Using human brain cells, Goldman's team was able to restore proper nerve function in nearly the entire brains of mice much more efficiently than has been done previously. While the work is years away from a clinical study in humans, it serves as a milestone for researchers seeking to use stem cells and related cells known as progenitors to treat human disease.
"The results are much better than we expected," says Goldman, who is professor of Neurology and chief of the department's Division of Cell and Gene Therapy at Rochester. "The percentage of cells in this experiment that began producing myelin is extraordinary, probably thousands of times as many as in previous experiments."
The work has implications for a wide variety of children's diseases known as pediatric leukodystrophies, where the myelin is damaged or doesn't work correctly, such as Canavan disease, Krabbe disease, or Tay-Sachs disease.
"While these diseases are generally rare, there are a lot of them, and together they affect thousands of children and their families," says Goldman. "We've got a long ways to go, but we're optimistic that these findings could make a difference in the lives of these patients."
The work focuses on myelin, the fatty substance that covers nearly all the nerve cells in our bodies like insulation wrapped around a wire and helps signals in the nervous system go from one point to another. When the myelin breaks down, as in multiple sclerosis or the le
Contact: Tom Rickey
University of Rochester Medical Center