Researchers emphasized that more work needs to be done to determine whether FTI drugs will reverse progeria-associated cardiovascular disease in humans the same way they do in mice. In children suffering from progeria, the cardiovascular disease process often remains relatively stable until late in life, when it dramatically accelerates.
"If these drugs are found to have similar effects in children, this could mark a major breakthrough for treating this devastating disease," said NHLBI's Dr. Nabel, who was a co-author of the study. "In addition, these findings shed light on the potential role of FTI drugs to treat other forms of coronary artery disease."
In 2007, researchers led by Mark Kieran, M.D., Ph.D., at the Dana-Farber Cancer Institute in Boston launched a clinical trial of another FTI drug, lonafarnib, in 28 progeria patients, ranging in age from 3 to 15, from 16 countries. Results of that study are not expected for at least a year.
Earlier this year, in experiments involving a different type of mouse model for progeria, a group of researchers from Spain and France reported that a combination of statin and bisphosphonate drugs slowed development of the visible symptoms of aging and extended life spans in the animals. Both drugs act upon the same biological pathway as FTI drugs.
While progeria affects only a few dozen children worldwide, efforts aimed at untangling the biological roots of this rare disease may prove valuable in understanding the human aging process in general. "What we learn through studies of rare genetic disorders often has implications for more common conditions," said NHGRI Scie
|Contact: Geoff Spencer|
NIH/National Human Genome Research Institute