Posted by Niemann–Pick disease type C (NPC) and Alzheimer’s disease (AD) are both characterised by cognitive decline and neurological impairment. In each condition, abnormal cholesterol accumulation and disrupted cholesterol homeostasis in the brain may contribute to disease progression and symptom severity. Cyclodextrins have emerged as potential therapeutic agents capable of lowering free cholesterol in the brain.
A well-known water-soluble β-cyclodextrin polymer (β-CDP) — composed of multiple β-CD units crosslinked with epichlorohydrin synthesized by Cyclolab, Hungary — exhibits improved safety and prolonged circulation time in the bloodstream. In this study, a group of Japanese scientists eminent in the application of cyclodextrins as active pharmaceutical ingredient candidates investigated the therapeutic potential of β-CDP in NPC and AD. β-CDP reduced free cholesterol levels and restored autophagy function in cholesterol-loaded human neuroblastoma cells. Subcutaneous administration in NPC mice also resulted in extended systemic circulation and decreased cholesterol accumulation.
Together, these findings indicate that β-CDP may represent a safe and promising therapeutic option for the treatment of NPC and AD.
Yuto Kubohira, Nanami Okano, Toru Taharabaru, Arina Ishimatsu, Takumi Era, Kazunori Yanagihara, Kandai Ishikura, Yoshinori Nakagawa, Taishi Higashi, Keiichi Motoyama: A water-soluble
β-cyclodextrin polymer reduces cholesterol accumulation and autophagy dysfunction in
vitro and in Niemann-Pick type C disease model mice, Carbohydrate Polymers (2025),
https://doi.org/10.1016/j.carbpol.2025.124676