Cystic Fibrosis treatment has a recent breakthrough. A gene therapy has been administed to a number of patients on a double blind, and results show that it stabilizes the patients' lungs.
According to the BBC News, the clogged lungs of the 136 patients showed no decline after inhaling healthy copies of the gene. The therapy has been administered once a month and for a year. It is reported that patients with most clogged lungs before the trial have shown a 3% improvement on their lung condition, whereas those who were not given the therapy have reported an average of 3 to 4 % decline in that same span of time as the trial.
Prof Eric Alton, of Imperial College London, has been the team leader of the trial. However, in a statement, he warned that the therapy could not go straight to the clinic yet because the result was just modest and variable. He said that he together with his colleauge hoped to move for a further study next year. The result is published in Lancet Respiratory Medicine show.
New Scientist explained in its article that Cystic Fibrosis (sometimes referred to as mucoviscidosis) is the most common genetic disease. It has been affecting over 70,000 people worldwide. The common indications are CFTR, mutations in a single gene, and clog lungs with thick mucus. Most often than not, the lung function steadily deteriorates in spite of taxing physiotherapy to clear it.
For quite sometime, it has been reported that researchers have tried long enough to put healthy copies of CFTR (Cystic fibrosis transmembrane conductance regulator) into damaged lung cells, however the patients' immune system's defences have sabotaged all efforts.
But then a research group of Prof Eric Alton has cracked the code and succeded in using CFTR-based gene therapy to treat CF patients. Fierce BioTech Research that the research consortium has been working with the liposomes, and has gotten a small yet noteworthy development in the patients' lung function.
