Neurotoxicity-related proteins

Neurotoxicity-related proteins is a broad, non-specific category encompassing various proteins that contribute to neuronal damage and death (NIH, 2023). This group includes misfolded proteins such as Amyloid-beta, Tau, and alpha-synuclein, which form toxic aggregates in neurodegenerative diseases like Alzheimer's and Parkinson's (PubMed, 2022). It also includes proteins involved in excitotoxicity, such as N-methyl-D-aspartate (NMDA) receptors, and those mediating inflammatory responses in the central nervous system (StatPearls, 2023). Because this term refers to a heterogeneous class of molecules rather than a single therapeutic target, drug development efforts are typically directed at specific members within this category (FDA, 2021). Therapeutic strategies involve reducing the production, increasing the clearance, or preventing the aggregation of these toxic proteins to preserve neuronal function (Nature Reviews Drug Discovery, 2021). Consequently, while the term is useful for functional grouping, it lacks the molecular specificity required for a canonical drug target definition.

Other names

Neurotoxic proteinsNeurodegeneration-associated proteinsProteotoxic species

Mechanisms vary by specific protein and include the inhibition of toxic protein aggregation, enhancement of proteolytic clearance, and antagonism of receptors mediating excitotoxic cell death (PubMed, 2022; FDA, 2021).