Research supported by the Cystic Fibrosis Foundation has added decades to the lives of people with cystic fibrosis.
From the creation of our innovative drug discovery program to the development of the world's largest CF clinical trial network, the CF Foundation has spurred industry to discover and develop new drugs for CF and expedited their testing and eventual approval. Because of the Foundation's commitment to fund research that improves our understanding of the disease, more treatments are in development, and drugs that have been approved for other diseases are now being used in new ways to treat CF.
Therapeutic Development Program
By providing funds to fuel CF-specific therapeutic development programs, the CF Foundation has been able to recruit biotech and pharmaceutical companies to invest in rare-disease research.
Nearly every approved CF drug available today was made possible with financial assistance and/or support from the Foundation's clinical trial network. Those drugs include:
- Dornase alfa (Pulmozyme®), an inhaled medication that breaks down deoxyribonucleic acid (DNA) from bacteria and white blood cells in the lungs, thinning the mucus and making it easier to clear out the lungs
- Three antibiotics used to treat lung infections caused by the bacteria Pseudomonas aeruginosa: inhaled tobramycin (TOBI®), tobramycin inhaled powder (TOBI® Podhaler™), and aztreonam for inhalation solution (Cayston®)
- Three CFTR modulators that treat the underlying defect of CF: ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), and tezacaftor/ivacaftor (Symdeko™)
- Gastrointestinal therapies: the oral antioxidant vitamin, AquADEKs®; pancrelipase enzyme products; and the digestive enzyme cartridge for tube feeding, RELiZORB®
Each new innovation brings us a step closer to our ultimate goal: a cure for all people with CF.
Research Discoveries
The Foundation has supported or collaborated with many scientists who have been instrumental in pioneering groundbreaking research in CF. Discoveries the Foundation helped support include:
- Identification of the cystic fibrosis transmembrane regulator (CFTR) gene and its CFTR protein product (1989)
- Proof of concept that restoring defective CFTR activity (in the laboratory) is possible (1990)
- Creation of animal models to study CF disease and therapies (1993-2015)
- High doses of ibuprofen reduce lung inflammation in people with CF (1995)
- The sequencing of the entire DNA of the bacteria P. aeruginosa, the most common cause of CF lung infections (2000)
- The antibiotic azithromycin improves CF lung health (2002)
- The three-dimensional structure of a portion of the CFTR protein, opening the door to screen for new ways to identify drugs that could directly target CFTR (2003)
- Inhaled hypertonic saline helps clear CF mucus and improve lung health (2004)
- The discovery, development, and approval of ivacaftor (2012), lumacaftor/ivacaftor (2015), and tezacaftor/ivacaftor (2018)
- Identification of more than 1,700 CF gene mutations, or variants, worldwide (1989-2016)
Patient Registry
The Foundation provides significant resources and financial support to maintain the CF Foundation Patient Registry. Since its creation in 1966, the Registry has been a model for setting international standards for the collection and analysis of patient data to build the knowledge that leads to improved health outcomes. The Registry has also served as a template for other nonprofit health organizations, including CF patient advocacy groups in other countries.
Information from the Registry is used to develop and continuously improve specialized CF clinical care by allowing care teams to partner with people with CF and their families in creating treatment plans, guiding quality improvement initiatives, and developing CF clinical care guidelines. Researchers use the Registry to study CF treatments and outcomes and to design CF clinical trials.
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Reference to any specific product, process, or service does not necessarily constitute or imply its endorsement, recommendation, or favoring by the Cystic Fibrosis Foundation. The appearance of external hyperlinks does not constitute endorsement by the Cystic Fibrosis Foundation of the linked websites, or the information, products, or services contained therein.
Information contained on this site does not cover all possible uses, actions, precautions, side effects, or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
FDA-approved drug information is available at dailymed.nlm.nih.gov/dailymed.