A single injection of the cholesterol-binding agent CYCLO was enough to rid mice of excessive accumulations of cholesterol in cells, a finding that could point the way to treat the lethal Niemann-Pick type C disease.
NP-C disease is the result of an extremely high accumulation of cholesterol in lysosomes and the condition triggers a series of ailments that leads to a child's death before the age of 20. The compound appears to overcome the genetic defect that triggers the disease.
"What we've shown is that very quickly after administration of this compound, the huge pool of cholesterol that has just been accumulating in the cells is suddenly released and metabolized normally," said John Dietschy, senior author of the study. Other researchers in the field say that the news is promising, but that there's still a lot to learn about the molecular activity at work.
- read the AFP report