Combining growth factor and stem cell therapy for amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease where motor neurons within the brain and spinal cord are lost, leading to paralysis and death. Certain growth factors should, in principle, be able to protect dying motor neurons. However, targeted delivery to the spinal cord or brain has been a constant problem. There is also accumulating evidence that glial cells might play a crucial role in maintaining motor neuron function and survival in ALS. Stem cells isolated and expanded in culture can be modified to release growth factors and generate glial cells following transplantation into the spinal cord or brain. As such, they might be able to both detoxify the local environment around dying motor neurons and deliver trophic factors. Here we examine the feasibility of translating these findings into new treatments for ALS patients.