The study is investigating the safety of a study medication called VES001 and how the body reacts to it. It is thought the drug may decrease neurodegeneration by acting on a gene known to cause FTD.

FTD accounts for around 5-10% of all cases of dementia, and typically presents when people are in their 50s and 60s. It is caused by damage to cells in areas of the brain called the frontal and temporal lobes, which control personality, emotions, and behaviour. They are also responsible for speech and understanding of words.

There is currently no cure for FTD and progression of the disease cannot be slowed down. Drugs that are commonly used to treat other types of dementia are not recommended for people with FTD.

Jonathan Rohrer, Professor of Neurology at Dementia Research Centre, UCL Queen Square Institute of Neurology, and consultant at UCLH, is leading the study at UCLH. Patients at UCLH will have the study specific tests and procedures at the NIHR UCLH Clinical Research Facility site  which is located within the National Hospital for Neurology and Neurosurgery. 

In many people the cause of FTD is unknown but in about a third of people there is a genetic component. A mutation affecting a gene called progranulin (PGRN) leads to a lack of the progranulin protein in the brain and leads to a subtype of FTD called GRN-FTD.

In the research study, people with a mutation in the PGRN gene will be recruited to be given the drug, VES001, a capsule taken by mouth. VES001 is a new drug, that works on specific types of receptors in the brain that interact with progranulin called sortilin.  Modifying sortilin leads to an increase in the progranulin protein back to normal levels. It is hoped that this will in turn decrease neurodegeneration in people with GRN-FTD. 

The first participant in the study to receive the study medication said: “I took part in this study because I want to help future generations, especially my children’s generation find treatments for FTD.  I had close family members who passed away with FTD. I want to do all I can to help find a cure for this debilitating disease. The staff at the UCLH CRF have been great, everyone has been totally invested in the trial.”

Professor Jonathan Rohrer said: “This is an important step forward for the FTD community. It is the first oral therapy being trialled in FTD and in this first trial we are giving it to people without symptoms of FTD but who know they will develop symptoms in the next 10-20 years because they carry an abnormality in the progranulin gene. If we can show that this drug increases progranulin levels back to normal then future trials will hopefully be focused on trying to prevent people ever developing symptoms of FTD”.

At least three and up to six patients aged between 18 to 75 years will take part in this study.  In addition to UCLH, the study is also being conducted at a hospital in the Netherlands.

The sponsor of the study is Vesper Bio ApS, a Danish Biotechnology company. 

Links

Source