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 mor...
Source: NHS News Feed - Category: Consumer Health News Tags: Genetics/stem cells Heart/lungs Source Type: news

Related Links:

Abstract Once neglected in research and underappreciated in practice, there is renewed interest in bronchiectasis unrelated to cystic fibrosis. Bronchiectasis is a chronic lung disease characterised by chronic cough, sputum production and recurrent pulmonary exacerbations. It is diagnosed radiologically on high resolution computed tomography chest scan by bronchial dilatation (wider than the accompanying artery). The causes of bronchiectasis are diverse and include previous respiratory tract infections, chronic obstructive pulmonary disease, asthma, immunodeficiency and connective tissue diseases. A large proporti...
Source: Med J Aust - Category: General Medicine Authors: Tags: Med J Aust Source Type: research
The nervous system mediates key airway protective behaviors, including cough, mucus secretion, and airway smooth muscle contraction. Thus, its involvement and potential involvement in several airway diseases h...
Source: Respiratory Research - Category: Respiratory Medicine Authors: Tags: Review Source Type: research
CONCLUSION: These results demonstrated the high frequency of M. fortuitum in respiratory samples and that this bacterium causes transient infection or colonization in patients with underlying pulmonary conditions, such as cystic fibrosis and cigarette smoking-induced. Additionally, it appears that infection with M. fortuitum is particularly common and may be important in patients with HIV. PMID: 30052175 [PubMed - as supplied by publisher]
Source: Journal of Medical Microbiology - Category: Microbiology Authors: Tags: J Med Microbiol Source Type: research
The objective of this review is to provide an overview of NCFB and critically evaluate the evidence supporting the outcome measures used in recent clinical trials of inhaled antibiotics. These include quantitative changes in bacterial load, sputum purulence and yield, inflammatory markers, and lung function, as well as clinical changes in exacerbations, exacerbation frequency, hospitalizations, and health-related quality of life. Expert commentary: Recently completed large trials of inhaled antibiotics in NCFB did not consistently meet pre-specified endpoints, suggesting that we have not yet found the best enrollment crite...
Source: Expert Review of Respiratory Medicine - Category: Respiratory Medicine Tags: Expert Rev Respir Med Source Type: research
Publication date: July 2018Source: The Lancet Respiratory Medicine, Volume 6, Issue 7Author(s): Margaret Rosenfeld, Claire E Wainwright, Mark Higgins, Linda T Wang, Charlotte McKee, Daniel Campbell, Simon Tian, Jennifer Schneider, Steve Cunningham, Jane C Davies, Margaret Rosenfeld, Claire E. Wainwright, Mark Higgins, Linda T. Wang, Charlotte McKee, Daniel Campbell, Simon Tian, Jennifer Schneider, Steve Cunningham, Jane C. DaviesSummaryBackgroundIvacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Publication date: June 2018Source: The Lancet Respiratory Medicine, Volume 6, Issue 6Author(s): André Schultz, Daan Caudri
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
This study is registered with the UK Clinical Research Network (14615) and with the International Standard Randomised Controlled Trial Network Registry (12473810).FindingsBetween Jan 23, 2012, and July 4, 2017, 124 patients were prospectively recruited to the trial and had 200 sputum induction procedures for stage 1. 167 (84%) procedures were successful and the procedure was well tolerated. Of the 167 paired samples, 63 (38%) sputum-induction samples were pathogen positive compared with 24 (14%) cough swabs (p
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Publication date: July 2018Source: The Lancet Respiratory Medicine, Volume 6, Issue 7Author(s): Margaret Rosenfeld, Claire E Wainwright, Mark Higgins, Linda T Wang, Charlotte McKee, Daniel Campbell, Simon Tian, Jennifer Schneider, Steve Cunningham, Jane C Davies, Margaret Rosenfeld, Claire E. Wainwright, Mark Higgins, Linda T. Wang, Charlotte McKee, Daniel Campbell, Simon Tian, Jennifer Schneider, Steve Cunningham, Jane C. DaviesSummaryBackgroundIvacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Publication date: June 2018Source: The Lancet Respiratory Medicine, Volume 6, Issue 6Author(s): André Schultz, Daan Caudri
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
This study is registered with the UK Clinical Research Network (14615) and with the International Standard Randomised Controlled Trial Network Registry (12473810).FindingsBetween Jan 23, 2012, and July 4, 2017, 124 patients were prospectively recruited to the trial and had 200 sputum induction procedures for stage 1. 167 (84%) procedures were successful and the procedure was well tolerated. Of the 167 paired samples, 63 (38%) sputum-induction samples were pathogen positive compared with 24 (14%) cough swabs (p
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
More News: Bronchoscopy | Cardiology | Child Development | Children | Clinical Trials | Cough | CT Scan | Cystic Fibrosis | Gene Therapy | Genetics | Health | Heart | Heart Transplant | Lung Transplant | Medical Ethics | National Institute for Health Research (NIHR) | Oxford University | PET Scan | Physiotherapy | Respiratory Medicine | Science | Statistics | Stem Cell Therapy | Stem Cells | Study | Transplant Surgery | Transplants