Nilotinib was approved by the US Food and Drug Administration (FDA) for chronic myeloid leukaemia in 2007. Nilotinib has been proposed for repurposing as a disease-modifying treatment for neurodegenerative diseases including Alzheimer’s disease (AD). In 2017, the Georgetown University group started a Phase II clinical trial enrolling 37 people with mild to moderate AD (mean age 70.7 years old). The randomised, double‐blind and placebo‐controlled study investigated the safety and tolerability of nilotinib. Participants were given either a placebo or nilotinib daily for 12 months.
According to the data published online on 29 May 2020 in the journal Annals of Neurology, nilotinib was safe and well-tolerated, although more adverse events, particularly mood swings (agitation and irritation), were found at the higher dose. In addition, researchers reported that some AD biomarkers were reduced at 6 and/or 12 months in the nilotinib group compared to the placebo. A multicentre and larger Phase III trial is planned, to evaluate the safety and efficacy of nilotinib in people with AD or mild cognitive impairment.