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UB, Rochester Scientists Isolate Stem Cells That Could Treat MS

Fraser Sim, PhD, in his laboratory

Fraser Sim, PhD

Published October 20, 2011

UB and University of Rochester researchers have discovered a precise way to isolate stem cells that make myelin, a breakthrough that could pave the way for clinical trials using stem cells to treat multiple sclerosis and other demyelinating diseases.

“The use of the new techniques described in this work will permit us to better understand how human cells behave in the brain and help us predict which medications may be successful in the treatment of myelin loss.”
Fraser Sim, PhD
Assistant professor of pharmacology and toxicology

Progenitor Cell Study Marks First Step to Human Trials

Until now, researchers had difficulty separating out the relevant progenitor cells—those that develop into myelin-producing cells. The body's inability to make myelin, a fatty acid that coats neurons and allows them to signal effectively, causes demyelinating diseases.

“You need to have the right cells in hand before you can even think about getting to a clinical trial to treat people,” says Fraser Sim, PhD, assistant professor of Pharmacology and Toxicology, first author on the study.

“This is a significant step.”

Stem Cells Produce Fourfold Increase of Myelin in Mice

Sim and University of Rochester graduate student Crystal McClain looked at gene activity in different type of stem cells, concluding that stem cells carrying a protein known as CD140a would most likely become oligodendrocytes, the brain cells that make myelin.

The researchers injected the cells into the brains of mice 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 neurons with myelin.

This marked a fourfold improvement over the team’s previously published results.

“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,” says study co-author Steven Goldman, MD, PhD, professor and chair of the Department of Neurology at the University of Rochester Medical Center.

“They are our best candidates right now for someday helping patients with MS or children with fatal hereditary myelin disorders.” 

The results of the current study are published in Nature Biotechnology in an article titled CD140a identifies a population of highly myelinogenic, migration-competent and efficiently engrafting human oligodendrocyte progenitor cells.

Stem Cell Injections Could One Day Treat MS Patients

Eventual treatment of diseases such as MS could involve injecting stem cells to create myelin in the brain, Sim says.

“Another approach might involve using certain medications to turn on these cells already present in the brains of patients and thereby create new myelin,” he adds.

“The use of the new techniques described in this work will permit us to better understand how human cells behave in the brain and help us predict which medications may be successful in the treatment of myelin loss.”

The new approach may be applicable to Krabbe’s Disease, which also involves myelin breakdown. Sim is collaborating on related work with researchers at the Hunter James Kelly Research Institute, a partnership between UB and the Hunter’s Hope Foundation, located in UB’s New York State Center of Excellence in Bioinformatics and Life Sciences.