Cystic Fibrosis can be a debilitating medical condition, and there is no cure. Meanwhile, those who did not take the gene therapy actually saw their lungs worsen by an average of 3 to 4 percent over the same period of time.
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A consortium of scientists at the Imperial College London, Oxford, the University of Edinburgh and National Health Service clinics, conducted the study.? Cystic fibrosis clogs the airways making it vulnerable to recurrent infections of a life threatening nature. Gene therapy is expected to cost up to $1 million for a treatment.
Murphy also reported that patients suffer a 2% drop in lung function per year as the illness progresses.
Even though the trial displayed significant results in lung functions of the patients, the researchers say that more study is required to examine the effectiveness and to better the therapy before it is deemed suitable for official clinical use of patients. While that may not sound like much, the researchers were happy to know that their technique worked at all.
“We are looking to undertake follow-up studies assessing higher, more frequent doses as well as combinations with other treatments”. Still, the study researchers feel that the results are quite positive. She added, “To see a 3.7 per cent increase in lung function is considered a promising finding”. “The sweat test is painless”, the Cystic Fibrosis Foundation reports. Respiratory distress in CF, defined as acute difficulty in breathing, infection and/or hospitalization, is most commonly related to lung infection and inflammation induced lung tissue damage attributable to an overwhelming and dysfunctional response by dysregulated neutrophils. The drugs target the molecular abnormalities of Cystic Fibrosis, instead of just treating the resulting symptoms.
It has been said that nearly ten thousand people suffering from the cystic fibrosis have got a new hope after a gene therapy was shown to reverse the condition. Those with Cystic Fibrosis are faced with a life of antibiotics and therapy, and face an average age of death of around forty years old. Diagnosis is usually carried out through a sweat test.
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Alton further informed that scientists have already started developing a second therapy that would be using a safe virus for delivering the gene into cells.
