Scientists were able to successfully remove a gene that caused Alzheimer's disease from the human brain, possibly paving the way for a new kind of treatment against the dreaded illness.
There have been numerous proposals on a possible treatment or cure for Alzheimer's disease, including an enzyme inhibitor and frontal lobe stimulation. This one is different though, primarily because the tests were carried out not on laboratory mice, but on humans.
Scientists Erase Alzheimer's Disease Gene From Human Brain
Scientists from the Gladstone Institutes, a San Francisco-based independent biomedical research institution, were able to remove the primary genetic risk factor for Alzheimer's disease, which is a gene named apoE4.
In their findings, published in the Nature Medicine journal, the researchers found that human brains that possess even one copy of apoE4 are over twice as likely to eventually develop Alzheimer's disease within their lifetime. In addition, brains that have two copies of the gene carry an increased risk by 12 times.
The researchers created neurons from the skin cells of Alzheimer's disease patients that have two copies of the apoE4 gene, and of healthy individuals that have two copies of the apoE3 gene. The scientists discovered that the apoE4 protein is not able to properly function in human neurons, and is broken down into fragments.
This leads to several problems that are commonly found among victims of Alzheimer's disease, including the formation of beta-amyloid protein. Excessive beta-amyloids may clump together to create plaques, which disrupt neurons and result in symptoms that are associated with the illness.
The findings came as a surprise to the researchers because in previous studies where mice were used for Alzheimer's disease research, the apoE4 gene did not result in increased beta-amyloid levels.
New Possible Cure For Alzheimer's Disease
To repair the abnormalities in the human brain caused by the apoE4 gene, the researchers created compounds that transformed the apoE4 protein into the harmless apoE3 protein. These so-called structure correctors eliminated all the possible signs for Alzheimer's disease, and the researchers are now looking to further improve the compounds to prepare for human testing in the future.
According to Yadong Huang, the lead author of the study, the development of an Alzheimer's disease cure has been "largely a disappointment" in the past decade, with many drugs working in mice but failing in clinical trials with humans. With this new research tested on humans and not mice, this may be science's best shot at finding the elusive Alzheimer's disease cure.