Posted by This work presents the most recent advances in the research and clinical use of HPβCD against Niemann–Pick disease, a congenital condition involving cholesterol accumulation inside lysosomes in brain cells, Alzheimer’s and Parkinson’s. The mechanism of action is discussed in details: HPβCD plays a complex role in each of these ailments, going beyond the mere sequestering of cholesterol molecules and involving an overall regulation of protein expression that helps restore the normal functioning of the organism. The possible role of autophagy is also discussed see the featured image).
Spetial emphasis is given on clinical results. The ones with a few children suffering in Niemann Pick disease type C gave promising results proving that HPBCD can slow down the progression of the disease, but a larger trial has not shown significant difference compared to the control group.
The first clinical trial to evaluate the potential medicinal efficacy, toxicological safety and tolerability of HPβCD in the treatment of early-stage Alzheimer’s disease launched by Cyclo Therapeutics, Inc, in collaboration with various research institutes started the recruitment of patents in November 2022.
No data on human studies concerning Parkinson’s disease.
Braga, S.S. Molecular Mind Games: The Medicinal Action of Cyclodextrins in Neurodegenerative Diseases. Biomolecules 2023, 13, 666. https://doi.org/10.3390/biom13040666
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