A groundbreaking trial has found a new genetic therapy for Alzheimer’s disease that can lower the levels of the tau protein, which is known to cause the disease.
The trial was conducted by Dr. Catherine Mummery at UCL and UCLH, making it the first time that a “gene silencing” approach has been used in dementia and Alzheimer’s disease.
The approach used a drug called BIIB080 to “silence” the gene coding for the tau protein.
This prevents the gene from being translated into the protein in a doseable and reversible way. It also lowers the production of that protein and alters the course of the disease.
Currently, there are no treatments targeting tau. The drugs approved for use by the FDA target a separate disease mechanism in AD, the accumulation of amyloid plaques.
The phase 1 trial looked at the safety of BIIB080, what it does in the body, and how well it targets the MAPT gene.
It involved 46 patients, with an average age of 66, and was supported by the NIHR UCLH Biomedical Research Center.
The trial showed that the drug was well-tolerated, with all patients completing the treatment period and over 90% completing the post-treatment period.
Patients in both the treatment and placebo groups experienced mild or moderate side effects, the most common being a headache after injection of the drug.
However, no serious adverse events were seen in patients given the drug.
The research team also looked at two forms of the tau protein in the central nervous system over the duration of the study.
They found a greater than 50% reduction in levels of total tau and phosphor tau concentration in the CNS after 24 weeks in the two treatment groups that received the highest dose of the drug.
The trial was a big step forward in demonstrating that we can successfully target tau with a gene-silencing drug to slow, or possibly even reverse, Alzheimer’s disease and other diseases caused by tau accumulation in the future.
However, further research is necessary to understand the extent to which the drug can slow the progression of physical symptoms of the disease and evaluate the drug in larger and more diverse populations.
How to prevent Alzheimer’s disease effectively
Alzheimer’s disease is a complex condition with multiple risk factors that can increase a person’s likelihood of developing the disease.
While there is no surefire way to prevent Alzheimer’s, there are steps individuals can take to reduce their risk.
Exercise regularly: Regular physical activity has been shown to reduce the risk of cognitive decline and may help slow the progression of Alzheimer’s disease.
Aim for at least 30 minutes of moderate-intensity exercise, such as brisk walking, on most days of the week.
Maintain a healthy diet: A healthy diet that includes plenty of fruits, vegetables, whole grains, lean protein, and healthy fats may help protect against Alzheimer’s disease.
Research suggests that the Mediterranean diet, which emphasizes these foods, may be particularly beneficial.
Engage in mentally stimulating activities: Keeping your brain active through activities such as reading, puzzles, or learning a new skill may help build cognitive reserve and reduce the risk of cognitive decline.
Get enough sleep: Chronic sleep deprivation has been linked to cognitive decline and an increased risk of Alzheimer’s disease. Aim for seven to nine hours of sleep per night.
Manage chronic health conditions: Chronic health conditions such as high blood pressure, diabetes, and high cholesterol can increase the risk of Alzheimer’s disease. Work with your healthcare provider to manage these conditions and reduce your risk.
Stay socially connected: Social isolation and loneliness have been linked to an increased risk of cognitive decline and dementia.
Stay connected with family and friends and consider joining social groups or volunteering in your community.
Protect your head: Traumatic brain injury has been linked to an increased risk of Alzheimer’s disease. Wear a helmet during activities that could result in head injury, such as biking or skiing.
If you care about brain health, please read studies about vitamin D deficiency linked to Alzheimer’s and vascular dementia, and higher magnesium intake could help benefit brain health.
For more information about brain health, please see recent studies about antioxidants that could help reduce dementia risk, and coconut oil could help improve cognitive function in Alzheimer’s.
The study was conducted by Catherine J. Mummery et al and published in Nature Medicine.
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