Neural Stem Cells as a Therapeutic Approach for Amyotrophic Lateral Sclerosis

Abstract

Proliferating neural stem cells (NSCs) were first identified in the late 1960s in the adult rat brain1 as multipotent self-renewing stem cells, able to differentiate into neurons, astrocytes, and oligodendrocytes. NSC transplantation is being evaluated to treat traumatic brain or spinal cord injury and neurodegenerative diseases. Amyotrophic lateral sclerosis (ALS) is a fatal neuro­degenerative disorder characterized by progressive loss of upper and lower motor neurons and chronic inflammation leading to paralysis and death due to respiratory failure. In the December issue of Science Translational Medicine, Teng et al.2 reported successful use of transplanted, undifferen­tiated multipotent NSCs to prolong survival in a mouse model of ALS. They found that the experimental treatment is both safe and potentially beneficial to preserve neurons that have been spared by the disease at the time of treatment. The results further indicate the benefits of early intervention and suggest that targeting the spinal cord at different sites will protect both motor and respiratory function. The authors conclude that a combination of therapeutic approaches, from gene therapy to cell transplantation, targeting different pathogenic mechanisms of the disorder, is likely to lead to successful therapy.