Alzheimer's Disease Damage Completely Erased in Human Cells by Changing Structure of One Protein

Alzheimer's disease is a degenerative condition that largely affects the elderly. A carer holds the hand of an elderly patient. Pascal Lachenaud/AFP/Getty Images

Updated |Scientists in California successfully changed a protein associated with Alzheimer's disease into a more harmless form, allowing them to erase brain cell damage. The breakthrough is especially important because the research was not conducted on mice but rather human cells.

Researchers from the Gladstone Institutes in San Francisco took stem cells from Alzheimer's patients who had two copies of the apoE4 gene as well as stem cells from healthy volunteers who also had a pair of the apoE3 gene, a study published online Tuesday said. They then created neurons, or brain cells, from these stem cells.

Through studying these neurons, the team was able to identify exactly why apoE4 puts individuals at such increased risk for Alzheimer's disease. For example, having one apoE4 gene more than doubles the chances of developing Alzheimer's disease, and possessing two copies of the gene increases risk of developing the neurodegenerative brain disease by 12-fold, a statement on the study reported.

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Now we know this is because the apoE4 gene, which creates the apoE4 protein, increases the production of the amyloid beta protein. This finding surprised the researchers, as the presence of the apoE4 gene in mouse models did not increase amyloid beta production.

"What's important from this study is that we used human cells," Yadong Huang, senior investigator at Gladstone and lead author of the study told Newsweek. "The many previous studies were performed on animal models or animal cells."

Increased amyloid beta production is significant because in some cases, these proteins can clump together and form plaques. These plaques can disrupt neuron messaging and in turn cause many of the symptoms associated with Alzheimer's disease, such as cognitive problems.

Once the team confirmed that the presence of apoE4 promoted Alzheimer's disease, they then worked on a way to prevent this. They developed a way to change the structure of the apoE4 protein in order to make it harmless. This erased any damage of Alzheimer's disease and helped increase brain cell survival in the human cell model.

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The results are promising, especially since they were seen in human cells and not an animal model. Still, the research is not quite a cure, at least not yet. The results will have to be repeated in human patients. The researchers are now working to translate this finding into a compound that can be used on an industrial level so that eventual human trials will be possible.

"It's hard to predict how long it would take but we are working very hard right now to narrow it down from many small molecule candidates to finally focus on one or two and then hopefully move to the clinical trials," said Huang.

Correction: This article has been corrected to state that the healthy volunteers had a pair of the apoE3 gene.

Update: This article has been updated with additional quotes from Yadong Huang.