Current Pharmaceutical Design - Volume 18, Issue 5, 2012

Cystic fibrosis (CF) is an inherited disease of impaired epithelial transmembrane ion transport which is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Conventional treatment strategies for CF lung disease, the major contributor to morbidity and mortality in patients with CF, are predominantly symptomatic and do not address the underlying defects. A better understanding of the Read More

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the under Read More

These are exciting times with the appearance of small molecule compounds in clinical trials which target the basic defects caused by mutation in the CFTR gene. This progress was enabled by years of basic research probing the molecular and cellular consequences caused by mutation and the development of methods by which to study the primary anion transport defect in a high-throughput manner by robotics. Future progress Read More

Although the development of gene therapy for cystic fibrosis (CF) was high priority for many groups in academia and industry in the first 10 to 15 years after cloning the gene, more recently active research into CF gene therapy is only being performed by a small number of committed, mainly academic, groups. However, despite the waning enthusiasm, which is largely due to the realisation that gene transfer into lungs is more Read More

New therapeutic strategies are targeting correction of the basic defect in cystic fibrosis (CF) disease. In fact, completion of the first successful clinical drug trials now signals the start of a new era in CF therapy. Many promising drug candidates are emerging into the clinical drug pipeline. However, their translation from the bench to the bed side is challenged by the lack of accurate and reliable biomarker assays that allow Read More

Since the discovery of the CFTR gene mutations which cause cystic fibrosis (CF) in 1989 the average life expectancy of CF patients has almost doubled and now exceeds 37 years. The advances in molecular diagnostics and medical treatments expanded beyond the CF patient population as some of the newest treatments are also being tested for treatment of complex diseases such as COPD and other inherited disorders. Ra Read More

The field of inhaled antibiotics that target Pseudomonas aeruginosa infections has made substantial contributions to the health, disease management, and life expectancies of individuals with cystic fibrosis (CF) over the last three decades [1-3]. This paper reviews some of the recent clinical developments in the field of inhaled antibiotics for CF and briefly describes formulations and ongoing developments for US and/o Read More

The development of antimicrobial treatments for respiratory pathogens in cystic fibrosis (CF) has been an integral component to the increased survival of CF patients over the past fifty years. Despite significant treatment advances, however, respiratory failure secondary to chronic bacterial pulmonary infection remains the primary cause of death in CF patients. The purpose of this review is to discuss emerging pathogens (ot Read More

The concentrations of nitric oxide are decreased in airways of patients with cystic fibrosis. The reasons for this nitric oxide deficiency are incompletely understood but may include reduced production from nitric oxide synthases due to decreased expression, the enzymes in airway epithelial cells, reduced availability of L-arginine, the substrate for nitric oxide synthases, and the presence of endogenous inhibitors of t Read More

Lung transplantation has evolved as an accepted therapy in selected adults and children with end-stage lung disease. Outcomes following lung transplantation have improved in the recent era with a 5-year survival of >70% and an overall good functional status of surviving recipients. Many of the advances have been achieved by the use of modern immunosuppressive agents. To date, multiple strategies exist that may be e Read More