Brain Stem Cells Reverse Myelin Deficiency in Mice

June 6 -- THURSDAY, June 5 (HealthDay News) -- Researchers report they have used neural stem cells to correct a congenital brain disorder in mice.

Dr. Steven Goldman, of the University of Rochester Medical Center in New York, and his colleagues used a type of neural stem cell called "glial progenitor cells" (GPCs), derived from human fetuses, to correct both behavioral and physiological abnormalities in a mouse model of a myelin-deficiency disorder.

The study represents "a very important advance," said Dr. James Goldman, an investigator in the Columbia Neural Stem Cell Program at Columbia University Medical Center, who was not involved in the study.

Though Steven Goldman and others previously had shown that injection of GPCs into mouse brains could lead to remyelination of demyelinated neurons, that observation did not include any change in disease progression.

"The fact that they were able to get at least some of these animals to survive, and show that physiologically and behaviorally they are doing well, is an advance," said James Goldman.

The findings were reported in the June issue of Cell Stem Cell.

Myelin is a structure, comprised of protein and fat, that envelops long neuronal fibers called axons. Axons are the conduits for neural impulses, both conscious and unconscious. Just as electrical cable must be insulated to prevent signal loss over distance, myelin ensures that nerve impulses can traverse long axonal processes in the central nervous system without degrading.

Myelin is formed by neural support cells called oligodendrocytes, which are derived from GPCs. Disorders that arise from the absence or degradation of myelin represent a "substantial proportion of adult neurological diseases," said Steven Goldman. They run the gamut, from autoimmune disorders like muscular dystrophy, to lysosomal storage diseases like Tay-Sachs, to congenital defects like Pelizaeus-Merzbacher Disease, an X-linked condition where myelin doesn't form.

In this study, Goldman and his team used "shiverer" mice, whose congenital lack of myelin basic protein causes them to shake and seize uncontrollably, giving them their name. They typically die by 5 months of age.