Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells

Background/Objectives: <i>Amyotrophic lateral sclerosis</i> (ALS), or Lou Gehrig’s disease, is a debilitating, incurable neurodegenerative disorder characterised by motor neuron death in the spinal cord, brainstem, and motor cortex. With an incidence rate of about 4.42 cases per 100,000...

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Main Authors: Lauren Frawley, Noam Tomer Taylor, Olivia Sivills, Ella McPhillamy, Timothy Duy To, Yibo Wu, Beek Yoke Chin, Chiew Yen Wong
Format: Article
Language:English
Published: MDPI AG 2024-12-01
Series:Biomedicines
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Online Access:https://www.mdpi.com/2227-9059/13/1/35
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author Lauren Frawley
Noam Tomer Taylor
Olivia Sivills
Ella McPhillamy
Timothy Duy To
Yibo Wu
Beek Yoke Chin
Chiew Yen Wong
author_facet Lauren Frawley
Noam Tomer Taylor
Olivia Sivills
Ella McPhillamy
Timothy Duy To
Yibo Wu
Beek Yoke Chin
Chiew Yen Wong
author_sort Lauren Frawley
collection DOAJ
description Background/Objectives: <i>Amyotrophic lateral sclerosis</i> (ALS), or Lou Gehrig’s disease, is a debilitating, incurable neurodegenerative disorder characterised by motor neuron death in the spinal cord, brainstem, and motor cortex. With an incidence rate of about 4.42 cases per 100,000 people annually, ALS severely impacts motor function and quality of life, causing progressive muscle atrophy, spasticity, paralysis, and eventually death. The cause of ALS is largely unknown, with 90% of cases being sporadic and 10% familial. Current research targets molecular mechanisms of inflammation, excitotoxicity, aggregation-prone proteins, and proteinopathy. Methods: This review evaluates the efficacy of three stem cell types in ALS treatment: mesenchymal stem cells (MSCs), neural stem cells (NSCs), and induced pluripotent stem cells (iPSCs). Results: MSCs, derived from various tissues, show neuroprotective and regenerative qualities, with clinical trials suggesting potential benefits but limited by small sample sizes and non-randomised designs. NSCs, isolated from the fetal spinal cord or brain, demonstrate promise in animal models but face functional integration and ethical challenges. iPSCs, created by reprogramming patient-specific somatic cells, offer a novel approach by potentially replacing or supporting neurons. iPSC therapy addresses ethical issues related to embryonic stem cells but encounters challenges regarding genotoxicity and epigenetic irregularities, somatic cell sources, privacy concerns, the need for extensive clinical trials, and high reprogramming costs. Conclusions: This research is significant for advancing ALS treatment beyond symptomatic relief and modest survival extensions to actively modifying disease progression and improving patient outcomes. Successful stem cell therapies could lead to new ALS treatments, slowing motor function loss and reducing symptom severity.
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spelling doaj-art-607d51257cb24e68a74373afbea5618d2025-01-24T13:23:47ZengMDPI AGBiomedicines2227-90592024-12-011313510.3390/biomedicines13010035Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem CellsLauren Frawley0Noam Tomer Taylor1Olivia Sivills2Ella McPhillamy3Timothy Duy To4Yibo Wu5Beek Yoke Chin6Chiew Yen Wong7School of Medical, Indigenous and Health Sciences, University of Wollongong, Wollongong 2500, AustraliaSchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney 2052, AustraliaSchool of Medical, Indigenous and Health Sciences, University of Wollongong, Wollongong 2500, AustraliaSchool of Medical, Indigenous and Health Sciences, University of Wollongong, Wollongong 2500, AustraliaSchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney 2052, AustraliaSchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney 2052, AustraliaSchool of Health Sciences, IMU University, Kuala Lumpur 57000, MalaysiaSchool of Health Sciences, IMU University, Kuala Lumpur 57000, MalaysiaBackground/Objectives: <i>Amyotrophic lateral sclerosis</i> (ALS), or Lou Gehrig’s disease, is a debilitating, incurable neurodegenerative disorder characterised by motor neuron death in the spinal cord, brainstem, and motor cortex. With an incidence rate of about 4.42 cases per 100,000 people annually, ALS severely impacts motor function and quality of life, causing progressive muscle atrophy, spasticity, paralysis, and eventually death. The cause of ALS is largely unknown, with 90% of cases being sporadic and 10% familial. Current research targets molecular mechanisms of inflammation, excitotoxicity, aggregation-prone proteins, and proteinopathy. Methods: This review evaluates the efficacy of three stem cell types in ALS treatment: mesenchymal stem cells (MSCs), neural stem cells (NSCs), and induced pluripotent stem cells (iPSCs). Results: MSCs, derived from various tissues, show neuroprotective and regenerative qualities, with clinical trials suggesting potential benefits but limited by small sample sizes and non-randomised designs. NSCs, isolated from the fetal spinal cord or brain, demonstrate promise in animal models but face functional integration and ethical challenges. iPSCs, created by reprogramming patient-specific somatic cells, offer a novel approach by potentially replacing or supporting neurons. iPSC therapy addresses ethical issues related to embryonic stem cells but encounters challenges regarding genotoxicity and epigenetic irregularities, somatic cell sources, privacy concerns, the need for extensive clinical trials, and high reprogramming costs. Conclusions: This research is significant for advancing ALS treatment beyond symptomatic relief and modest survival extensions to actively modifying disease progression and improving patient outcomes. Successful stem cell therapies could lead to new ALS treatments, slowing motor function loss and reducing symptom severity.https://www.mdpi.com/2227-9059/13/1/35amyotrophic lateral sclerosis (ALS)induced pluripotent stem cells (iPSCs)mesenchymal stem cells (MSCs)neural stem cells (NSCs)regenerative medicinestem cell therapy (SCT)
spellingShingle Lauren Frawley
Noam Tomer Taylor
Olivia Sivills
Ella McPhillamy
Timothy Duy To
Yibo Wu
Beek Yoke Chin
Chiew Yen Wong
Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
Biomedicines
amyotrophic lateral sclerosis (ALS)
induced pluripotent stem cells (iPSCs)
mesenchymal stem cells (MSCs)
neural stem cells (NSCs)
regenerative medicine
stem cell therapy (SCT)
title Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
title_full Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
title_fullStr Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
title_full_unstemmed Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
title_short Stem Cell Therapy for the Treatment of Amyotrophic Lateral Sclerosis: Comparison of the Efficacy of Mesenchymal Stem Cells, Neural Stem Cells, and Induced Pluripotent Stem Cells
title_sort stem cell therapy for the treatment of amyotrophic lateral sclerosis comparison of the efficacy of mesenchymal stem cells neural stem cells and induced pluripotent stem cells
topic amyotrophic lateral sclerosis (ALS)
induced pluripotent stem cells (iPSCs)
mesenchymal stem cells (MSCs)
neural stem cells (NSCs)
regenerative medicine
stem cell therapy (SCT)
url https://www.mdpi.com/2227-9059/13/1/35
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