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Society-Funded Study: Cell Transplants Lead to Neurological Recovery in Some Mice Born without Myelin

Society-Funded Study: Cell Transplants Lead to Neurological Recovery in Some Mice Born without Myelin

Researchers funded in part by the National MS Society have shown that human glial progenitor cells – immature myelin-making cells – restored myelin and neurological function when transplanted into the brains of some mice born without normal myelin formation. Martha Windrem, MA, and Society grantee Steven A. Goldman, MD, PhD (University of Rochester, NY) report their findings in the journal

Cell: Stem Cell

(2008 Jun 5;2[6]:553-65).


Multiple sclerosis involves a misguided immune attack against myelin, the insulating coating that surrounds nerve fibers, in the central nervous system. Myelin speeds nerve signal transmission and is also thought to protect the underlying nerve fibers from harm. One of several possibilities being explored for repairing the resulting damage is to transplant replacement myelin-making cells, called glia, which may have the capability to restore myelin. Dr. Goldman’s team and others have conducted previous studies to establish methods for extracting glial progenitors from human brain tissue and transplanting them in attempts to establish normal myelin in rodents.

This Study:

In an attempt to achieve more widespread myelination than in previous studies, the team used a new regimen involving high-density injections of cells into multiple parts of the brain and spinal cord in 26 newborn “shiverer” mice. These mice are born with a defect that prevents myelin formation; they normally die within five months and experience profound neurological symptoms including tremors, weakness and seizures. To avoid the complications of cell rejection, the team used shiverer mice specially bred to be deficient in mounting immune attacks.

Most of the mice died around the same time as control mice that were not treated. However, in six mice, the transplanted cells multiplied, migrated to widespread areas of the brain and spinal cord, and formed normal, functional myelin. This caused progressive improvement in neurological symptoms. Four mice survived more than one year, and by then were free of neurological symptoms.

“To our knowledge, these data represent the first outright rescue of a congenital hypomyelinating disorder,” write the authors. Despite the differences between MS and the myelin defect in this mouse model – in MS, the damage to myelin is the result of the immune attack, not an inborn lack of myelin – these results represent a step forward in developing cell transplants as a potential MS treatment strategy. The authors caution, however, that much further research is necessary to fully understand these results before they can be applied to human disease.

The National MS Society’s grant to Dr. Goldman’s team was funded by gifts from the Charles and Margery Barancik Foundation and the Alan Buegeleisen Research Fund. In addition, Dr. Goldman is one of over 50 investigators around the world who are collaborating in a National MS Society-supported targeted initiative to find ways to repair and protect the nervous system in MS. Read more about this and other efforts torestore functionin people with MS.