Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Abstract

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research organization that evaluates medical evidence and convenes public deliberative bodies to help stakeholders interpret and apply evidence to improve patient outcomes and control costs. Through all its work, ICER seeks to help create a future in which collaborative efforts to move evidence into action provide the foundation for a more effective, efficient, and just health care system. More information about ICER is available at http://www.icer-review.org. The funding for this report comes from government grants and non-profit foundations, with the largest single funder being the Laura and John Arnold Foundation. No funding for this work comes from health insurers, pharmacy benefit managers, or life science companies. ICER receives approximately 20% of its overall revenue from these health industry organizations to run a separate Policy Summit program, with funding approximately equally split between insurers/PBMs and life science companies. For a complete list of funders and for more information on ICER's support, please visit http://www.icer-review.org/about/support/.

Potential targeted therapy based on deep insight into the relationship between the pulmonary microbiota and immune regulation in lung fibrosis

“…In this case, lung 3D cultures, including precision-cut lung slices (PCLS) and hydrogels, have emerged as valuable tools for drug discovery of IPF (125)(126)(127). For instance, a new study demonstrated PCLS maintains the native lung microbiota and is a relevant in vitro model to study lung fibrosis and drug testing in a diseased tissue condition (128). We hope that more prospective studies will be conducted to evaluate the efficacy and safety of PCLS in treating IPF.…”

Cystic Fibrosis: Recent Insights into Inhaled Antibiotic Treatment and Future Perspectives

“…Changes in antimicrobial treatment or in continuous antibiotic regimens are also made to prevent and reduce deterioration in lung function before the insurgence of pulmonary exacerbations [8,15,16]. Although the recent introduction of novel therapies targeting the defective CF transmembrane conductance regulator (CFTR) protein has transformed the landscape of CF [17,18], the antibiotic treatment of respiratory infections will remain the mainstay of CF therapy for the foreseeable future [19,20]. Accordingly, new treatment strategies for better management of acute and chronic episodes have been at the forefront of efforts to improve outcomes in CF.…”

Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing

“…30 Although there is no cure for cystic fibrosis, symptom-based therapies (such as antibiotics, bronchodilators, and mucus thinning medications) and CFTR modulating drugs have become the first-line treatments to relieve symptoms and reduce the risk of complications. 31 Currently, gene manipulation technologies and molecular targets are also being explored. The use of CRISPR/Cas-9 technology for genome editing has great potential, although it is in the early stages of development.…”