Scientists Announce Breakthrough In Alzheimer's Disease Treatment

SAN FRANCISCO, CA — Scientists in San Francisco have erased damage in human brain cells caused by a gene they describe as the primary genetic risk factor for Alzheimer’s disease.

Researchers at Gladstone Institutes — affiliated with the University of California, San Francisco — discovered that a gene called apoE4 is the primary genetic risk factor for Alzheimer’s. They report that having a single copy of the gene doubles a person’s chance of developing Alzheimer’s, while having two copies increases the risk by twelve-fold.

Once they discovered the role of apoE4 in the development of Alzheimer's, the scientists changed the gene by adding a small molecule. Not only did that make it harmless, it eliminated the signs of Alzheimer’s disease, restored normal function to the cells, and improved cell survival.

Gladstone reports that it switched to using human brain cells for research because of the poor track record of using mice.

"Drug development for Alzheimer’s disease has been largely a disappointment over the past 10 years," says lead author Yadong Huang, MD, PhD, a senior investigator and director of the Center for Translational Advancement at Gladstone. "Many drugs work beautifully in a mouse model, but so far they’ve all failed in clinical trials. One concern within the field has been how poorly these mouse models really mimic human disease."

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Using stem cell technology, the research team created neurons from skin cells donated by Alzheimer’s patients with two copies of the apoE4 gene, as well as from healthy individuals who had two copies of the apoE3 gene.

The scientists confirmed that apoE4 protein cannot function properly and is broken down into disease-causing fragments in the cells. This process results in a number of problems commonly found in Alzheimer’s disease, including the accumulation of the protein tau and of amyloid peptides.

"There’s an important species difference in the effect of apoE4 on amyloid beta,' says Chengzhong Wang, PhD, the first author on the paper and former research scientist at Gladstone. "Increased amyloid beta production is not seen in mouse neurons and could potentially explain some of the discrepancies between mice and humans regarding drug efficacy. This will be very important information for future drug development."

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The researchers are now collaborating with fellow scientists at universities and in the pharmaceutical industry so that their discovery can be tested in human patients in the future.

The research is published in the medical journal Nature Medicine. It was funded by the National Institute on Aging, the California Institute of Regenerative Medicine, and the Roddenberry Foundation.

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