In major breakthrough research led by the Queen Mary University of London, a gene has been discovered by scientists that can naturally suppress the signs of Alzheimer’s disease in human body cells.
A new rapid drug-screening system has also been developed by the researchers for the treatment that will be able to potentially delay or prevent the disease. The breakthrough research has also been published in the journal Molecular Psychiatry.
The major challenge in testing Alzheimer’s drugs during clinical trials is that the participants need to have symptoms of the disease. But once the person develops symptoms, it gets too late for the treatment to have a significant effect as many brain cells are already dead.
What is the current way to test a potential treatment for Alzheimer's?
Currently, the test for the potential preventive treatment of Alzheimer's is done by identifying participants who are at a higher risk of developing Alzheimer's and then to observe if the treatment prevents the onset of the disease.
This includes people with Down’s Syndrome (DS) who have around 70% chance of developing Alzheimer's in their lifetime. The reason is because they carry extra chromosome 21 which includes the gene for amyloid precursor protein that causes early Alzheimer when overdosed or mutated.
How the latest research was done?
In the published study, the researchers collected hair cells from people with Down Syndrome and reprogrammed them to become stem cells, it was then directed to turn into brain cells in a dish.
The researchers in these brain-like cells saw Alzheimer’s progression- progressive neuronal death, amyloid plaque-like lesions and abnormal accumulation of a protein called tau inside neurons.
As per Professor Dean Nizetic, the lead researcher from the Queen Mary University of London, the work represents a remarkable achievement, as this is the first cell-based system with a full trio of Alzheimer’s-pathologies, that too without any artificial gene overexpression. This system also opens up the prospect of the screening of new drugs that aim at delaying or preventing Alzheimer's before the neuronal death starts.
What does this newly found system show for Alzheimer's treatment?
As shown by the researchers, this system can be used as an early preventive-drug testing platform. They took two different drugs that are known to inhibit b-amyloid production, they tested them on these brain cells, and in six weeks showed that they have prevented the onset of Alzheimer’s-pathology.
Although these two particular drugs failed the clinical trials for other reasons and have not been proven to be a suitable treatment for Alzheimer, the team still showed the proof of principle that this system can be used on any drug compound and in just six weeks show whether or not it has the potential for further investigation.
Proof of Alzheimer suppressor gene:
The researchers also found proof of the existence of a naturally functioning Alzheimer’s suppressor gene (BACE2 gene). It was acting in a similar way to tumour suppressor genes in cancer. The increased activity of this gene contributes to the slowing down/prevention of Alzheimer’s in human brain tissue and in the future can be used as a biomarker to determine people’s risk of developing the disease or even as a new therapeutic approach by boosting its action.
Contribution to the study from people with DS:
The new discoveries in this study were dependent on the contributions from people with Down Syndrome (DS) who accepted to participate in the study whose results can be beneficial for people with or without DS in preventing Alzheimer’s.
The Down’s Syndrome Association provided essential support and help with the recruitment of the participants in the study.
Chief Executive of the Down’s Syndrome Association, Carol Boys commented that these are exciting results from an extremely eminent group of the researchers and also another small step towards a possible treatment and intervention of Alzheimer’s disease. He added that the Down’s Syndrome Association is delighted that they were able to support this brilliant work.
What does this research mean for future treatment of Alzheimer's?
Professor Dean Nizetic further explained that although it's still early days but this system raises a theoretical possibility for further development as a tool to predict who might develop Alzheimer's. The same stem cell process can be used on anyone’s hair follicles, whose resulting brain cells may or may not develop Alzheimer’s pathology in the dish.
He added that the idea will be to catch the people at the higher risk of early disease in a cell-based system before it even starts in a person’s brain and allow for the possibilities of individualized preventive interventions. It is still a long way for reaching this goal.
Professor John Hardy from UCL, co-author of the study added that we have the potential now to develop a new human model of the disease which will be a great step forward.
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