Effect of Stem Cell to Treat Amyotrophic Lateral Sclerosis
Journal Title: The 1st Annual Meeting of Georgian Center for Neuroscience Research - Year 2020, Vol 2, Issue 20
Abstract
Background and objective: Amyotrophic lateral sclerosis (ALS), known as motor neuron disease, is characterized by the degeneration of both upper (the nerve cells in the brain) and lower motor neurons (the brain stem nuclei and ventral roots of the spinal cord), which leads to muscle weakness and eventual paralysis. Currently, there are no effective therapies for the treatment of ALS, except for Riluzole, which has only limited clinical benefits. Stem cellbased therapy as a new treatment strategy for ALS has been extensively studied and has been shown to be somewhat effective. Stem cell therapy aims to produce new motor neurons and to modify pathophysiology which may help stop or slow the progression of the disease in people with ALS. Stem cells possess the ability to self-renew and maintain an undifferentiated state. Several types of stem cells have been studied for treating ALS, including neural stem cells (NSCs), mesenchymal stem cells (MSCs), embryonic stem cells (ESCs) and induced pluripotent stem cells (IPSCs). In this review, we focus on different types of stem cells and their role in the treatment of ALS. Search methods: Google scholar and PubMed databases were searched for the period of 2019 by using the search terms of stem cell and ALS. Findings: Several studies and preclinical models have been shown that cell therapy has immune and anti-inflammatory effects on brain tissue and stem cells have differentiation potential into central nervous cells. However, iPSCs and ESCs can form tumors. iPSCs carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming. Studies of neural stem cells have difficulties in isolation as well as in vitro expansion. MSCs are known as safe cell types that can be easily extracted from various adult tissues (i.e., bone marrow and adipose tissue). For example, some studies showed that MSCs prevented motor neuron death, maintained the number of motor neurons and slowed the progression of ALS by release of immune-modulating factors, such as regulatory T lymphocytes and T helper 2 factors. In addition, after transplantation, no serious adverse effects on neural function have been reported. Also, Super oxide dismutase 1 (SOD1) mutant mice showed that adipose tissue-derived mesenchymal stem cells transplantation could not only have maintained the number of motor neurons but also up-regulated the levels of glial-derived neurotrophic factor (GDNF) and basic fibroblast growth factor (bFGF) in the spinal cord. Conclusion: Riluzole is the only FDA-approved treatment for ALS that has little positive impact on patient survival and function. The results of most clinical trials on other drugs have also been disappointing. Therefore, stem cell transplantation has been investigated as a promising new method for the treatment of ALS. MSCs are potentially effective cell types for ALS treatment.
Authors and Affiliations
Athar Talebi, Nasroallah Moradi-kor
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