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Stem cell therapy investigated as possible treatment for PMD

 

Narrator:       This is Science Today.   Pelizaeus-Merzbacher disease, or PMD, is a rare congenital disorder in which brain cells called oligodendrocytes don't make a substance called myelin that is required for proper neuron function. In a landmark clinical trial, researchers at the University of California, San Francisco, are investigating stem cell therapy as a possible treatment for PMD. David Rowitch, a professor of pediatrics and neurosurgery, is leading the study.

Rowitch:         This is a disease in which there is a global lack of myelin. And in that background it is theoretically easier to detect a small pool of cells that may be producing myelin. That's one of the reasons to think about PMD as a proof of concept of neural stem cell therapy because we have a very precise way of measuring the impact of cells that might only be affecting a very small part of the brain, but yet we can still measure it and say these cells have been grafted, they are functioning, and so it could have implications for our understanding of how neural stem cells might be able to function in other types of diseases.

Narrator:       For Science Today, I'm Larissa Branin.