From
The Wall Street Journal:
Research led by the National Institutes of Health may suggest new avenues of treatment for a rare childhood disorder–and insights into the aging process.
A group of scientists led by NIH director Francis S. Collins are reporting that the drug
everolimus clears out a protein called progerin from cells of children with progeria. This protein builds up to toxic levels in patients with progeria, a rapid-aging disorder that causes children to die of heart attacks or strokes in their teens.
The study, published today in Science Translational Medicine , found that everolimus appears to rev up cells’ own recycling system so they clear the toxic progerin out more rapidly. The underlying defect driving the genetic disease remains, Collins told the Health Blog, “but the amount of protein is reduced by 50% in treated cells, which has a profound effect on survival.”
In the paper, the researchers concluded that the data are so compelling that the drug should be tested in children with progeria.