An experimental cancer drug prevents, and even reverses, potentially fatal heart and blood vessel damage in mice with a model of progeria, the inherited disorder that causes the most dramatic form of premature ageing in humans.
There is no cure for progeria, which affects about one child in 4 million. Children with progeria look normal at birth, but they grow slowly and quickly show signs of ageing, such as hair loss, dry skin and loss of body fat. The most serious damage is to the blood vessels: the children develop heart and blood vessel disease and usually die of a heart attack or stroke at about 13.
Based on earlier experiments in cells and mice, a team at the US National Institutes of Health tested an experimental cancer drug, tipifarnib, in genetically modified mice that develop blood vessel damage like that of children with progeria.
Tipifarnib belongs to a group of drugs called farnesyltransferase inhibitors (FTIs), which have been tested in patients with myeloid leukaemia, neurofibromatosis and other conditions. The team had already found that FTIs reverse structural abnormalities in skin cells taken from progeria patients and grown in the lab.
The results were good: as well as preventing heart and blood vessel disease, the drug reversed damage that had already developed in the mice. The scientists stress that more studies are needed to confirm whether FTIs can prevent and treat this disease in children with progeria. Another study suggested that combining statins with bisphosphonates slows the visible signs of progeria and extends the lives of lab animals.
Although few children have progeria, studying it helps us understand ageing in other people: the faulty protein responsible for progeria is found in small amounts in everyone and may play a part in normal ageing.
Update: in 2020 the US approved lonafarnib, another drug of this group, as the first treatment for progeria.
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