2-Hydroxypropyl-beta-cyclodextrin (HP-beta-CD) (HP-beta-CD)

2-Hydroxypropyl-beta-cyclodextrin (HP-beta-CD) is a chemically modified cyclic oligosaccharide consisting of seven glucopyranose units, widely utilized in the pharmaceutical industry as an excipient to enhance the solubility and stability of hydrophobic drugs [PubChem, 2024]. Beyond its role as a formulation aid, HP-beta-CD has been developed as a primary therapeutic agent for Niemann-Pick disease type C (NPC), a fatal lysosomal storage disorder characterized by the accumulation of unesterified cholesterol [NIH, 2021]. The molecule functions by forming inclusion complexes with sequestered cholesterol, facilitating its transport out of the endolysosomal system and effectively bypassing the functional requirement for the defective NPC1 or NPC2 proteins [PubMed, 2010]. In addition to NPC, HP-beta-CD is being investigated for its potential to reduce atherosclerotic plaques and clear amyloid-beta aggregates in neurodegenerative diseases [Journal of Controlled Release, 2019]. However, its clinical utility is constrained by significant safety concerns, most notably dose-dependent ototoxicity that can lead to permanent hearing loss [PubMed, 2017]. Despite these challenges, it remains a pivotal compound in the study and treatment of lipid trafficking disorders.

Other names

HydroxypropylbetadexHPBCD2-Hydroxypropyl-β-cyclodextrinKleptose HPB

HP-beta-CD acts as a cholesterol-shuttling agent that forms inclusion complexes with unesterified cholesterol, facilitating its egress from the lysosomal/late endosomal compartment to the cytosol and plasma membrane, thereby bypassing defective NPC1/NPC2 transport proteins [PubMed, 2010].