logo
Share SHARE
FONT-SIZE Plus   Neg

Breakthrough Discovery: First-Ever Treatment For Progeria Proves Promising

More than nine years have elapsed since scientists discovered the cause of Progeria - the deadly genetic condition of premature aging in children. Although as yet there is no cure, a drug originally developed for cancer has demonstrated efficacy in the first-ever clinical drug trial for children with Progeria.

Progeria, which is derived from the Greek word for old age, is an extremely rare disease and is estimated to affect approximately 1 in 4 - 8 million newborns. Children with progeria may suffer heart attacks and strokes as early as age 5 years, and their average life expectancy is just 13 years.

In the two-and-a-half year drug trial, twenty-eight children from sixteen countries were enrolled and they were treated with farnesyltransferase inhibitor lonafarnib, supplied by Merck & Co. (MRK) , given orally, twice-a-day over the course of the study.

According to the trial results, there were significant improvements in weight gain, bone structure and, most importantly, the cardiovascular system of the children.

Progeria is caused by a mutation in the gene called LMNA that produces a progerin protein, damaging the cell function. In children with progeria, a molecule called a "farnesyl group" gets attached to the progerin protein. The farnesyltransferase inhibitor drug is known to work by blocking the attachment of the farnesyl group onto progerin.

The study results were published September 24 in Proceedings of the National Academy of Sciences.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Editors Pick
Bank of Nova Scotia or otherwise known as Scotiabank, Friday reported lower net profit for the third quarter, in the absence of a year-ago gain. Earnings per share, however, came in line with the Street view. Further, the company said it raised its quarterly dividend by 2 cents. Non-traditional discount and variety stores operator Big Lots Inc. (BIG) Friday reported a decline in net profit for the second quarter, while net sales grew 1.2 percent from the prior year. Comparable sales for the quarter increased 2.8 percent. Google has rejected the European Union's charges that it abused its market power and the demanded that it change the way it ranks online comparison shopping services in its search results, setting up a potentially long legal battle with the EU regulator empowered to levy billions of euros in fines
comments powered by Disqus
RELATED NEWS
Trade MRK now with 
Follow RTT