Show Cancer Drug Shows Promise ol I. I lit Fighting Rare Aging Syndrome Rosie Los Angeles Times Research groups at the University of California Los Angeles and the National Institutes of Health have discovered discovered discovered dis dis- covered a potential treatment for a rare genetic disease in which children age prematurely and usually die by bythe bythe bythe the time they reach their teens The finding reported online Tuesday in the Proceedings of the National Academy of Sciences showed that a class of fighting cancer-fighting drugs corrected abnormalities in cells taken from patients with Hutchinson- Hutchinson Gilford syndrome which h affects 1 in 4 million children The drugs might help prolong th the e childrens children's lives or help block a host o of f problems such as poor growth loss los s of hair and body fat and sis the scientists said The finding also opens a potential avenue of research into the causes o of f normal aging they said Children who have sy syndrome syndrome syn syn- n drome iden identified fied more than a century ry ago appear normal when they are born but within the first year the their it growth slows dramatically By abo aboul it age 2 they have lost all their h hair r They generally never reach 4 feet in height nor surpass a weight of 25 to 35 pounds In 2003 a team of scientists led by Dr Francis Collins director of the NIH's National Human Genome Research Institute reported that they had found the gene that causes the defect Known as it directs the creation of a protein called lamin A. A Under normal circumstances lamin A proteins accumulate in a layer just inside the cell nucleus But in Hutchinson-Gilford Hutchinson n ria a patients lamin A never gets to the correct destination Instead it builds up on the membrane of the nucleus The nucleus becomes misshapen with bulging projections pushing into the cells cell's cytoplasm In two separate papers Collins' Collins team and that of Drs Stephen Young and Loren Fong at UCLA reported that they had eradicated the projections projections projections in human cells by using a class of drugs known as inhibitors Young and Fong earlier showed th the e same effect using the drugs on mouse e l cells containing the defective human an gene The drugs prevent the lamin A from reaching the nuclear membrane so it cannot build up there The scientists are uncertain whether the drug treatment will translate translate translate trans trans- late into a useful therapy The next step both teams of scientists scientists scientists said would be to treat mice genetically engineered with the human gene for Hutchinson-Gilford Hutchinson Collins said a human clinical trial co could ld begin as early as next spring if the results were promising Some inhibitors have been extensively tested tested tested test test- ed for safety including in children because of their promise in fighting certain cancers Several are now in advanced clinical trials We just really are phenomenally fortunate said co-author co Dr Leslie Gordon medical director of the nonprofit nonprofit nonprofit non non- profit Research Foundation and assistant professor of pediatrics at Brown University in Rhode Island Gordon who has a a child with eria said it was unlikely that the drugs would have undergone such costly and extensive testing for eria itself because the disease is so rare |