A cancer drug could help children with the premature ageing disorder progeria, a study has suggested.
University of California, Los Angeles, researchers found a farnesyltransferase inhibitor (FTI) appeared to reduce the effects of the disease in mice.
Progeria is a rare genetic disease, affecting one in four million children.
Experts in the condition said the study, in Science, offered hope the drug could be safely given to children affected by progeria.
The condition, which is actually called Hutchinson-Gilford Progeria Syndrome (HGPS), stems from a mutation that leads to the accumulation of an abnormal protein on the scaffolding of the cell nucleus.
The abnormal protein causes misshapen cell nuclei, and ultimately leads to all of the disease findings of progeria.
Those affected can experience dwarfism, baldness, wrinkles, hardened arteries, and osteoporosis.
Most children with the condition die from heart disease before the age of 15.
Bone health
The UCLA researchers gave a farnesyltransferase inhibitor (FTI) in mice with progeria.
FTIs were initially developed to treat cancer, and are now given to patients with breast and bowel cancer as well as leukaemia patients and those with multiple myeloma – cancer of the bone marrow.
They had already shown that FTIs could prevent misshapen nuclei in progeria cells. It works preventing the abnormal protein from reaching the scaffolding of the cell nucleus.
By the end of the 20-week study, six of 14 untreated progeria mice had died compared to only one of 13 FTI-treated mice.
There were only two rib fractures in the mice given the drug, compared with 14 in untreated animals.
The majority of treated mice also showed improvements in body weight, bone health, grip strength, and survival compared with the untreated mice.
Dr Loren Fong, an assistant professor of medicine at the David Geffen School of Medicine at UCLA, who worked on the study, said: "This is the first study in an animal model to show that an FTI could be useful in treating Progeria and related conditions.
"We believe that these studies should give some hope to Progeria patients and their families."
However, Dr Fong said that although the study showed the drug appeared to benefit those animals given it, it was not a cure.
But he speculated a higher dose might be more effective.
Dr Leslie Gordon, director of the Progeria Research Foundation, said: "This study gives us pieces of information critical to our movement toward clinical trials in children with progeria.
"This type of evidence will help us to ensure that children can safely take this drug."
