Cancer drugs may treat aging syndrome in children

Drugs being developed to treat cancer may also help children with a disease called Progeria (also known as Hutchinson-Gilford syndrome), 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 (FTIs) could restore the normal shape of cells damaged by Hutchinson-Gilford progeria syndrome (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 Stephen 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 as well as 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.

Progeria is caused by mutations in the gene for a protein called lamin A, important for normal function in cells. The abnormal protein results in a deformed cell nucleus, causing miscommunications with other cells.

Young’s team found in July that FTIs corrected this deformity in mice bred to develop a disease similar to progeria.

In their study published this week, the researchers report that they confirmed their findings in cells taken from children with progeria. A second team at the National Human Genome Research Institute (NHGRI), the Howard Hughes 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 Collins 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.”

Collins 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 Johnson & Johnson.

But other approaches may work, too, said Young - including newer drugs 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 is too early to test such drugs in progeria patients. “What’s attractive about it is the drugs are incredibly safe,” Young said.

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