Danish basic research behind international medical breakthrough: New oral treatment may prevent dementia

The first clinical trial in patients has just been completed and shows that the treatment can restore levels of the protein progranulin to normal in individuals carrying a gene mutation that typically leads to frontotemporal dementia (FTD).

“This means we now have hope for a treatment that could potentially prevent the development of this form of dementia in people at genetic risk – thereby conceptually transforming the future of dementia therapy,” says Professor Anders Nykjær.

He is one of the leading researchers who helped uncover the functions of the sortilin receptor and how it regulates the breakdown of the protein progranulin in nerve cells – a process that plays a central role in the development of frontotemporal dementia (FTD).

“For several years, we have been studying the role of the sortilin receptor in neurodegeneration. Seeing this knowledge translated into a treatment that now shows promising clinical results is a major step forward for the field – and a testament to the vital importance of basic research for the development of new therapies.”

FTD is the most common cause of dementia in people under the age of 60 and progresses rapidly. At present, there are no approved treatments.

The new treatment, VES001, has been developed by Vesper Bio with contributions from Anders Nykjær and international collaborators. Vesper Bio is a Danish biotechnology company founded by Anders Nykjær and Mads Kjølby. The treatment is based on a targeted mechanism that selectively inhibits the sortilin receptor to prevent the breakdown of progranulin, without disrupting other vital brain functions.

The trial demonstrated normalisation of progranulin levels in cerebrospinal fluid and a favourable safety profile, with no serious adverse effects in patients who have not yet developed symptoms of the disease. The next step will be a larger clinical trial involving symptomatic patients.

Q&A on the Disease and the Treatment

What is frontotemporal dementia (FTD)?
FTD is a group of brain disorders that affect the frontal and temporal lobes. It impacts behaviour, language, and the ability to manage everyday activities. It is the most common cause of dementia in people under the age of 60 and progresses very rapidly. The disease is also known as “Bruce Willis dementia”, as actor Bruce Willis has publicly shared that he is living with frontotemporal dementia.

What is FTD-GRN?
FTD-GRN is a form of FTD that can be caused by mutations in the progranulin gene (GRN). This leads to low levels of the protein progranulin, which is essential for the health of nerve cells.

What is VES001?
VES001 is a new oral treatment developed by the Danish company Vesper Bio. It is designed to raise progranulin levels by inhibiting the sortilin receptor, without affecting other brain functions that are important for health.

Why is this breakthrough important?
This is the first time an oral treatment has been shown to normalise progranulin levels in people with GRN mutations. It could potentially prevent them from developing symptoms of FTD.

What did the trial show?
In the Phase Ib/IIa study, progranulin levels increased by more than 95% in cerebrospinal fluid. The treatment was safe and well tolerated, with no serious side effects.

What are the next steps?
Vesper Bio plans to launch a Phase IIb/III trial in 2026 to test the treatment’s effect in people with symptoms of FTD-GRN.

What role has Danish research played?
Danish researchers have contributed to the fundamental understanding of the sortilin receptor’s role and the concept of selective inhibition, which forms the scientific basis for VES001.

When might patients expect a treatment?
There are still several clinical stages to complete before approval can be considered. It is too early to give a timeline, but these results represent an important step towards a possible treatment.