Stem Cell Transplants May Show Promise for MS
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Can Stem Cell Transplants Help Treat MS? continued...
So is it possible that these same stem cell transplants could also benefit these other diseases? Although the possibility exists, Rowitch is noncommittal at this point. “We don’t have data that this could work in MS or other diseases,” he says.
With PMD, the cells that produce myelin are not doing their job. Other diseases involve multiple causes or pathways. If further research in treating PMD pans out, the next step will be to look at MS and other diseases that affect myelin, Rowitch says.
Nancy L. Sicotte, MD, is the director of the Multiple Sclerosis Program at Cedars-Sinai Medical Center in Los Angeles. She says that MS may be more complicated to treat with stem cell therapy.
“With MS, we would be trying to introduce stem cells into an inflamed nervous system,” she says. "To be effective, we have to stop the inflammation process, which we haven’t fully been able to do yet.”
Still, “stem-cell based therapies hold a lot of promise and potential,” Sicotte says. “You always have to temper that with the fact that it takes time to bring a great idea in the lab to humans.”
A Big Deal
A related study by researchers at Oregon Health & Science University's (OHSU) Doernbecher Children’s Hospital in Portland showed that banked brain stem cells can survive and make myelin in mice with symptoms of myelin loss. This work served as one of the building blocks for the study in children with PMD.
This mouse study also gives scientists a glimpse into how these cells behave once they are transplanted, says researcher Stephen A. Back, MD, PhD. He is a clinician-scientist in the Papé Family Pediatric Research Institute at OHSU Doernbecher. “When implanted, they preferentially make myelin-forming cell.”
This is a big deal.
“Stem cells are capable of making new myelin in a brain showing deterioration, and that is very exciting,” he says. “We were surprised to see how well the new myelin was able to form in symptomatic animals.”
The implications are far-reaching. For example, “if we show in a rare disorder like PMD that patients benefit from the transplants, then we will want to do newborn screening to pick up babies with the disorders and get them transplanted as soon as possible,” Back says. “The sooner you get to these kids, the better, [since] the disease can progress like gangbusters once it starts.”