In a new study, researchers found a gene can naturally suppress the signs of Alzheimer’s Disease in human brain cells.
They also developed a new rapid drug-screening system for treatments that could potentially delay or prevent the disease.
The research was led by the Queen Mary University of London.
The main challenge in testing Alzheimer’s drugs in clinical trials is that participants need to have symptoms.
But once people have symptoms, it is usually too late for treatments to have a significant effect, as many brain cells have already died.
The only current way to test potential preventative treatments is by identifying participants who are at higher risk of developing Alzheimer’s and seeing if treatments prevent the onset of their disease.
This includes people with Down’s syndrome (DS) who have around a 70% chance of developing Alzheimer’s during their lifetime.
This is because the extra chromosome 21 they carry includes the gene for amyloid precursor protein which causes early Alzheimer’s when overdosed or mutated.
In the study, the researchers collected hair cells from people with DS and reprogrammed them to become stem cells, which were then directed to turn into brain cells in a dish.
In these brain-like cells, they saw Alzheimer’s-like pathology develop rapidly, including the hallmark trio of signs of Alzheimer’s progression—amyloid plaque-like lesions, progressive neuronal death, and abnormal accumulations of a protein called tau inside neurons.
The researchers showed that the system could be used as an early preventative-drug testing platform.
They took two different drugs which are known to inhibit β-amyloid production, tested them on these brain cells, and in six weeks showed that they prevented the onset of Alzheimer’s-pathology.
Although these two particular drugs have failed clinical trials for other reasons and therefore aren’t suitable treatments for Alzheimer’s, the team showed the proof-of-principle that the system can be used on any drug compound, and within six weeks show whether or not it has potential for further investigation.
The team also found proof of the existence of a naturally-functioning Alzheimer’s suppressor gene (BACE2 gene).
Acting in a similar way to tumor suppressor genes in cancer, the increased activity of this gene contributes to the prevention/slowing down of Alzheimer’s in human brain tissue, and could in the future be used as a biomarker to determine people’s risk of developing the disease, or as a new therapeutic approach by boosting its action.
One author of the study is Professor Dean Nizetic from the Queen Mary University of London.
The study is published in Molecular Psychiatry.
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