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

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 FM, Kushner SA, Lendemeijer B, Kittel-Schneider S, Ziegler GC, Gruber-Schoffnegger D, Pasterkamp RJ, Kasri A, Potier MC, Knoblich JA, Brüstle O, Peitz M, Pich EM, Harwood AJ, Abranches E, Falk A, Vernon AC, Grünblatt E, Srivastava DP (2023)

Publication Type: Journal article, Review article

Publication year: 2023

Book Volume: 2

Article Number: 101125

DOI: 10.1016/j.nsa.2023.101125

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.

Involved external institutions

Odense Universitetshospital (OUH) Denmark (DK) Universitätsklinikum Würzburg Germany (DE) Karolinska Institute Sweden (SE) King’s College London United Kingdom (GB) Psychiatrische Universitätsklinik Zürich Switzerland (CH) Institut für Molekulare Biotechnologie / Institute of Molecular Biotechnology (IMBA) Austria (AT) Rheinische Friedrich-Wilhelms-Universität Bonn Germany (DE) National Institute for Biological Standards and Control (NIBSC) United Kingdom (GB) Consiglio Nazionale delle Ricerche (CNR) / National Research Council of Italy Italy (IT) Università degli studi della Campania Luigi Vanvitelli Italy (IT) Università degli Studi di Brescia Italy (IT) Leopold-Franzens-Universität Innsbruck / University of Innsbruck Austria (AT) Helsingin yliopisto / University of Helsinki Finland (FI) Semmelweis University / Semmelweis Egyetem Hungary (HU) Erasmus University Medical Center (MC) Netherlands (NL) University Medical Centre Utrecht (UMC Utrecht) Netherlands (NL) Evotec AG Germany (DE) Pitié-Salpêtrière University Hospital / Hôpital universitaire Pitié-Salpêtrière France (FR) Cardiff University United Kingdom (GB)

How to cite

APA:

Dutan Polit, L., Eidhof, I., McNeill, R.V., Warre-Cornish, K.M., Yde Ohki, C.M., Walter, N.M.,... Srivastava, D.P. (2023). Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders. , 2. https://doi.org/10.1016/j.nsa.2023.101125

MLA:

Dutan Polit, Lucia, et al. "Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders." 2 (2023).

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