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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
New drugs in cystic fibrosis: what has changed in the last decade?
Juliana Roda1, Catarina Pinto-Silva2, Iris A I Silva3
1Pediatric Gastroenterology and Nutrition Unit, Centro Hospitalar e Universitario de Coimbra EPE Hospital Pediátrico de Coimbra, Avenida Afonso Romão 3000-602 Coimbra, Portugal.
Abstract:
Cystic fibrosis (CF), a life-limiting chronic disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene, affects more than 90,000 people worldwide. Until recently, the only available treatments were directed to symptom control, but they failed to change the course of the disease. New drugs developed in the last decade have the potential to change the expression, function, and stability of CFTR protein, targeting the basic molecular defect. The authors seek to provide an update on the new drugs, with a special focus on the most promising clinical trials that have been carried out to date. These newly approved drugs that target specific CFTR mutations are mainly divided into two main groups of CFTR modulators: potentiators and correctors. New therapies have opened the door for potentially disease-modifying, personalized treatments for patients with CF.
Insights
New cystic fibrosis (CF) drugs target the underlying CFTR gene defect, offering disease-modifying potential. These CFTR modulators represent a significant advancement in personalized treatment for CF patients.
Area of Science:
- Medical Genetics
- Pulmonology
- Pharmacology
Background:
- Cystic fibrosis (CF) is a chronic, life-limiting genetic disease affecting over 90,000 individuals globally.
- Traditional CF treatments primarily manage symptoms and do not alter the disease's progression.
- The disease stems from mutations in the cystic fibrosis transmembrane regulator (CFTR) gene.
Purpose of the Study:
- To provide an updated overview of novel therapeutic agents for cystic fibrosis.
- To highlight promising clinical trials investigating new CFTR modulators.
- To discuss the potential of these therapies to modify the disease course.
Main Methods:
- Review of recent pharmaceutical developments targeting the CFTR protein.
- Analysis of clinical trial data for emerging CFTR modulators.
- Categorization of new drugs into potentiators and correctors.
Main Results:
- Development of new drugs that modulate CFTR protein expression, function, and stability.
- Identification of two main classes of CFTR modulators: potentiators and correctors.
- Demonstration of these drugs targeting specific CFTR mutations.
Conclusions:
- New CFTR modulator therapies offer the potential for disease modification in cystic fibrosis.
- These targeted treatments represent a shift towards personalized medicine for CF patients.
- Advancements in drug development hold promise for improving long-term outcomes in CF.
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