Children with an incurable genetic disorder that causes dramatically premature ageing are to begin treatment with a transplant drug within months in a trial that promises to deliver the first effective treatment for the condition.
Scientists have discovered that a drug called rapamycin can roll back the effects of Hutchinson-Gilford progeria in human cells, paving the way for a similar drug to be given to patients on an experimental basis. The extremely rare genetic condition causes the symptoms of very old age to begin in the first years of childhood. Affected patients usually die in their early teens.
Francis Collins, director of the US National Institutes of Health, who led the research, told The Times he hoped that the trial could begin as early