WEDNESDAY, June 4 (HealthDay News) -- Mice with a congenital brain disorder improved after receiving human neural stem cell transplants, a U.S. study finds.
The mice lacked myelin, a substance that plays a critical role in the transmission of electrical signals between nerve cells. When myelin is missing or damaged, electrical signals aren't properly transmitted. These "shiverer" mice typically die within months of birth.
Demyelination also occurs in people with multiple sclerosis.
Previous research has examined the use of cell transplantation for restoring absent or lost myelin to diseased nerve fibers. But, until now, no transplantation of human neural stem cells or of their derivatives (glial progenitor cells) had been successful in test animals.
In this new study, researchers from the University of Rochester Medical Center and a number of other universities (Cornell, UCLA and Baylor) created a new method for harvesting and purification of human fetal glial progenitor cells.
They also developed a new cell delivery strategy that uses multiple injection sites to encourage widespread and dense take-up of the transplanted cells through the central nervous system.
When the researchers used these new approaches, the transplanted cells took hold throughout the brain and spinal cord, and the mice showed robust, efficient and functional myelination. Some of the mice showed neurological improvement and a fraction of them were save by the procedure.
"The neurological recovery and survival of the mice receiving transplants was in sharp contrast to the fate of their untreated controls, which uniformly died by five months," researcher Dr. Steve Goldman, of the departments of neurology and neurosurgery at the University of Rochester Medical Center, said in a prepared statement.
"To our knowledge, these data represent the first outright rescue of a congenital hypomyelinating disorder by means of stem or progenitor cell transplantation," Goldman said. "Although much work needs to be done to maximize the number of individuals that respond to transplantation, I think that these findings hold great promise for the potential of stem cell-based treatment in a wide range of hereditary and ischemic myelin disorders in both children and adults."
The study was published in the June issue of Cell Stem Cell.
The Multiple Sclerosis International Federation has more about demyelination.