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

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Conclusions: Patients with CF cough more than patients with PCD. The cough frequency in CF and PCD is associated with parameters reflecting disease severity. Cough frequency is a possible endpoint in clinical trials and cough epochs/h may be more useful than coughs/h.Respiration
Source: Respiration - Category: Respiratory Medicine Source Type: research
CONCLUSION: UI prevalence seemed higher on population with OLD, to a greater degree of severity. Quality of life self-evaluation was further decreased when UI was associated with pulmonary disease. PMID: 30366709 [PubMed - as supplied by publisher]
Source: Progres en Urologie - Category: Urology & Nephrology Tags: Prog Urol Source Type: research
(University of Gothenburg) As a cold ends, a severe mucus cough starts. Sound familiar? Two studies now give explanations: First, crucial mechanisms of the mucus in both diseased and healthy airways; second, what happens in such chronic lung diseases as cystic fibrosis and chronic obstructive pulmonary disease (COPD).
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
CONCLUSION: This report raises concerns regarding the spread and severity of B. cepacia infection in non-compromised patients in the community and the need to suspect and identify it. Since the organism is inherently resistant to antipseudomonal penicillins, aminoglycosides and polymyxin B, differentiation from Pseudomonas spp. and determining antimicrobial susceptibility is paramount for treatment . PMID: 30345929 [PubMed - as supplied by publisher]
Source: Infectious Disorders Drug Targets - Category: Infectious Diseases Authors: Tags: Infect Disord Drug Targets Source Type: research
Purpose of review Pulmonary exacerbations are described as worsening of the daily symptoms of cystic fibrosis airways disease, typically with increased cough and sputum production. There are often associated signs such as weight loss and reduced lung function. These events occur frequently and are associated with considerable cost and morbidity. Although approved maintenance therapies are shown to reduce exacerbations, they still occur and are associated with poor outcomes despite treatment. Guidelines to define best practices found a paucity of evidence upon which to base recommendations. Recent findings There are on...
Source: Current Opinion in Pulmonary Medicine - Category: Respiratory Medicine Tags: CYSTIC FIBROSIS: Edited by Harry G.M. Heijerman and Nick Simmonds Source Type: research
CONCLUSIONS: Regular use of nebulised hypertonic saline by adults and children over the age of 12 years with CF results in an improvement in lung function after four weeks (very low-quality evidence from three trials), but this was not sustained at 48 weeks (low-quality evidence from one trial). The review did show that nebulised hypertonic saline reduced the frequency of pulmonary exacerbations (although we found insufficient evidence for this outcome in children under six years of age) and may have a small effect on improvement in quality of life in adults.Evidence from one small cross-over trial in children indicates th...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
CONCLUSIONS: This review, limited to a single, well-designed randomized controlled study, shows no clear evidence to support the routine use of bronchoalveolar lavage for the diagnosis and management of pulmonary infection in pre-school children with cystic fibrosis compared to the standard practice of providing treatment based on results of oropharyngeal culture and clinical symptoms. No evidence was available for adult and adolescent populations. PMID: 30221745 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
ConclusionsThis case illustrates the importance of respiratory syncytial virus infection in an immunocompetent child. Pediatricians need to have a high index of suspicion and knowledge of recurrent symptoms associated with severe damage of the lung epithelium to establish the correct diagnosis.
Source: Journal of Medical Case Reports - Category: General Medicine Source Type: research
Biopsies and bronchoscopies are the gold standard for diagnosing lung diseases, including pneumonia and cancer. However, these procedures are difficult to provide, requiring general anesthesia and an operating room. Deton hopes to simplify the proces...
Source: Medgadget - Category: Medical Devices Authors: Tags: Diagnostics Exclusive Medicine Oncology Pathology Thoracic Surgery Source Type: blogs
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
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