A new oral treatment is offering hope for people at risk of frontotemporal dementia (FTD), a serious brain disease that currently has no approved treatments.
For the first time ever, a clinical trial has shown that this treatment can restore important protein levels in people who carry a gene mutation linked to FTD.
The treatment, called VES001, was developed by Vesper Bio, a Danish biotech company co-founded by Professor Anders Nykjær and Mads Kjølby. It is based on years of research by scientists at DANDRITE and other international partners.
The treatment works by blocking a brain receptor called sortilin, which is responsible for breaking down a protein called progranulin. People with a certain gene mutation don’t have enough of this protein, which plays a key role in keeping nerve cells healthy.
Professor Nykjær was part of the team that first discovered how the sortilin receptor affects progranulin levels. “We now have hope for a treatment that could stop this form of dementia from developing in people who are at genetic risk,” he said. “This is a major step forward.”
FTD is a group of diseases that affect the front and side areas of the brain, impacting behavior, language, and thinking. It is the leading cause of dementia in people under 60 and moves quickly once it begins. Actor Bruce Willis has been diagnosed with this condition, bringing more attention to it in recent years.
One type of FTD, called FTD-GRN, is caused by changes in the GRN gene. These changes lower progranulin levels in the brain, which leads to the death of nerve cells and the symptoms of FTD. The new treatment VES001 is designed to fix this by stopping the breakdown of progranulin, allowing levels of the protein to return to normal.
In the latest trial, people who carried the GRN mutation but had not yet developed symptoms were given VES001. The results were impressive—progranulin levels in the fluid around the brain rose by more than 95%, and no serious side effects were reported. This suggests that the treatment is both effective and safe at this early stage.
The success of this trial marks the first time that any oral treatment has been able to restore progranulin to normal levels in people with this gene mutation. While this trial was small and focused on people who hadn’t developed symptoms yet, the next step will be to test the treatment in patients who are already showing signs of FTD.
Vesper Bio plans to begin a larger trial in 2026 to see how VES001 works in people with FTD symptoms. If these trials are successful, it could lead to a treatment that not only slows down but also possibly prevents this disease.
Although it may still take several years before this treatment is available to the public, these results are an exciting step forward. For the first time, researchers may be able to prevent one of the most serious forms of early-onset dementia, and that’s thanks in large part to basic scientific research carried out in Denmark and beyond.
If you care about dementia, please read studies about low choline intake linked to higher dementia risk, and how eating nuts can affect your cognitive ability.
For more information about brain health, please see recent studies that blueberry supplements may prevent cognitive decline, and results showing higher magnesium intake could help benefit brain health.
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