Cancer drug reverses damage from genetic disorder

An experimental anti-cancer drug can prevent--and even reverse--potentially fatal cardiovascular damage in a mouse model of progeria, a rare genetic disorder that causes the most dramatic form of human premature aging, NIH researchers reported today.

A team led by Francis S. Collins, M.D., Ph.D., and Elizabeth G. Nabel, M.D., described its effort to use transgenic mice to identify and test potential therapies for children with Hutchinson-Gilford progeria syndrome. At birth, children with progeria appear normal. But soon growth slows, and the children begin to show signs of accelerated aging, such as hair loss, wrinkled skin and loss of body fat. The most lethal damage, however, occurs within the children's major blood vessels. The children develop premature cardiovascular disease, which typically leads to death from heart attack or stroke at about the age of 13.

The NIH-led team examined the effects of the experimental cancer drug tipifarnib--one in a class of FTI drugs--in a strain of mice genetically engineered to develop cardiovascular damage similar to that seen in progeria patients. The team had previously found that FTI drugs could reverse structural abnormalities in skin cells taken from progeria patients and grown in the laboratory.

- here's the release for more

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