Applied

JF - Neuroscience Applied

M1 - 101125

ER -

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

Abstract

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