Doctors are wary of raising expectations among families affected by cystic fibrosis – which affects 10,000 patients in Britain – after repeated claims since the gene was discovered in 1989 that a gene-therapy cure was just around the corner.
Katie Murphy, research and development officer of Cystic Fibrosis Ireland, said that, “Any increase in lung function would be considered an advance”. 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.
The goal of gene therapy for cystic fibrosis is to replace the faulty CFTR gene with a working one.
A second new trial is planned that will deliver the therapy through a specially engineered virus rather than encasing it in fat globules – the method used to date – which might give longer term benefits. It may be possible to enhance the technique in the future to boost lung function dramatically.
“A larger trial with a stratified trial entry design, powered to assess subgroups, and that addresses the mechanisms of response heterogeneity will be important to verify or refute these data”, they wrote.
Further study is needed to improve the Cystic Fibrosis gene therapy, making it more effective and testing it on more patients. This happens by having the patient inhaling molecules of DNA. The results were published Friday in the Lancet Respiratory Medicine journal.
“Our only hope was that some of the patients, on some occasions, might see some benefit”, said Dr. Eric Alton of Imperial College London, the study’s lead author.
There has been some other progress in Cystic Fibrosis treatment lately with the FDA approval for a new drug.
It is the first gene treatment to ever improve he devastating condition which causes the lungs to secrete a thick mucus, clogging up the airways and triggering life-threatening infections. Diagnosis is usually carried out through a sweat test.
“This is an extraordinary time for therapeutic development in cystic fibrosis and the need is urgent to stop so many young lives being cut short because of this cruel condition”. A high level of chloride means that the person has cystic fibrosis. This results in serious and recurring lung infections that damage the lungs and cause the vast majority of deaths among those with the disease, the researchers explained. But at least this study does offer renewed hope that better treatement might one day be available. “Although we know that the therapy isn’t ready to prescribe to patients, personally I am delighted with the result – it’s better than I expected”, says Deborah Gill at the University of Oxford, whose team designed the sequence of genetic instructions administered in the trial. Participants were randomly assigned to either 5ml of nebulised (inhaled) pGM169/GL67A (gene therapy) or saline (placebo) at monthly intervals over 1 year.