Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease

S Fadilah S. Abdul Wahid, Zhe Kang Law, Nor Azimah Ismail, Raymond Azman Ali, Nai Ming Lai

Research output: Contribution to journalReview article

5 Citations (Scopus)

Abstract

Background: Amyotrophic lateral sclerosis (ALS), which is also known as motor neuron disease (MND) is a fatal disease associated with rapidly progressive disability, for which no definitive treatment as yet exists. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Based on data from preclinical studies, cell-based therapy is a promising treatment for ALS/MND. Objectives: To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment. Search methods: On 21 June 2016, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, and Embase. We also searched two clinical trials' registries for ongoing or unpublished studies. Selection criteria: We planned to include randomised controlled trials (RCTs), quasi-RCTs and cluster RCTs that assigned people with ALS/MND to receive cell-based therapy versus a placebo or no additional treatment. Co-interventions were allowable, provided that they were given to each group equally. Data collection and analysis: We followed standard Cochrane methodology. Main results: No studies were eligible for inclusion in the review. We identified four ongoing trials. Authors' conclusions: Currently, there is a lack of high-quality evidence to guide practice on the use of cell-based therapy to treat ALS/MND. We need large, prospective RCTs to establish the efficacy of cellular therapy and to determine patient-, disease- and cell treatment-related factors that may influence the outcome of cell-based therapy. The major goals of future research should be to determine the appropriate cell source, phenotype, dose, and route of delivery, as these will be key elements in designing an optimal cell-based therapy programme for people with ALS/MND. Future research should also explore novel treatment strategies, including combinations of cellular therapy and standard or novel neuroprotective agents, to find the best possible approach to prevent or reverse the neurological deficit in ALS/MND, and to prolong survival in this debilitating and fatal condition.

Original languageEnglish
Article numberCD011742
JournalCochrane Database of Systematic Reviews
Volume2016
Issue number11
DOIs
Publication statusPublished - 8 Nov 2016

Fingerprint

Amyotrophic Lateral Sclerosis
Cell- and Tissue-Based Therapy
Randomized Controlled Trials
Therapeutics
Placebos
Motor Neuron Disease
Neuroprotective Agents
MEDLINE
Patient Selection
Registries
Quality of Life
Clinical Trials
Phenotype
Survival

ASJC Scopus subject areas

  • Medicine(all)
  • Pharmacology (medical)

Cite this

Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease. / S. Abdul Wahid, S Fadilah; Law, Zhe Kang; Ismail, Nor Azimah; Ali, Raymond Azman; Lai, Nai Ming.

In: Cochrane Database of Systematic Reviews, Vol. 2016, No. 11, CD011742, 08.11.2016.

Research output: Contribution to journalReview article

@article{0e1033448eaf44278c0667cfc084c743,
title = "Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease",
abstract = "Background: Amyotrophic lateral sclerosis (ALS), which is also known as motor neuron disease (MND) is a fatal disease associated with rapidly progressive disability, for which no definitive treatment as yet exists. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Based on data from preclinical studies, cell-based therapy is a promising treatment for ALS/MND. Objectives: To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment. Search methods: On 21 June 2016, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, and Embase. We also searched two clinical trials' registries for ongoing or unpublished studies. Selection criteria: We planned to include randomised controlled trials (RCTs), quasi-RCTs and cluster RCTs that assigned people with ALS/MND to receive cell-based therapy versus a placebo or no additional treatment. Co-interventions were allowable, provided that they were given to each group equally. Data collection and analysis: We followed standard Cochrane methodology. Main results: No studies were eligible for inclusion in the review. We identified four ongoing trials. Authors' conclusions: Currently, there is a lack of high-quality evidence to guide practice on the use of cell-based therapy to treat ALS/MND. We need large, prospective RCTs to establish the efficacy of cellular therapy and to determine patient-, disease- and cell treatment-related factors that may influence the outcome of cell-based therapy. The major goals of future research should be to determine the appropriate cell source, phenotype, dose, and route of delivery, as these will be key elements in designing an optimal cell-based therapy programme for people with ALS/MND. Future research should also explore novel treatment strategies, including combinations of cellular therapy and standard or novel neuroprotective agents, to find the best possible approach to prevent or reverse the neurological deficit in ALS/MND, and to prolong survival in this debilitating and fatal condition.",
author = "{S. Abdul Wahid}, {S Fadilah} and Law, {Zhe Kang} and Ismail, {Nor Azimah} and Ali, {Raymond Azman} and Lai, {Nai Ming}",
year = "2016",
month = "11",
day = "8",
doi = "10.1002/14651858.CD011742.pub2",
language = "English",
volume = "2016",
journal = "Cochrane database of systematic reviews (Online)",
issn = "1361-6137",
publisher = "John Wiley and Sons Ltd",
number = "11",

}

TY - JOUR

T1 - Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease

AU - S. Abdul Wahid, S Fadilah

AU - Law, Zhe Kang

AU - Ismail, Nor Azimah

AU - Ali, Raymond Azman

AU - Lai, Nai Ming

PY - 2016/11/8

Y1 - 2016/11/8

N2 - Background: Amyotrophic lateral sclerosis (ALS), which is also known as motor neuron disease (MND) is a fatal disease associated with rapidly progressive disability, for which no definitive treatment as yet exists. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Based on data from preclinical studies, cell-based therapy is a promising treatment for ALS/MND. Objectives: To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment. Search methods: On 21 June 2016, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, and Embase. We also searched two clinical trials' registries for ongoing or unpublished studies. Selection criteria: We planned to include randomised controlled trials (RCTs), quasi-RCTs and cluster RCTs that assigned people with ALS/MND to receive cell-based therapy versus a placebo or no additional treatment. Co-interventions were allowable, provided that they were given to each group equally. Data collection and analysis: We followed standard Cochrane methodology. Main results: No studies were eligible for inclusion in the review. We identified four ongoing trials. Authors' conclusions: Currently, there is a lack of high-quality evidence to guide practice on the use of cell-based therapy to treat ALS/MND. We need large, prospective RCTs to establish the efficacy of cellular therapy and to determine patient-, disease- and cell treatment-related factors that may influence the outcome of cell-based therapy. The major goals of future research should be to determine the appropriate cell source, phenotype, dose, and route of delivery, as these will be key elements in designing an optimal cell-based therapy programme for people with ALS/MND. Future research should also explore novel treatment strategies, including combinations of cellular therapy and standard or novel neuroprotective agents, to find the best possible approach to prevent or reverse the neurological deficit in ALS/MND, and to prolong survival in this debilitating and fatal condition.

AB - Background: Amyotrophic lateral sclerosis (ALS), which is also known as motor neuron disease (MND) is a fatal disease associated with rapidly progressive disability, for which no definitive treatment as yet exists. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Based on data from preclinical studies, cell-based therapy is a promising treatment for ALS/MND. Objectives: To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment. Search methods: On 21 June 2016, we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, and Embase. We also searched two clinical trials' registries for ongoing or unpublished studies. Selection criteria: We planned to include randomised controlled trials (RCTs), quasi-RCTs and cluster RCTs that assigned people with ALS/MND to receive cell-based therapy versus a placebo or no additional treatment. Co-interventions were allowable, provided that they were given to each group equally. Data collection and analysis: We followed standard Cochrane methodology. Main results: No studies were eligible for inclusion in the review. We identified four ongoing trials. Authors' conclusions: Currently, there is a lack of high-quality evidence to guide practice on the use of cell-based therapy to treat ALS/MND. We need large, prospective RCTs to establish the efficacy of cellular therapy and to determine patient-, disease- and cell treatment-related factors that may influence the outcome of cell-based therapy. The major goals of future research should be to determine the appropriate cell source, phenotype, dose, and route of delivery, as these will be key elements in designing an optimal cell-based therapy programme for people with ALS/MND. Future research should also explore novel treatment strategies, including combinations of cellular therapy and standard or novel neuroprotective agents, to find the best possible approach to prevent or reverse the neurological deficit in ALS/MND, and to prolong survival in this debilitating and fatal condition.

UR - http://www.scopus.com/inward/record.url?scp=84997079040&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84997079040&partnerID=8YFLogxK

U2 - 10.1002/14651858.CD011742.pub2

DO - 10.1002/14651858.CD011742.pub2

M3 - Review article

VL - 2016

JO - Cochrane database of systematic reviews (Online)

JF - Cochrane database of systematic reviews (Online)

SN - 1361-6137

IS - 11

M1 - CD011742

ER -