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Smart Cholesterol-Mopping Polymer Nanoparticles in Niemann-Pick Disease Type C

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Pioneering strategy shows great promise in treating the deadly Niemann-Pick Disease Type C

There’s no effective treatment for Niemann-Pick Disease Type C, a rare genetic condition that affects children. Cyclodextrins, a family of naturally occurring sugars, are a possible treatment option.

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Niemann-Pick Disease Type C (NPC) is caused by the accumulation of cholesterol, particularly in the brain. This cholesterol build up causes brain damage, and inevitably a decline in cognitive skills and mobility. Signs and symptoms usually first appear in childhood and progress to cause life-threatening complications by the second or third decade of life. NPC affects 1 out of every 100 000 people in the EU.

Examining the potential of cyclodextrins

Cyclodextrins (CDs) represent a potential therapeutic intervention in treating this often fatal disease because of their ability to remove cholesterol. However, CDs don’t effectively cross the blood-brain barrier (BBB). What’s more, high doses of CDs are usually needed to have a satisfactory therapeutic effect. “Our approach indicates that potentially we can overcome the problem by embedding CDs in very large molecular structures,” comments Marie Skłodowska-Curie fellow Antonino Puglisi, who conducted the experimental studies for the EU-funded NanoMop project. “By doing so, we addressed an important question that needs to be answered urgently and will contribute to the treatment of this neglected disease.” To provide better and more effective therapies for NPC, project partners designed, synthesised and characterised a novel class of polymer nanoparticles. They created a pH-sensitive nanoarchitecture through an easy-to-use method.

Innovative approach to cholesterol-related diseases

The researchers developed a synthetic method to create polymeric nanoparticles containing the beta-Cyclodextrin (beta-CD). The beta-CD was released from the polymer in a slightly acidic setting. Typically, the beta-CD substance can’t overcome the BBB because of its molecular weight and its hydrophilicity. They found that when the beta-CD is bound to the polymer and organised into a nanoparticle, this can potentially overcome the BBB issue. As a result, the beta-CD can be released from the nanoparticle and remove cholesterol. “Such CD-based nanoparticles represent a promising therapeutic tool in the treatment of cholesterol-associated conditions,” Puglisi explains. In particular, they potentially tackle some of the limitations associated with existing CD-based treatment based on monomeric CDs that have a challenging bioavailability and delivery. “Besides providing advancements in the potential treatment of NPC, NanoMop’s results may also contribute towards the treatment of other neurodegenerative disorders that exhibit impaired cholesterol metabolism, such as Alzheimer’s, Parkinson’s and Huntington’s diseases,” concludes Puglisi. NanoMop’s results were recognised by Innovation Radar, a European Commission initiative that identifies high-potential innovations and the innovators behind them thanks to EU-funded research.


NanoMop, cholesterol, nanoparticle, brain, cyclodextrin, β-CD, Niemann-Pick Disease Type C, BBB, blood-brain barrier, β-Cyclodextrin

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