New research shows the potential regenerative power of hematopoietic stem cells in ALS and may be a feasible treatment approach in the future.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a neurological disease characterized by the progressive destruction of motor neurons, leading to disability and later death. There is no cure for the disease, and the only FDA-approved treatment (riluzole) adds only an additional few months of life to patients with ALS. Many factors are associated with the development of ALS, yet the exact causes of ALS development remain poorly understood.
Emerging research data from animal and clinical studies indicate that damage of the blood-brain and blood-spinal cord barriers may be an important mechanism in ALS development. Vascular changes may be the main factors that compromise the blood-central nervous system (CNS) barriers and have been seen to occur in mouse models prior to the development of motor neuron damage. Impairment of the blood-CNS barriers may lead to the entry of substances from the circulation that are harmful to the CNS.
Given the possible role of blood-CNS barrier damage in the pathogenesis of ALS, a means to replace the damaged endothelial cells may be a viable treatment approach for ALS. Research scientists from the University of South Florida conducted studies in symptomatic mouse models of ALS using CD34+ (hematopoietic) stem cells. The main goal was to determine the effects of stem cells on motor neuron survival, motor function, and cell differentiation. Different doses of stem cells were transplanted intravenously into symptomatic mice and the effects evaluated.
Stem cell-treated mice had higher body weights over time compared to controls. They also showed the best performance results in tests of functional ability. The transplanted stem cells differentiated and attached to spinal cord capillary walls. There was a significant decrease in astrocyte reactivity and rates of astrocytosis (abnormal increase in astrocytes due to nearby neuron destruction) in mice transplanted with stem cells. Greater spinal cord neuron survival was also observed in mice that received higher doses of stem cells. These results show the potential regenerative power of hematopoietic stem cells in ALS mice models and may represent a feasible treatment approach for patients with ALS.
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Reference:Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier. (2018). Medicalxpress.com. Retrieved 23 April 2018, from https://medicalxpress.com/news/2017-05-stem-cell-transplants-advance-als.html