UCLA researchers found that an experimental cancer drug improves the signs of progeria in a mouse model. Progeria is a rare genetic disease causing accelerated aging and cardiovascular disease in children. The new UCLA findings help to define a new strategy for treating children with progeria.
One in four million children are born with progeria that can result in dwarfism, baldness, wrinkles, hardened arteries, and osteoporosis. Most children with progeria die from heart disease before age 15.
In a new study, published Feb. 16 online in the journal Science, two UCLA researchers, Drs. Loren Fong and Stephen Young, tested a farnesyltransferase inhibitor (FTI) in mice with progeria. FTIs were initially developed by pharmaceutical companies to treat cancer. The majority of FTI-treated progeria mice showed improvements in body weight, bone integrity, grip strength, and survival compared with untreated control mice.
"This is the first study in an animal model to show that an FTI could be useful in treating progeria and related conditions," said Dr. Loren Fong, an assistant professor of medicine at the David Geffen School of Medicine at UCLA. "We believe that these studies should give some hope to progeria patients and their families."
The UCLA investigators gave an FTI to mice with progeria and normal mice, and compared both groups of mice to control mice that did not receive the drug.
By the end of the 20-week study, six of 14 nontreated progeria mice had died compared to only one of 13 FTI-treated mice. Only two rib fractures occurred in the FTI-treated progeria mice compared with 14 rib fractures in the untreated animals. All of the untreated mice exhibited an abnormally diminished grip compared with only about 30 percent of the FTI-treated mice.
Dr. Fong noted that although the FTI clearly improved the disease in the mice, the drug did not, unfortunately, completely cure all signs of the disease. Dr Fong speculated that the failure of the drug to completely prevent the disease could be due to inadequate drug dosage, which could be optimized in future studies.