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Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials

Stem cell-based therapies (SCT) to treat neurodegenerative disorders have promise but clinical trials have only recently begun, and results are not expected for several years. While most SCTs largely lead to a symptomatic therapeutic effect by replacing lost cell types, there may also be disease-mod...

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Detalles Bibliográficos
Autores principales: Marsili, Luca, Sharma, Jennifer, Outeiro, Tiago Fleming, Colosimo, Carlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9953050/
https://www.ncbi.nlm.nih.gov/pubmed/36831041
http://dx.doi.org/10.3390/biomedicines11020505
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author Marsili, Luca
Sharma, Jennifer
Outeiro, Tiago Fleming
Colosimo, Carlo
author_facet Marsili, Luca
Sharma, Jennifer
Outeiro, Tiago Fleming
Colosimo, Carlo
author_sort Marsili, Luca
collection PubMed
description Stem cell-based therapies (SCT) to treat neurodegenerative disorders have promise but clinical trials have only recently begun, and results are not expected for several years. While most SCTs largely lead to a symptomatic therapeutic effect by replacing lost cell types, there may also be disease-modifying therapeutic effects. In fact, SCT may complement a multi-drug, subtype-specific therapeutic approach, consistent with the idea of precision medicine, which matches molecular therapies to biological subtypes of disease. In this narrative review, we examine published and ongoing trials in SCT in Parkinson’s Disease, atypical parkinsonian disorders, Huntington’s disease, amyotrophic lateral sclerosis, and spinocerebellar ataxia in humans. We discuss the benefits and pitfalls of using this treatment approach within the spectrum of disease-modification efforts in neurodegenerative diseases. SCT may hold greater promise in the treatment of neurodegenerative disorders, but much research is required to determine the feasibility, safety, and efficacy of these complementary aims of therapeutic efforts.
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spelling pubmed-99530502023-02-25 Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials Marsili, Luca Sharma, Jennifer Outeiro, Tiago Fleming Colosimo, Carlo Biomedicines Review Stem cell-based therapies (SCT) to treat neurodegenerative disorders have promise but clinical trials have only recently begun, and results are not expected for several years. While most SCTs largely lead to a symptomatic therapeutic effect by replacing lost cell types, there may also be disease-modifying therapeutic effects. In fact, SCT may complement a multi-drug, subtype-specific therapeutic approach, consistent with the idea of precision medicine, which matches molecular therapies to biological subtypes of disease. In this narrative review, we examine published and ongoing trials in SCT in Parkinson’s Disease, atypical parkinsonian disorders, Huntington’s disease, amyotrophic lateral sclerosis, and spinocerebellar ataxia in humans. We discuss the benefits and pitfalls of using this treatment approach within the spectrum of disease-modification efforts in neurodegenerative diseases. SCT may hold greater promise in the treatment of neurodegenerative disorders, but much research is required to determine the feasibility, safety, and efficacy of these complementary aims of therapeutic efforts. MDPI 2023-02-09 /pmc/articles/PMC9953050/ /pubmed/36831041 http://dx.doi.org/10.3390/biomedicines11020505 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Marsili, Luca
Sharma, Jennifer
Outeiro, Tiago Fleming
Colosimo, Carlo
Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title_full Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title_fullStr Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title_full_unstemmed Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title_short Stem Cell Therapies in Movement Disorders: Lessons from Clinical Trials
title_sort stem cell therapies in movement disorders: lessons from clinical trials
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9953050/
https://www.ncbi.nlm.nih.gov/pubmed/36831041
http://dx.doi.org/10.3390/biomedicines11020505
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