Nilotinib: from animal-based studies to clinical investigation in Alzheimer's disease patients

Since their first description in the brains of patients suffering from Alzheimer’s disease (AD), more than 100 years ago, extracellular amyloid-β (Aβ) plaques and intracellular neurofibrillary tangles have been the principal focus of AD research. However, this focus has led to the failure of several long and promising clinical trials, and the efficacy of new Aβ-targeting drugs to slow down the disease progression is still controversial despite being successful in reducing the Aβ load. Thus, despite the discouraging results, the lessons that have been learned from the Aβ debacle have prompted studies focusing on new molecular targets that regulate different cellular pathways and have formulated new hypotheses for investigating the involvement of different brain regions beyond the temporal lobe, including the hippocampal region.