Using genetically modified stem cells for treating Alzheimer’s disease

Dr. Mathew Blurton-Jones of U.C. Irvine: his new study may help open new approaches to Alzheimer’s

New work from UC Irvine using genetically modified stem cells to treat Alzheimer’s disease offers some encouraging early signs.

Alzheimer’s involves a buildup of plaques in the brain formed by excess amyloid‑beta protein. Researchers still don’t know whether this buildup causes the disease or results from it. But if it is a cause, then reducing the plaques could slow or even stop the disease.

Neprilysin’s role

This is where an enzyme called neprilysin plays a role. Neprilysin breaks down amyloid‑beta. Dr. Mathew Blurton‑Jones explains:

“Studies suggest that neprilysin decreases with age and may therefore influence Alzheimer’s risk. If amyloid accumulation drives the disease, then therapies that reduce amyloid‑beta or boost its breakdown could help, especially if started early.”

The UC Irvine team took neural stem cells and genetically modified them to produce 25 times more neprilysin than normal cells. They then injected these cells into two types of mice with Alzheimer’s, targeting the hippocampus and the subiculum—the regions hit hardest by the disease.

The results, published in Stem Cells Research and Therapy, were promising. Mice that received the modified stem cells had far fewer amyloid‑beta plaques. The effect lasted at least a month after the transplant.

Mouse Models

Results in mice don’t always translate to humans, but Dr. Blurton‑Jones notes the value of seeing the same outcome in two different Alzheimer’s models:

“Every mouse model of Alzheimer’s is different. By studying the question in two independent models, we can be more confident that the results are meaningful and broadly applicable.”

Researchers still need to determine whether this approach improves brain function. But these findings suggest a possible new way to reduce plaque buildup and maybe slow the disease.

We have invested almost $50 million in more than a dozen research projects aimed at developing new treatments—and ultimately a cure—for Alzheimer’s.

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