14 DAY TRIAL // The evolution of progeria, a genetic disorder that causes premature aging, can be slowed down, stopped or even reversed through the use of an experimental cancer drug, as shown by tests on mice. The discovery was announced by the National Institutes of Health (NIH) in the online edition Proceedings of the National Academy of Sciences, on October 6th.
Francis S. Collins, M.D., Ph.D., at the National Human Genome Research Institute (NHGRI), and Elizabeth G. Nabel, M.D., director of the National Heart, Lung and Blood Institute (NHLBI) led the team of scientists in charge of the tests. They found out that by using the drug tipifarnib, usually employed in cancer treatments, they were able to inhibit the symptoms of progeria in genetically modified lab mice. The animals were engineered so that they would exhibit the same wrinkling of the skin, hair loss and blood vessel damages that are typical to the Hutchinson-Gilford progeria syndrome.
Besides preventing the appearance of heart diseases in mice that had just developed progeria, the drug also canceled the effects of the syndrome in test subjects that were already at an advanced stage, by repairing damaged veins and arteries. These results puzzled even the scientists, as no one imagined that they would be this successful in trying to find a possible cure for a seemingly incurable disease. Given the fact that most children with this affliction die around the age of 13, the NIH team hopes to develop a drug similar to tipifarnib, to be used on humans with the same successful results as in mice.
The senior author of the study, Dr. Collins, the former director of NHGRI, emphasized that more research needs to be done before a viable cure for progeria is discovered. The main problem biologists are now facing is translating the results they had on mice to human clinical tests. And although mouse DNA and genetic make-up is remarkably similar to that of humans, it may be a long time before all the proper modifications are made to tipifarnib. If this particular drug doesn't work, scientists hope to be able to use the generic class it came from, farnesyltransferase inhibitors (FTI), to synthesize a viable alternative.