Stem cell-derived oligodendrocyte progenitor-based therapy of progressive MS

Project Summary:

Progressive demyelination of axons and the neuron from which they arise accounts for a substantial component of the disability of late-stage progressive MS, both primary and secondary.  Because preclinical studies indicate that demyelinated axons may be remyelinated by the introduction of human oligodendrocyte progenitor cells, this project aims to develop a strategy that can both remyelinate axons before they are lost and to also restore function to already demyelinated brain and spinal cord tissue.

The project will compare oligodendrocyte progenitor cells obtained from three different sources (human induced pluripotential cells, directly induced neural stem cells, and human embryonic stem cells) for their effects upon transplantation into mouse models of chronic demyelination, identifying which is the most effective for treating progressive MS. Unlike most cell therapeutics for MS, which are targeted at non-brain cells to suppress inflammation, this project aims to begin clinical trials of stem cell-derived brain cells transplanted directly into the brain to achieve remyelination and structural repair of already injured, demyelinated brain.