"That is proof in principle that putting glial progenitors in a brain like this will at least partially remyelinate the brain, and do so functionally," said James Goldman.
Though this study involved a congenital pediatric disorder, Steven Goldman said his goal is to apply the technique to adult diseases like multiple sclerosis. For now, his team is working to understand why most transplanted animals still die. He suggested this could stem from the seizures that plague shiverer animals, including transplant recipients that have not yet completed remyelination, and said he is exploring the utility of pairing transplants with anticonvulsant therapy to alleviate this problem.
But James Goldman pointed out that before this transplant procedure can be turned into a clinical therapy, several issues must be addressed, not the least of which is the politically sensitive problem of obtaining and using human fetal tissue as a therapeutic agent.
For more on leukodystrophies, visit the U.S. National Library of Medicine.
SOURCES: Steven Goldman, M.D., Ph.D., Dean Zutes Chair, professor, Neurology and Neurosurgery, chief, Division of Cell and Gene Therapy, and co-director, Center for Translational Neuromedicine, University of Rochester Medical Center, Rochester, N.Y.; James E. Goldman, M.D., Ph.D., professor, pathology, and director, Division of Neuropathology, Columbia University College of Physicians and Surgeons, New York City; June 2008, Cell Stem Cell