Target intelligence / Profile preview

Pathological accumulation

Molecular classification
Other
01

Overview

Pathological accumulations refer to the abnormal buildup of substances—predominantly misfolded proteins, lipids, or mineral deposits—within or outside of cells, leading to progressive tissue damage and organ dysfunction [NIH, 2023]. In neurodegenerative disorders such as Alzheimer's and Parkinson's, these accumulations manifest as protein aggregates like amyloid-beta plaques, tau tangles, or alpha-synuclein inclusions that disrupt synaptic signaling and trigger apoptosis [PubMed: 32051515]. This phenomenon is also central to systemic diseases like transthyretin amyloidosis and atherosclerosis, where deposits impair cardiac or vascular integrity [StatPearls: Amyloidosis]. Therapeutic strategies focus on preventing the formation of these aggregates through molecular stabilizers or facilitating their removal using immunotherapy and enhanced autophagy pathways [Nature Reviews Drug Discovery, 2021]. However, targeting these accumulations is complex, as treatments must distinguish between toxic aggregates and functional protein monomers to avoid adverse effects like Amyloid-Related Imaging Abnormalities (ARIA) [FDA: Aducanumab Label]. Because 'Pathological accumulations' describes a broad class of disease manifestations rather than a single molecular target, it is typically categorized as a pathological endpoint rather than a specific receptor or enzyme.

Other names
Protein aggregationPathological depositsProteopathyInclusion bodiesAmyloidosisAggregopathyIntracellular inclusions
02

Mechanism of action

Monoclonal antibody-mediated immune clearance of aggregates, kinetic stabilization of precursor proteins to prevent misfolding, and RNA interference to reduce synthesis of amyloidogenic precursors.

03

Biological functions

Other
04

Disease associations

Neurodegenerative diseaseCardiovascular diseaseMetabolic diseaseAmyloidosisPrion disease
05

Safety considerations

Amyloid-Related Imaging Abnormalities (ARIA-E and ARIA-H)NeuroinflammationOff-target depletion of functional monomeric protein formsInfusion-related reactionsPotential for immune-mediated tissue damage
06

Interacting drugs

Aducanumab

5 more in the full profile.

07

Biomarkers

Amyloid PET imagingTau PET imagingCerebrospinal fluid Amyloid-beta 42/40 ratioPlasma p-tau217Serum Amyloid A levelsB-type natriuretic peptide (BNP) for cardiac amyloidosis

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