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Delving into a new delivery option
October 2016
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PORTLAND, Ore.—In hopes of finding an option for a rare, fatal genetic disorder, a team of scientists from Oregon State University and other organizations are exploring a new drug delivery system. Niemann Pick Type C1 disease, or NPC1, affects roughly one in 120,000 children globally, causing abnormal cholesterol accumulation, progressive neurodegeneration and eventual death. While there is no treatment, a promising compound is in clinical trials, but it requires direct brain injection, causes lung damage and the required high doses cause significant hearing loss. However, a new nanotech-based delivery system could deliver the drug more effectively and improve its efficacy almost five times, enabling much lower doses. This approach features the HPβCD drug attached to a nanotech-sized lipid particle that carries it into cells, where it flushes out cholesterol and was found to work in synergy with the drug to boost its effectiveness. In animal models, HPβCD slowed disease progression, but did not reverse it. This delivery system features prolonged circulation, “targeting ligands” that can help cross the blood-brain barrier and an ability to incorporate multiple drugs with differing mechanisms of action.

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