Cystic fibrosis currently has no treatment capable of interrupting its progression. For the first time, gene therapy has succeeded in this.
First hope for treatment in cystic fibrosis. Inhaled gene therapy stabilizes the pulmonary symptoms of the disease. The results of a phase II clinical trial are published in the Lancet Respiratory Medicine. The patients inhaled for a year a solution to replace the defective genes responsible for the disease.
136 patients with cystic fibrosis over 12 years of age were included in this randomized trial. 78 of them received 5 milliliters of a solution containing DNA fragments (pGM169 / GL67A), the rest a saline solution (placebo). Participants inhaled their treatment once a month for a year. In the case of gene therapy, this made it possible to deliver corrected copies of defective genes (CFTR) directly to the surface of the lungs.
Few side effects
Researchers regularly assessed participants’ lung function by measuring the volume of air exhaled in one second. After two years, the improvement is more marked by 3.7% in the volunteers undergoing gene therapy compared to the placebo group. According to Prof. Stephen Hyde, co-author of the study, it is more the result of stabilization of cystic fibrosis than of a real improvement. But “stabilizing lung disease is a goal in itself. We are continuing other studies on non-viral gene therapies, with different doses and drug combinations and more efficient vectors. “
This is the first time that gene therapy has demonstrated its benefits in cystic fibrosis. But these good results are tempered by a very variable reaction. Patients with the worst respiratory function at the start of the clinical trial saw the effect of therapy doubled. For others, it was minimal.
The good news, however, is the good tolerance of this new option. Only two treatment breaks are to be deplored, one in each group. Six serious side effects were listed.
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