Concise review: Stem cell therapies for amyotrophic lateral sclerosis: recent advances and prospects for the future.
Amyotrophic lateral sclerosis (ALS) is a lethal disease involving the loss of motor neurons. Although the mechanisms responsible for motor neuron degeneration in ALS remain elusive, the development of stem cell-based therapies for the treatment of ALS has gained widespread support. Here, we review the types of stem cells being considered for therapeutic applications in ALS, and emphasize recent preclinical advances that provide supportive rationale for clinical translation. We also discuss early trials from around the world translating cellular therapies to ALS patients, and offer important considerations for future clinical trial design. Although clinical translation is still in its infancy, and additional insight into the mechanisms underlying therapeutic efficacy and the establishment of long-term safety are required, these studies represent an important first step toward the development of effective cellular therapies for the treatment of ALS.
Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
J Simon
JS
Lunn
Stacey A
SA
Sakowski
Eva L
EL
Feldman
D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D000818:Animals; D035843:Biomedical Research / Q000379:methods* / Q000639:trends; D002454:Cell Differentiation; D005544:Forecasting; D006801:Humans; D008959:Models, Neurological; D009046:Motor Neurons / Q000166:cytology; D033581:Stem Cell Transplantation / Q000379:methods* / Q000639:trends; D013234:Stem Cells / Q000166:cytology*
Cell transplantation; Cellular therapy; Clinical translation; Mesenchymal stem cells; Neural stem cell; Progenitor cells; Stem cell transplantation; Transplantation