Genetically modified stem cells offer potential new path to treating Alzheimer’s disease

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

The Holy Grail of medical research is to find a cure for deadly diseases. But in the case of diseases like Alzheimer’s, where we don’t even have any truly effective treatments, any research that offers the potential of a new approach to slowing the progression of the disease has to be considered an advance. That’s why research from the University of California at Irvine is encouraging news indeed.

One of the characteristics of Alzheimer’s is a build-up of plaques in the brain caused by too much of the protein amyloid-beta. It’s not known if that build-up is a cause or an effect of the disease but if it is a cause then, in theory at least, reducing that build-up could help stop or slow the progression of Alzheimer’s.

This is where an enzyme called neprilysin comes in. Neprilysin helps break down the accumulation of amyloid-beta. As researcher Dr. Mathew Blurton-Jones says in a news release:

"Studies suggest that neprilysin decreases with age and may therefore influence the risk of Alzheimer's disease. If amyloid accumulation is the driving cause of Alzheimer's disease, then therapies that either decrease amyloid-beta production or increase its degradation could be beneficial, especially if they are started early enough." 

So the researchers at U.C. Irvine took some neural or brain stem cells and genetically modified them so that they would produce 25 times more neprilysin than ordinary neural stem cells. They then injected them into two different kinds of mice that had forms of Alzheimer’s, targeting the areas that are most affected by the disease, the hippocampus and the subiculum.

The results, published in the journal Stem Cells Research and Therapy, showed some promising results. The mice given the genetically modified stem cells were found to have significantly reduced amounts of the amyloid-beta plaques in their brain. This result continued for at least one month after the transplant.

Now a lot of encouraging results in mice haven’t panned out in humans, but Dr. Blurton-Jones points out the importance of seeing this result in mice with two different forms of Alzheimer’s:

"Every mouse model of Alzheimer's disease is different and develops varying amounts, distribution, and types of amyloid-beta pathology. By studying the same question in two independent transgenic models, we can increase our confidence that these results are meaningful and broadly applicable to Alzheimer's disease."

 It still remains to be seen whether this approach will improve the functioning of the brain, that’s research that still has to be done, but it does suggest that it might offer a new approach to reducing the build-up of plaques in the brain, and so possibly slow the progression of the disease.

We have awarded almost $50 million in funding for more than a dozen different research projects focusing on finding ways to develop new treatments and even a cure for Alzheimer’s.

kevin mccormack