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Expert Opin Biol Ther. 2018 Aug;18(8):865-881. doi: 10.1080/14712598.2018.1503248.

Advances in stem cell therapy for amyotrophic lateral sclerosis.

Author information

a ALS Centre Department of Neurology , "Maggiore della Carità" University Hospital Novara , Novara , Italy.
b Department of Biotechnology and Biosciences , University Milano Bicocca , Milano , Italy.
c Center for laser microscopy, Faculty of Biology , University of Belgrade , Belgrade , Serbia.
d Stem Cell Bioengineering Unit , Mossakowski Medical Research Center, Polish Academy of Sciences , Warsaw , Poland.
e Scientific Direction , IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo , Foggia , Italy.
f Cell Factory e biobanca, Fondazione Cellule Staminali , Terni , Italy.
g A. I. Virtanen Institute for Molecular Sciences, University of Eastern Finland , Neulaniementie 2, Kuopio , FINLAND.
h Department of Molecular Medicine , Institute of Basic Medical Sciences, University of Oslo and Norwegian Center for Stem Cell Research, Oslo University Hospital , Oslo , Norway.
i Department Pharmaceutical Sciences , Laboratory of Neuroplasticity, University of Piemonte Orientale , Novara , Italy.
j Department of Neuroscience , Uppsala University Biomedical Centre , Uppsala , Sweden.
k Department of Biomedical Sciences and Center for Developmental Biology and Reprogramming (CEDEBIOR) , University of Sassari, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche (CNR) , Sassari , Italy.
l Laboratory for Stem Cells, Croatian Institute for Brain Research , University of Zagreb School of Medicine , Zagreb , Croatia.
m Department of Stem Cell Biology , State Research Institute Centre for Innovative Medicine , Vilnius , Lithuania.
n Preclinical Research , Andalusian Initiative for Advanced Therapies, Andalusian Health Ministry , Sevilla , Spain.


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.


Amyotrophic lateral sclerosis; animal models; cellular models; clinical trials; stem cells; transplantation

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