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University of Exeter

Clive Ballard, MB ChB (Medicine), MMed Sci, M.R.C.Psych, MD. | England, United Kingdom

University of Exeter

Clive Ballard, MB ChB (Medicine), MMed Sci, M.R.C.Psych, MD. | England, United Kingdom

A Placebo Controlled, Randomized Double-blind Parallel Group 12-month Trial of Fasudil for the Treatment of Mild Cognitive Impairment Due to Alzheimer’s Disease, Nested within a Multi-Arm Phase 2 Clinical Trial Platform

There are 40 million people worldwide with Alzheimer’s Disease, which will increase to more than 100 million by 2050. In addition, it is estimated that at least 15% of people aged 60 or above have Mild Cognitive Impairment (MCI), and that up to 10% of these individuals will develop Alzheimer’s disease each year.  It is essential to develop more effective therapies to treat, delay and prevent Alzheimer’s disease, but there have been no new licensed treatments for 20 years, and new treatment approaches are urgently needed.  One way to achieve this is to look at compounds that are already used for other conditions that may also have actions that are beneficial for the treatment of Alzheimer’s disease.  This approach, known as repurposing, has identified valuable treatments in other areas of medicine.

Based on a recent expert consensus, fasudil is an excellent candidate for repurposing as a treatment for Alzheimer’s disease.  In studies looking at nerve cells in the laboratory and in several different mice with Alzheimer genes fasudil protects nerve cells and the junctions between nerve cells from the toxic effects of the Alzheimer changes in the brain, and improves the behavior and memory of treated mice.  Fasudil is used in Japan and China for treating heart disease and high blood pressure in the blood vessels to the lungs, and has a good safety profile in people with these conditions.

We are therefore proposing a 12 month clinical trial comparing treatment with fasudil to a placebo (dummy) treatment in 200 people with early Alzheimer’s disease.  We will measure safety and the potential benefits on sensitive computerized tests of cognitive function as the main outcome measures.  We will also look at overall benefits, benefits in every day functioning and changes in the brain measures through PET scanning, spinal fluid and blood.