TY - JOUR
T1 - Advances in stem cell therapy for amyotrophic lateral sclerosis
AU - on behalf of BIONECA COST ACTION WG Neurology
AU - Mazzini, Letizia
AU - Ferrari, Daniela
AU - Andjus, Pavle R.
AU - Buzanska, Leonora
AU - Cantello, Roberto
AU - De Marchi, Fabiola
AU - Gelati, Maurizio
AU - Giniatullin, Rashid
AU - Glover, Joel C.
AU - Grilli, Mariagrazia
AU - Kozlova, Elena N.
AU - Maioli, Margherita
AU - Mitrečić, Dinko
AU - Pivoriunas, Augustas
AU - Sanchez-Pernaute, Rosario
AU - Sarnowska, Anna
AU - Vescovi, Angelo L.
N1 - Funding Information:
The author(s) would like to acknowledge networking support by the COST Action CA16122. DM acknowledges Croatian Science Foundation, project IP-2016-06-9451 and co-financing by the European Union through the European Regional Development Fund, Operational Programme Competitiveness and Cohesion, grant agreement No. KK.01.1.1.01.0007, CoRE - Neuro. LB and AS acknowledge Polish National Centre for Research and Development grant Strategmed No. [1/234261/2/NCBR/2014]. The authors would like to apologize to colleagues whose excellent work could not be cited in this manuscript due to space limitations.
Funding Information:
Instituto Tecnologico y de Estudios Superiores de Monterrey (grant number CAT 014) and the Zambrano Hellion Foundation (Mexico)
Funding Information:
Carlos III Institute, Advanced Therapies and Transplant General Direction, ISCIII Spanish Cell Therapy Network, Prometeo Grant 2009/028, Rotary Club Elche-Illice and by the Fundación Diógenes ECTRIMS Fellowship grant; NMSS RG3630 and TEDCO MD Stem Cell Fund ESC 06–29–01 (Israel) Korea Healthcare Technology R&D Project of the Ministry for Health & Welfare Affairs (Republic of Korea)
Publisher Copyright:
© 2018, © 2018 Informa UK Limited, trading as Taylor & Francis Group.
Copyright:
Copyright 2019 Elsevier B.V., All rights reserved.
PY - 2018/8/3
Y1 - 2018/8/3
N2 - Introduction: Amyotrophic Lateral Sclerosis (ALS) is a progressive, incurable neurodegenerative disease that targets motoneurons. Cell-based therapies have generated widespread interest as a potential therapeutic approach but no conclusive results have yet been reported either from pre-clinical or clinical studies. Areas covered: This is an integrated review of pre-clinical and clinical studies focused on the development of cell-based therapies for ALS. We analyze the biology of stem cell treatments and results obtained from pre-clinical models of ALS and examine the methods and the results obtained to date from clinical trials. We discuss scientific, clinical, and ethical issues and propose some directions for future studies. Expert opinion: While data from individual studies are encouraging, stem-cell-based therapies do not yet represent a satisfactory, reliable clinical option. The field will critically benefit from the introduction of well-designed, randomized and reproducible, powered clinical trials. Comparative studies addressing key issues such as the nature, properties, and number of donor cells, the delivery mode and the selection of proper patient populations that may benefit the most from cell-based therapies are now of the essence. Multidisciplinary networks of experts should be established to empower effective translation of research into the clinic.
AB - Introduction: Amyotrophic Lateral Sclerosis (ALS) is a progressive, incurable neurodegenerative disease that targets motoneurons. Cell-based therapies have generated widespread interest as a potential therapeutic approach but no conclusive results have yet been reported either from pre-clinical or clinical studies. Areas covered: This is an integrated review of pre-clinical and clinical studies focused on the development of cell-based therapies for ALS. We analyze the biology of stem cell treatments and results obtained from pre-clinical models of ALS and examine the methods and the results obtained to date from clinical trials. We discuss scientific, clinical, and ethical issues and propose some directions for future studies. Expert opinion: While data from individual studies are encouraging, stem-cell-based therapies do not yet represent a satisfactory, reliable clinical option. The field will critically benefit from the introduction of well-designed, randomized and reproducible, powered clinical trials. Comparative studies addressing key issues such as the nature, properties, and number of donor cells, the delivery mode and the selection of proper patient populations that may benefit the most from cell-based therapies are now of the essence. Multidisciplinary networks of experts should be established to empower effective translation of research into the clinic.
KW - Amyotrophic lateral sclerosis
KW - animal models
KW - cellular models
KW - clinical trials
KW - stem cells
KW - transplantation
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U2 - 10.1080/14712598.2018.1503248
DO - 10.1080/14712598.2018.1503248
M3 - Review article
C2 - 30025485
AN - SCOPUS:85051491676
SN - 1471-2598
VL - 18
SP - 865
EP - 881
JO - Expert Opinion on Biological Therapy
JF - Expert Opinion on Biological Therapy
IS - 8
ER -