A compound used to solubilize drugs surprisingly improves the symptoms of a rare genetic disorder, Niemann-Pick type C, and a new study in PNAS provides some clues as to how. Researchers at the University of Texas Southwestern Medical School in Dallas fed NPC mice a form of cyclodextrin, a doughnut-shaped chemical with a water-soluble ring and a fat-soluble hole that enables the compound to solubilize an otherwise insoluble molecule, such as a drug. The hallmark of NPC is disrupted cholesterol trafficking inside cells, causing the substance to accumulate and leading to problems in the liver and brain. When the researchers, led by linkurl:John Dietschy,;http://www.utsouthwestern.edu/findfac/professional/0,,11822,00.html administered one dose of cyclodextrin to young NPC mice, the amount of accumulated cholesterol in cells decreased markedly, liver function and neural function improved, and the mice lived longer than untreated NPC mice.
Chemical structure of beta-cyclodextrin
"There are major biochemical shifts, very suddenly," Dietschy told...
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