Grant: Project Grant

Alzheimer's disease is the most prevalent neurodegenerative disease and the most common cause of dementia. It affects not only the people living with the disease but also theirs families and surroundings. Unfortunately, there is no cure or treatment for Alzheimer's disease and current medications have only marginal (if any) effects, including the recently approved vaccine in the USA. Our project aims to develop a novel therapeutic approach for Alzheimer's disease and related dementias by successfully restoring cognitive functions. It applies to microRNAs, which have been previously linked to Alzheimer's progression in animal models and in humans. We will focus on one particular microRNA, miR-132, shown to be strongly and consistently lower in Alzheimer's brain. We and others have obtained strong evidence that miR-132 is the prime microRNA target (among more than 100 in the brain) to move towards clinical development. This is based in published studies in cells and mice showing that miR-132 can specifically control genes involved in Alzheimer pathology and importantly memory. We now aim to provide the necessary preclinical details (e.g. pharmacodynamics, toxicity) in two animal species, mice and monkeys, for future clinical studies in humans. This will be referred to as a microRNA replacement therapy, which is also underdevelopment in humans for other complexe diseases such as cancer. Overall, we aim to provide an innovative and much-needed new alternative to Alzheimer's disease treatment, as all "typical" drugs tested so far against amyloid of tau have failed.