Related Experiment Video
Updated: Dec 8, 2025
08:00
Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
11.0K
Drug development for cystic fibrosis.
Don B Sanders1, James F Chmiel1
1Division of Pediatric Pulmonology, Allergy and Sleep Medicine, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at IU Health, Indianapolis, Indiana, USA.
Pediatric Pulmonology
|September 17, 2020
Summary
Drug development for cystic fibrosis (CF) has advanced significantly, increasing survival rates. Innovative approaches are needed to further improve therapies and achieve a cure for CF.
Area of Science:
- Medical Research
- Pharmacology
- Pulmonology
Background:
- The first drug approval for cystic fibrosis (CF) was in 1993, leading to increased median survival from 30 to 44.4 years.
- Effective modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) now benefit 90% of eligible CF patients in the US and Europe.
- Advances in CF therapies have improved patient longevity and reduced morbidity, but also increased care complexity.
Purpose of the Study:
- To provide an overview of the drug development process for cystic fibrosis, from preclinical stages to Phase IV.
- To highlight special considerations for CF drug development.
- To emphasize the need for innovative approaches in CF drug development.
Main Methods:
- Review of existing literature on drug development phases (preclinical to Phase IV).
- Integration of specific considerations relevant to cystic fibrosis care and treatment.
- Analysis of the evolving landscape of CF drug development.
Main Results:
- Significant progress in CF drug development has extended patient survival and improved quality of life.
- The availability of CFTR modulators represents a major therapeutic advancement.
- Challenges remain in detecting meaningful outcome changes in healthier CF populations.
Conclusions:
- Continued evolution of drug development strategies is crucial for advancing CF care.
- Innovative approaches are necessary to overcome challenges in detecting treatment efficacy.
- The ultimate goal remains the development of a one-time cure for all individuals with cystic fibrosis.
Related Concept Videos
01:24Cystic Fibrosis: Management
333
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...
Sinus disease and chronic...
333
01:23Cystic Fibrosis: Pathogenesis
601
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,...
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,...
601
01:28Preclinical Development: Overview
5.6K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
5.6K
01:11Clinical Trials: Overview
4.4K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
4.4K
01:19Antiasthma Drugs: Leukotriene Modifiers
1.6K
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:
Leukotriene modifiers work through two distinct mechanisms:
1.6K
01:27Chronic Obstructive Pulmonary Disease-IV: Assessement and Diagnostic Studies
2.9K
Assessing and diagnosing Chronic Obstructive Pulmonary Disease (COPD) involves a detailed approach that includes a comprehensive review of medical history, physical examination, and a variety of diagnostic tests. This thorough evaluation is essential to ensure an accurate diagnosis and guide effective management strategies.
Medical History
Medical History
2.9K