Dementia, caused by Alzheimer’s disease (AD) or related diseases, is a major cause of disability and dependency among older adults worldwide, affecting memory, cognitive abilities, and behavior, ultimately interfering with one’s ability to perform daily activities. The impact of dementia is not only significant in financial terms, but also represents substantial human costs to countries, societies, families and individuals. In 2019, dementia affected 55 million people worldwide, with a predicted increase to 78 million in 2030 and 139 million by 2050. However, no cure is currently available for AD or dementia.
The solution: Accumulation of the protein tau in the brain is a pathological hallmark of AD, and the decline of cognition in patients with AD closely correlates with the degree and site of tau pathology. Many pharmaceutical companies therefore target pathological tau for disease intervention, with a strong focus on the development of tau antibodies. A major limitation of the currently investigated tau antibodies is, however, that they cannot enter neurons and thus are unable to act where tau accumulation occurs. There is thus a clear need to develop drugs that inhibit tau aggregation inside neurons. My lab achieved a breakthrough towards the development of a drug that inhibits pathogenic tau accumulation by repurposing a small molecule for the treatment of AD. The aim of our PoC project is tovalidate the pre-clinical application of the drug in transgenic tau-overexpressing mice.
