Cardiovascular and cerebral arterial diseases have become the leading cause of human mortality worldwide. The primary pathology is atherosclerotic plaque, characterized by the accumulation of cholesterol within the inner lining of the arteries, which obstructs blood flow. 2-hydroxypropyl-β-cyclodextrin (HPβCD) has been shown to encapsulate cholesterol, leading to plaque regression in animal models, and has been used to treat elevated cholesterol in patients with Niemann-Pick type C disease. However, its effects on humans with atherosclerosis and hypercholesterolemia had not been reported before. This pioneering study demonstrated that intravenous infusion of HPβCD significantly: (1) reduced the volume of plaques in the carotid bulb, (2) decreased blood cholesterol and triglyceride levels, (3) improved hepatic function, and (4) reduced blood uric acid levels in a human subject with severe ischemic heart disease. This new discovery may herald a new era for the treatment of atherosclerotic cardiovascular diseases and metabolic syndrome. We appreciate that further randomised clinical trials are needed to consolidate these findings and establish HPβCD as a therapeutic agent.
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