Progeria is a rare genetic disorder that causes the most dramatic form of human premature aging.
Tipifarnib belongs to a class of drugs known as farnesyltransferase inhibitors (FTIs), which are being tested in people with myeloid leukemia, neurofibromatosis and other conditions.
The team of National Institutes of Health (NIH) researchers, led by Francis S. Collins, M.D., Ph.D., of the National Human Genome Research Institute (NHGRI), and Elizabeth G. Nabel, M.D., director of the National Heart, Lung and Blood Institute (NHLBI), used transgenic mice to identify and test potential therapies for children with Hutchinson-Gilford progeria syndrome.
In this study, the researchers examined the effects of an experimental cancer drug, tipifarnib, in a strain of mice genetically engineered to develop cardiovascular damage similar to that seen in progeria patients.
Earlier, the team found that FTI drugs could reverse structural abnormalities in skin cells taken from progeria patients and grown in the laboratory.
"This approach worked much better than we thought it would. Not only did this drug prevent these mice from developing cardiovascular disease, it reversed the damage in mice that already had disease," said Collins.
It was suggested that more work is needed 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. In addition, these findings shed light on the potential role of FTI drugs to treat other forms of coronary artery disease," said Nabel.
The results of the study are published in the latest edition of the Proceedings of the National Academy of Sciences.