Precision with Stem Cells a Step Forward for Treating Multiple Sclerosis

Scientists have improved upon their own previous world-best efforts to pluck out just the right stem cells to address the brain problem at the core of multiple sclerosis and a large number of rare, fatal children’s diseases.

Details of how scientists isolated and directed stem cells from the human brain to become oligodendrocytes – the type of brain cell that makes myelin, a crucial fatty material that coats neurons and allows them to signal effectively – were published online and in the October issue of Nature Biotechnology by scientists at the University of Rochester Medical Center and the University at Buffalo.

Scientists injected the cells into the brains of mice that were born without the ability to make myelin. Twelve weeks later, the cells had become oligodendrocytes and had coated more than 40 percent of the brain’s neurons with myelin – a four-fold improvement over the team’s previous results published in Cell Stem Cell and Nature Medicine.

“These cells are our best candidates right now for someday helping patients with M.S., or children with fatal hereditary myelin disorders,” said Steven Goldman, M.D., Ph.D., the leader of the team and professor and chair of the Department of Neurology at the University of Rochester Medical Center. “These cells migrate more effectively throughout the brain, and they myelinate other cells more quickly and more efficiently than any other cells assessed thus far. Now we finally have a cell type that we think is safe and effective enough to propose for clinical trials.”

The first author and co-corresponding author of the paper is Fraser Sim, Ph.D., assistant professor of Pharmacology and Toxicology at the University at Buffalo, who did much of the work while he was a researcher at Rochester.

Sim and Rochester graduate student Crystal McClain ran extensive analyses looking at gene activity in different types of stem cells, leading to the conclusion that stem cells carrying a protein known as CD140a on their surface seemed to be most likely to become the desired cells – oligodendrocytes.

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