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Progeria could be treated with existing drug

07 November 2011

By Oliver Timmis

Appeared in BioNews 632

Progeria, an extremely rare genetic disease that is commonly used as model for ageing, could be treated with an existing drug.

A team at Durham University, led by Professor Christopher Hutchinson, found that levels of reactive oxygen species (ROS) were five to ten times greater in progeria patients than normal. They treated progeria cell models with N-acetyl cysteine (NAC), a drug known to have anti-oxidant properties, and found a reduction of ROS to a baseline level.

The researchers believe the drug soaked up the reactive oxygen, and reported that DNA damage in the cells then returned to 'approximately normal levels'.

Progeria is a disease that causes children to age eight times faster than normal. Patients are unlikely to live beyond their early teens, with heart disease usually being the cause of death.

ROS have also been implicated as having an important role in the normal ageing process. Reduction of ROS in healthy, older cells could potentially help to avoid some of the effects of ageing.

'If you give a child a drug which extends lifespan without adverse effects then you would expect these to be useful in an older population', explained Professor Hutchinson.

NAC, already used to treat aspirin overdoses and to thin the mucus of cystic fibrosis patients, can be sold without prescription as a dietary supplement. However, there have not been any trials of NAC in progeria patients and, given how rare the condition is (there are only 78 diagnosed patients worldwide), a full clinical trial will be near impossible.

Professor James Goodwin, head of research at Age UK, said the research offered 'valuable insights into the ageing process which have the potential to improve later life'.

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