Emerging Roles of Small-molecule Derivatives in Modulating Neurodegenerative Pathways: From Molecular Targets to Therapeutic Applications - PubMed
5 hours ago
- #drug-discovery
- #small-molecule-therapeutics
- #neurodegeneration
- Neurodegenerative disorders like Alzheimer's, Parkinson's, ALS, and Huntington's involve mechanisms such as protein aggregation, oxidative stress, and neuroinflammation.
- Current treatments are mostly symptomatic, with limited disease-modifying options.
- Small-molecule derivatives are being developed to target specific pathological pathways, offering new therapeutic opportunities.
- Recent progress includes molecules targeting amyloid-β metabolism, tau acetylation, α-synuclein aggregation, and NLRP3 inflammasome activation.
- Key signaling cascades like PI3K/Akt, MAPK/ERK, Nrf2/ARE, and Wnt/β-catenin are discussed.
- Challenges include optimizing pharmacokinetics, target selectivity, and blood-brain barrier penetration.
- Future directions involve biomarkers, advanced imaging, and computational tools for candidate validation.
- Multidisciplinary approaches are needed for successful translation of small-molecule therapeutics.