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: Quote) , 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
American Airlines Group said pilots at its subsidiaries American Airlines and US Airways, represented by Allied Pilots Association, have approved a new five-year contract which provides immediate pay raises of 23 percent and subsequent annual raises of three percent for the next five years. LEGO Group, TT Games and Warner Bros. Interactive Entertainment have announced their 2015 slate of video games, including LEGO Jurassic World, LEGO Marvel's Avengers, and as well as new hand-held and mobile titles. Social-networking giant Facebook, Inc. is said to be testing their new 'Place Tips' feature for iOS users that will rival a similar service being provided by Yelp or Foursquare. The feature will provide the user relevant content about their location as long as they are there through pop ups on their news feed page of Facebook.
comments powered by Disqus
RELATED NEWS
Trade MRK now with 
Follow RTT