Gene therapy breakthrough for cystic fibrosis

ConclusionThis RCT showed that a new non-viral-based gene therapy for cystic fibrosis was able to produce “modest” benefits in lung function compared to a placebo. The treatments were given once a month for a year. The study had many strengths, including its double-blind randomised design, recruiting adequate numbers to demonstrate real differences between groups, and using pre-specified outcomes and sub-analysis. This means we can be confident in the reliability of the findings presented. Although the findings of this study are encouraging, there are always limitations. These include: This study was relatively small, recruiting just 140 patients. This is normal for a phase II trial, but large clinical trials are needed to fully assess the effects and safety of this treatment in development. Patients recruited in this trial had to be clinically stable to be included. This means they might be at their optimum respiratory health at this stage. Therefore, we don’t know how the treatment would work in clinically unstable or very severe patient groups.It is important to realise that both groups’ lung function got worse over the year, so the treatment as it stands is quite limited. The new gene therapy was able to lessen some of the deterioration, but not in all. Nonetheless, this gives the researchers hope and scope to work out how to improve it. Optimising the dose, working out why it worked in some people and not others, and trialling the therapy in more people are...
Source: NHS News Feed - Category: Consumer Health News Tags: Genetics/stem cells Heart/lungs Source Type: news