Stem Cells May Lead to New Method to Treat Inherited Dementia

It turns out that there's a new method to help treat an inherited form of dementia. Scientists have attempted to turn stem cells derived from patients into neurons most affected by the disease, and have managed to identify a targetable defect that prevents neurodevelopment.

"Use of induced pluripotent stem cell (iPSC) technology-which involves taking skin cells from patients and reprogramming them into embryonic-like stem cells capable of turning into other specific cell types relevant for studying a particular disease-makes it possible to model dementias that affect people later in life," said Catherine Verfaillie, the senior author of the new study, in a news release.

Forntotemporal disorders are the result of damage to neurons in parts of the brain called the frontal and temporal lobes. This gradually leads to behavioral symptoms or language and emotional disorders. Mutations in a gene called progranulin (GRN) are commonly associated with frontotemporal dementia, but GRN mutations in mice do not mimic all of the features of the human disorder, which has limited progress.

Now, though, iPSC models could potentially be used to better understand dementia and develop new treatments. More specifically, the scientists created iPSCs from three patients carrying the GRN mutation. These immature cells were impaired at turning into mature, specialized cells called cortical neurons, which are the most affected cell type in frontotemporal dementia.

"Our findings suggest that signaling events required for neurodevelopment may also play major roles in neurodegeneration," said Philip Van Damme, one of the researchers. "Targeting such pathways as for instance the Wnt pathway presented in this study, may result in the creation of novel therapeutic approaches for frontotemporal dementia."

The findings are published in the journal Stem Cell Reports.

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