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Mol Neurodegener. 2017 Nov 13;12(1):85. doi: 10.1186/s13024-017-0227-3.

Advances, challenges and future directions for stem cell therapy in amyotrophic lateral sclerosis.

Author information

1
Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Faculty of Medicine, Dentistry and Health, University of Sheffield, 385a Glossop Rd S10 2HQ, Sheffield, UK.
2
Tongji University School of Medicine, 1239 Siping Rd, Yangpu Qu, Shanghai, China.
3
Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Faculty of Medicine, Dentistry and Health, University of Sheffield, 385a Glossop Rd S10 2HQ, Sheffield, UK. r.j.mead@sheffield.ac.uk.

Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative condition where loss of motor neurons within the brain and spinal cord leads to muscle atrophy, weakness, paralysis and ultimately death within 3-5 years from onset of symptoms. The specific molecular mechanisms underlying the disease pathology are not fully understood and neuroprotective treatment options are minimally effective. In recent years, stem cell transplantation as a new therapy for ALS patients has been extensively investigated, becoming an intense and debated field of study. In several preclinical studies using the SOD1G93A mouse model of ALS, stem cells were demonstrated to be neuroprotective, effectively delayed disease onset and extended survival. Despite substantial improvements in stem cell technology and promising results in preclinical studies, several questions still remain unanswered, such as the identification of the most suitable and beneficial cell source, cell dose, route of delivery and therapeutic mechanisms. This review will cover publications in this field and comprehensively discuss advances, challenges and future direction regarding the therapeutic potential of stem cells in ALS, with a focus on mesenchymal stem cells. In summary, given their high proliferation activity, immunomodulation, multi-differentiation potential, and the capacity to secrete neuroprotective factors, adult mesenchymal stem cells represent a promising candidate for clinical translation. However, technical hurdles such as optimal dose, differentiation state, route of administration, and the underlying potential therapeutic mechanisms still need to be assessed.

KEYWORDS:

Adipose derived stem cells; Amyotrophic lateral sclerosis; Neurodegeneration; Stem cell transplantation

PMID:
29132389
PMCID:
PMC5683324
DOI:
10.1186/s13024-017-0227-3
[Indexed for MEDLINE]
Free PMC Article

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