Related Experiment Video

Updated: Jun 20, 2026

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
07:04

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

Emerging treatments in cystic fibrosis

Andrew M Jones1, Jennifer M Helm

  • 1Manchester Adult Cystic Fibrosis Centre and The University of Manchester, University Hospitals South Manchester NHS Trust, Manchester, England. andmarkj@hotmail.com

Drugs
|September 15, 2009
PubMed

Insights

New cystic fibrosis (CF) drugs are in clinical trials, including inhaled antibacterials, anti-inflammatory agents, and ion channel modulators. Gene therapies targeting the CF transmembrane regulator (CFTR) gene offer promising future treatments for CF.

Area of Science:

  • Pulmonary Medicine
  • Pharmacology
  • Genetics

Background:

  • Cystic Fibrosis (CF) is a genetic disorder requiring innovative therapeutic strategies.
  • Current CF treatments focus on symptom management, but new drug development is crucial.

Purpose of the Study:

  • To review potential drug treatments for cystic fibrosis (CF) currently in clinical studies.
  • To highlight challenges in assessing the efficacy of novel CF therapies.

Main Methods:

  • Review of ongoing clinical trials for various drug classes targeting CF.
  • Categorization of potential CF therapeutics by mechanism of action.

Main Results:

  • Multiple inhaled antibacterials (e.g., colistin, tobramycin) are in development.
  • Anti-inflammatory agents (e.g., sildenafil, acetylcysteine) and ion channel modulators are under investigation.
  • Gene-based therapies (e.g., CFTR correctors, gene transfer) show significant promise.

Conclusions:

  • A diverse range of novel therapeutic agents are progressing through clinical evaluation for CF.
  • Successful development of these treatments could offer new categories of therapeutic options for CF patients.
  • Addressing challenges in clinical trial design is essential for accurate efficacy assessment.

More Related Videos

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study08:00

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

Nasal Potential Difference to Quantify Trans-epithelial Ion Transport in Mice08:55

Nasal Potential Difference to Quantify Trans-epithelial Ion Transport in Mice

Published on: July 4, 2018

Related Concept Videos

Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...
Cystic Fibrosis: Pathogenesis01:23

Cystic Fibrosis: Pathogenesis

Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Drugs for Treatment of Constipation-Predominant IBS01:21

Drugs for Treatment of Constipation-Predominant IBS

Pharmacological therapies for IBS-C are designed to alleviate abdominal discomfort and enhance bowel function. In patients with IBS-C, fiber supplements may help soften stools and decrease straining, but may also lead to increased gas production and bloating. Osmotic laxatives like milk of magnesia are frequently used to soften stools and increase stool frequency in IBS-C patients. In addition, two drugs approved for use in severe IBS-C adult cases are linaclotide (Linzess) and lubiprostone...
Acute Respiratory Failure-V01:29

Acute Respiratory Failure-V

The treatment for acute respiratory failure varies based on factors like the underlying cause, overall health, and severity. A collaborative healthcare team is essential for early detection, often through arterial blood gas analysis. Identifying the cause is the primary goal, with treatment strategies adjusted for ventilation/perfusion (V/Q) mismatch, shunting, or diffusion impairment.
Ensure that patients are monitored continuously for their response to therapy, including changes in...
Chronic Pancreatitis II: Collaborative Care01:29

Chronic Pancreatitis II: Collaborative Care

The management of chronic pancreatitis is multifaceted, involving a comprehensive approach that includes thorough assessment, diagnostic testing, and a variety of management strategies.
Assessment:
Antiasthma Drugs: Leukotriene Modifiers01:19

Antiasthma Drugs: Leukotriene Modifiers

Leukotriene modifiers, or cysteinyl leukotriene receptor antagonists, are medications used to manage chronic asthma. These agents target specific inflammatory mediators produced during arachidonic acid metabolism, an essential process in generating inflammation in the body.
Leukotriene modifiers work through two distinct mechanisms: