Abstract
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease of motor neurons. While the genetics behind familial as well as sporadic ALS are being increasingly uncovered, pathophysiology remains incompletely understood and there are no effective treatment options.
However, as preclinical results provided some rationale for the use of stem cells as support cells for the dying motor neurons, stem cells are being considered as a potential treatment strategy. Based on the preclinical models, translational human trials have been carried out using various types of stem cells, as well as a range of cell delivery methods.
To date, no trial has demonstrated a clear therapeutic benefit. Here, we provide a critical review of current clinical trials using either mesenchymal or neural stem cells to treat ALS patients.
In order to provide robust assessment of the efficacy of stem cells, it will be essential to standardize administration protocols, identify the most suitable cell type as well as to validate more reliable biomarkers of disease progression in longitudinal clinical studies.