A team of scientists at Imperial College London have revealed that they successfully used gene therapy to prevent the development of Alzheimer’s Disease in mice
Scientists have managed to prevent the development of Alzheimer’s Disease in mice. The research, published in Proceedings of the National Academy of Sciences, involved using a virus to insert a specific gene into the brains of mice. The results have provided hope of a potential new treatment for Alzheimer’s Disease, of which there is yet no cure.
The inserted gene, named PGC-1 alpha, has been linked to Alzheimer’s disease before, with earlier studies reporting lower levels of the gene in the brains of Alzheimer’s patients when compared to healthy individuals. Previous work conducted by the team at Imperial suggests that PGC-1 alpha prevents the formation of a peptide called amyloid-beta. This peptide aggregates to form amyloid plaques within the brains of Alzheimer’s patients—a well known hallmark of the disease.
When these amyloid plaques form within the hippocampus and the cortex of the brain, Alzheimer’s patients experience short-term and long-term memory loss respectively, as well as changes in mood, thinking, and reasoning. As Alzheimer’s advances, patients often become immobile, experience personality changes, and an inability to verbally communicate. The disease currently affects over half a million people in the UK, and costs the NHS £4.3 billion annually.
In this new study, researchers injected a modified virus, which is commonly used in gene therapy and known as a lentivirus vector which contains PGC-1 alpha, into the brain of a mouse during the early stages of Alzheimer’s disease. After four months, mice that received gene therapy had developed far fewer amyloid plaques when compared to untreated mice. The team also found that the treated mice performed just as well as healthy mice in memory tasks.
Despite the promising results of the study, Dr Magdalena Sastre, senior research author from the Department of Medicine at Imperial College, suggests that the public must not get too excited about these findings. She says: “There are many hurdles to overcome, and … we are still years from using this in the clinic,” although admitting that “gene therapy may have potential therapeutic use for [Alzheimer’s] patients.”
Meanwhile, at the University of Manchester, an alternative to gene therapy for the treatment of Alzheimer’s disease is also being researched. During the summer of this year, a team led by Dr David Brough discovered that the disease can be successfully treated with a commonly used anti-inflammatory drug called mefenamic acid. It was shown to target the inflammatory pathway that contributes towards the development of Alzheimer’s Disease. Using mice as an experimental model of Alzheimer’s disease, results from the study, published in Nature Communications, report that treatment with mefenamic acid completely reversed memory loss and brain inflammation in mice.
Whether it be gene therapeutics or switching the application of an already known drug, it is clear that the hunt for an effective treatment for Alzheimer’s disease is on.