Repurposing a human drug to treat Alzheimer’s disease

Alzheimer’s disease is a major cause of disability among older adults and represents substantial financial and human costs to patients, families and societies worldwide. Currently there is no cure for Alzheimer’s disease. Thus, novel effective treatments need to be developed that target the root causes of Alzheimer’s disease, in particular the pathogenic accumulation of the protein tau. During our ERC project, we achieved a breakthrough by demonstrating that an approved human drug efficiently inhibits pathogenic tau accumulation in several pre-clinical assays. The identified FDA/EMA-approved small molecule is orally bioavailable, safe in humans and reaches the brain at high concentrations. It is the aim of this proof of concept project to provide further pre-clinical validation of the drug and to pursue the needed steps towards IPR protection and commercialization. Because no disease-modifying therapy currently exists for AD, bringing the novel repurposed drug closer to the market promises an outstanding high gain.