RTÉ News: Cystic Fibrosis discovery may help sufferers

Gene therapy attacks the basic flaw of cystic fibrosis and owns the potential to reduce the classic and low-in-performance routine of medication for sufferers.

For those who don’t know: cystic fibrosis is an inherited health disorder caused by a faulty gene.

However, doctors are wary of raising hopes after repeated claims since the gene was discovered in 1989 that a cure was imminent.

The scientists are also developing a second therapy that uses a safe virus to deliver the DNA into cells. One inhaled, these molecules then deliver a normal working copy of the gene to lung cells. It may be possible to enhance the technique in the future to boost lung function dramatically. Gene therapy has proven to be an extremely challenging field. Further clinical research is now needed before we can say that it is a viable clinical therapy but it is encouraging that a proof of concept has been established. The research was published online Thursday in the journal, Lancet Respiratory Medicine.

This story was widely covered by United Kingdom media.

However he stressed that even though “a whole swathe” of patients improved, the treatment was not yet ready for use in clinics.

Despite frequent physiotherapy to remove the mucus, lung infections are common among patients with cystic fibrosis and lead to 90% of deaths from the disease. The treatment is still in the early stages of development and has been tested on only a few patients. These mutations make the lining of the lungs secrete unusually thick mucus. If the lungs become too damaged, the patient may need a lung transplant. Those who did best were those with the worst lung function.

The results of the trial were very much appreciated by the Cystic Fibrosis Ireland, a body that represents the interests of those with the life-threatening lung disease. “It is vital we are at the forefront of turning scientific discoveries into tangible benefits and affordable treatments for NHS patients”.

The Great britain Cystic Fibrosis Gene Therapy Consortium was at those performed the rigors and realized typically around three.seven percent spike in lungs among individuals. Most people with CF die from lung-related problems. There were 116 patients (83%) completing the treatment and included in the per protocol analysis. This excludes those who had dropped out for any reason. Most people are afflicted by repiratory tract maladies. Per protocol analysis gives a good idea of whether the medicine works in those who took it as intended.

But Miss Lloyd said he had already benefited from drugs developed as a result of other people taking part in previous trials. Both groups were followed for one year. Alton said that while some children reported feeling better, others didn’t feel any difference.

A total of six serious adverse events were recorded from the pGM169/GL67A group. Researchers caution that the effects shown through the data were inconsistent.

London: A therapy that repairs a mutated gene causing cystic fibrosis worked well enough in a United Kingdom trial to move ahead with follow-up testing, which could help bring forth a new treatment for the deadly lung disease.

“For the first time in the world we have seen a significant benefit in giving gene therapy to cystic fibrosis patients”, said Professor Eric Alton, the co-ordinator of the United Kingdom consortium of universities and hospitals that carried out the trial. The treatments were given once a month for a year.

This new study uses a different approach.

One expert in the United States was encouraged by the findings.

There has been some other progress in Cystic Fibrosis treatment lately with the FDA approval for a new drug.