British scientists at the Brompton hospital and Imperial College London have just been given a £20 million grant to start clinical trials of their very promising new Cystic Fibrosis DNA nebulizer.
British researchers are to trial a new gene therapy for cystic fibrosis in which sufferers simply inhale a spray laden with DNA.
The announcement comes just days after Gordon Brown, the chancellor, and his wife Sarah announced that their four-month-old son Fraser had been diagnosed with the inherited condition.
If it works, the £20m programme could lead to a treatment that will improve and extend the lives of thousands of cystic fibrosis sufferers around the world.
The technique being tested involves the use of a nebuliser that injects a fine spray into the airways. Within the spray are tiny soap-like bubbles called liposomes containing DNA without the cystic fibrosis defect.
The liposomes fuse with the outer surfaces of cells lining the lungs and the DNA passes by natural processes into their nuclei.
Once installed, it is hoped the DNA will prompt the cells to make the essential protein lacking in cystic fibrosis sufferers.
Because of the constant replacement of cells in the body, the treatment would have to be taken repeatedly to sustain its effect.
The scientists do not expect to provide a full cure, but Alton said: “What makes this trial very novel is that we will for the first time be trying to achieve clinical improvement rather than just alleviating symptoms.”