A clinical trial to test potential treatments for a major cause of vascular dementia

Lead applicant: Prof Joanna Wardlaw

Institution: University of Edinburgh

Grant type: Project

Amount awarded: £248,785

Duration: 2 years

Scientific title: Preventing cognitive decline and dementia from cerebral microvascular disease

Why did we fund this project?

Comments from members of our Research Network:

'Sensible trial using well established drugs. Might also advance knowledge of the role of small vessel disease in dementia in general, which is poorly understood at present'

'Important research on a wide ranging problem'

'Important to research this dementia as it affects so many people and nothing is currently available'

What do we already know?

Small vessel disease is the cause of a quarter of all strokes and it may be involved in the development of up to 40 per cent of dementias. It is a major cause of vascular dementia and may also contribute to mixed dementia, where someone is affected by both vascular dementia and Alzheimer's disease.

Small vessel disease is thought to be caused by damage to the lining of the tiny blood vessels in the brain;  this damage stops the vessels from functioning normally and can lead to problems with brain function, including with thinking and memory. There is no proven treatment for small vessel disease. Trials using antiplatelet drugs, which are used to prevent some cardiovascular conditions, have been ineffective or even hazardous; trials using treatments to lower blood pressure or statins have been disappointing. Two drugs that are licensed for other vessel diseases,  cilostazol and isosorbide mononitrate, may work on mechanisms that can benefit those with small vessel disease.

What does the project involve?

The researchers want to set up a short clinical trial of cilostazol and isosorbide mononitrate in 60 volunteers with small vessel disease, to find out about how the participants feel whilst taking the drugs. The trial also aims to test whether the drugs improve blood vessel function by using some detailed scanning and blood vessel measurements, and find out if both drugs might be better than just one. If these drugs are shown to be safe in people with small vessel disease, the researchers will test them in a larger clinical trial to find out if these drugs prevent worsening of the disease.

How will this benefit people with dementia?

There are currently no effective treatments for vascular dementia. This study will allow researchers to decide whether to progress to larger trials of the two promising drugs. If these drugs are shown to be effective in treating small vessel disease then this could damp down, or even remove, a major cause of vascular dementia.

Using drugs that have already been licensed for other conditions is an important strategy for finding new treatments for dementia as it will shorten the time it takes to bring potential treatments to those who need it most. This is known as drug repurposing and is an important part of our Drug Discovery programme.