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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Condition:   Cystic Fibrosis Intervention:   Other: expiratory muscle training Sponsors:   Cigdem Emirza;   Istanbul University Enrolling by invitation
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
AbstractPseudomonas aeruginosa (Pa) detection in the paranasal sinuses may help to prevent or postpone bacterial aspiration to the lower airways (LAW) and chronic lung infection in cystic fibrosis (CF). We assessed the ability of an ELISA test for measurement of specificPa secretory IgA (sIgA) in saliva (a potential marker of sinus colonization) to early detect changes in thePa LAW status (indicated by microbiological sputum or cough swab culture and specific serum IgG levels) of 65 patients for three  years, in different investigation scenarios. Increased sIgA levels were detected in saliva up to 22 months befor...
Source: Medical Microbiology and Immunology - Category: Microbiology Source Type: research
Publication date: Available online 24 January 2019Source: The Lancet Respiratory MedicineAuthor(s): John J McNamara, Susanna A McColley, Gautham Marigowda, Fang Liu, Simon Tian, Caroline A Owen, David Stiles, Chonghua Li, David Waltz, Linda T Wang, Gregory S SawickiSummaryBackgroundThe efficacy, safety, and tolerability of lumacaftor and ivacaftor are established in patients aged 6 years and older with cystic fibrosis, homozygous for the F508del-CFTR mutation. We assessed the safety, pharmacokinetics, pharmacodynamics, and efficacy of lumacaftor and ivacaftor in children aged 2–5 years.MethodsIn this multicentre, pha...
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Abstract BACKGROUND: Cystic fibrosis (CF) is the commonest inherited life-shortening illness in white populations, caused by a mutation in the gene that codes for the cystic fibrosis transmembrane regulator protein (CFTR), which functions as a salt transporter. This mutation mainly affects the airways where excess salt absorption dehydrates the airway lining leading to impaired mucociliary clearance. Consequently, thick, sticky mucus accumulates making the airway prone to chronic infection and progressive inflammation; respiratory failure often ensues. Other complications include malnutrition, diabetes and subfert...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Clearance of intrapulmonary mucus by the high-velocity airflow generated by cough is the major rescue clearance mechanism in subjects with mucoobstructive diseases and failed cilial-dependent mucus clearance, e.g., subjects with cystic fibrosis (CF) or chronic obstructive pulmonary disease (COPD). Previous studies have investigated the mechanical forces generated at airway surfaces...
Source: Proceedings of the National Academy of Sciences - Category: Science Authors: Tags: Biological Sciences Source Type: research
Conclusion: The CF lower airway microbiome is altered early in life
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Cystic fibrosis Source Type: research
Conclusion: In our data, post TB bronchiectasis remained the most common cause of non-CF bronchiectasis, Pseudomonas is the most common pathogen, obstructive pattern of spirometry is found in majority and hemoptysis is the most frequent complication.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Respiratory infections Source Type: research
Conclusion: This is the first study that shows secretion of A. fumigatus in a CF patient during coughing.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Cystic fibrosis Source Type: research
Conclusion: Br is especially important for pediatricians in our country. While infections in Br etiology have an important place, development of new tests and increased scans are leading to an increase in ID and PSD. The results of our study showed that this is the case in our region. ID and PSD should be checked in cases followed by diagnosis of bronchiectasis.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Paediatric respiratory epidemiology Source Type: research
Purpose: Limited number of studies exist investigating the effects of inspiratory muscle training (IMT) in bronchiectasis. This randomized controlled study was aimed to examine the impact of high-intensity IMT (H-IMT) on exercise capacity in patients with non-cystic fibrosis bronchiectasis.Methods: Forty-five patients with bronchiectasis participated in this study. Lung function, respiratory muscle strength and endurance, exercise capacity (incremental shuttle walk test, ISWT), dyspnea (MMRC), and quality of life (Leicester Cough Questionnaire, LCQ) were evaluated. The H-IMT was applied using a hand-held threshold loading ...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Physiotherapists Source Type: research
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