Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease (ALS/MND)

Review question

How effective and safe is cell-based therapy in people with ALS/MND, when we compare it with an inactive treatment or no treatment?


ALS/MND is a condition in which nerves in the brain and spinal cord that control movement (motor neurons) stop working. A person with ALS/MND has difficulty moving, swallowing, chewing and speaking, which become worse over time. Half of people with ALS/MND die within three years of their first symptoms. Weakness of muscles used in breathing often leads to death. The condition currently has no cure. Current treatment regimens largely focus on relieving symptoms to improve the quality of life of those affected. Cell-based therapy can be defined as injection of cellular material into a person to treat disease. Based on early studies, cell-based therapy is a promising new treatment. Various types of cell-based therapies can be used in ALS/MND, including stem cell therapy. Stem cell therapy aims to provide new motor neurons, which may help stop or slow down disease progression in people with ALS.

Study characteristics

We searched multiple databases for clinical trials.

Key results and quality of the evidence

We did not find any completed randomised controlled trials that assessed the effects of cell-based therapy. Four trials are in progress. As early studies are promising, we urgently need large, well-designed clinical trials to establish whether cell-based therapies have clinical benefit in ALS/MND. Major goals of future research are to identify the right type and amount of cells to use, and how best to administer them.

The evidence is up to date as of 21 June 2016.

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.

Read the full abstract...

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.


To assess the effects of cell-based therapy for people with ALS/MND, compared with placebo or no additional treatment.

Search strategy: 

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.