Beyond expectations: investigating nilotinib’s potential in attenuating neurodegeneration in alzheimer’s disease

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Abstract

Neurodegenerative diseases, such as Alzheimer’s disease (AD), pose a formidable global challenge. While therapeutic options are available, their limitations are significant, necessitating the development of innovative treatment approaches. Here, we highlight the importance of repurposing drugs and discuss the future of drug treatments for AD. We review the potential of tyrosine kinase inhibitors (TKI) for mitigating AD pathology and symptoms, as well as neurodegenerative processes more broadly. We focus on nilotinib, a selective BCR-ABL tyrosine kinase inhibitor, which has unique mechanisms of action involving the modulation of cell responses and removal of toxic proteins associated with AD pathogenesis. Encouraging studies have demonstrated its efficacy, calling for further investigation through clinical trials to assess its potential in various neurodegenerative conditions. However, despite these promising preclinical findings, no clinical studies have yet conclusively demonstrated its efficacy in treating AD. Considering the future directions in AD research, personalized medicine approaches hold promise by incorporating patient-specific factors, including sex and gender differences, to tailor nilotinib treatment for improved efficacy and safety profiles.
Original languageEnglish
Article number60
JournalAlzheimer's Research and Therapy
Volume17
Issue number1
DOIs
StatePublished - Dec 2025

Bibliographical note

Publisher Copyright:
© The Author(s) 2025.

Funding

This work was supported by American Heart Association (946666 to LSR), National Institute on Aging (1R03AG081865-01 to LSR), NIH 1R16NS134540-01 to BCA, and CIHR PJT-162,144 to BCA.

FundersFunder number
American Heart Association (AHA)946666
National Institute on Aging1R03AG081865-01
National Institutes of Health1R16NS134540-01, CIHR PJT-162,144

    ASJC Scopus Subject Areas

    • Neurology
    • Clinical Neurology
    • Cognitive Neuroscience

    Keywords

    • Alzheimer disease
    • Autophagy
    • Memory
    • Neurodegeneration
    • Protein aggregates
    • Repurposed

    Disciplines

    • Neuroscience and Neurobiology
    • Neurology

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