A new use for FTIs...

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Cancer drugs may treat aging syndrome in children

By Maggie Fox, Health and Science Correspondent

WASHINGTON (Reuters) - Drugs being developed to treat cancer may also

help children with a disease called progeria, which accelerates aging

and often kills patients when they are in their teens, U.S.

researchers reported on Tuesday.

The researchers found a group of drugs known as farnesyltransferase

inhibitors or FTIs could restore the normal shape of cells damaged by

Hutchinson-Gilford progeria syndrome or HGPS.

The drugs are already being tested in children and appear to be safe,

the researchers said in two separate reports published in the

Proceedings of the

National Academy of Sciences.

" We are doing experiments to see if it actually works in animals, "

said Young of the University of California Los Angeles, who

led the first study, in a telephone interview.

His team, which is also testing other types of drugs, said the

findings could help lead to treatments for progeria and perhaps

related disorders, including osteoporosis and hardening of the

arteries but also rare conditions caused by similar processes.

One in 4 million children are born with progeria, which causes

dramatically accelerated aging and early heart disease. Children

become wizened, losing their hair, developing wrinkles and thin bones.

They usually die from hardened arteries before they are 15.

It is caused by mutations in the gene for a protein called lamin A,

important for normal function in cells. The " bad " protein deforms the

nucleus, causing miscommunications with other cells.

Young's team found in July that the farnesyltransferase inhibitors

would correct this deformity in mice bred to develop a disease similar

to progeria.

In their study published this week, they said they confirmed their

findings in cells taken from children with progeria. A second team at

the National Human Genome Research Institute, the

Medical Institute and elsewhere, made similar findings.

" FTIs, originally developed for cancer, are capable of reversing the

dramatic nuclear structure abnormalities that are the hallmark of

cells from children with progeria, " said NHGRI director Dr. Francis

in a statement.

" This is a stunning surprise, rather like finding out that the key to

your house also works in the ignition of your car. "

said it might be possible that the drugs, or similar drugs,

could affect normal aging.

" We are exploring the possibility that FTIs might also slow down the

aging process in normal cell cultures, but we don't have any data on

that yet, " he said in an e-mail.

Two FTIs are in phase III clinical testing, the last stage before U.S.

Food and Drug Administration approval. One is called lonafarnib and is

made by Schering-Plough and another called tipifarnib is made by

& .

But other approaches may work, too, said Young -- including newer

therapies called antisense and RNA interference, which directly affect

the genetic defects as opposed to interfering with the faulty protein.

In addition, Young's team found that an osteoporosis drug called

alendronate may help treat progeria by interfering with the faulty

protein. Unpublished experiments have shown it also improves the

misshapen nucleus.

But they said it was too early to test such drugs in progeria

patients. " What's attractive about it is the drugs are incredibly

safe, " Young said.