When a group of parents started the Cystic Fibrosis Foundation in 1955, there were no treatments for cystic fibrosis. These parents set their sights high, to:
- Advance understanding of this little-known disease
- Create new treatments and specialized care for their children
- Find a cure
In the following years, the fundraising and commitment of the CF community has enabled the Foundation to support fundamental research in the laboratory that has led to groundbreaking discoveries, including identifying the gene and protein responsible for cystic fibrosis. By expanding our knowledge of the underlying biology of the disease and its effect on the body, researchers have paved the way for creating new treatments.
The Foundation's steadfast commitment to advancing CF research has helped enable more than a dozen new treatments for the disease. We have made incredible progress, including the approvals by the U.S. Food and Drug Administration (FDA) of ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), tezacaftor/ivacaftor (Symdeko®), elexacaftor/tezacaftor/ivacaftor (Trikafta™), Cayston®, and tobramycin (TOBI®).
Watch this video to see how clinical research has made a difference in the lives of people with CF.
Research by dedicated scientists and clinicians from a wide range of disciplines advances our understanding of cystic fibrosis every day, helping to shape clinical care practices for people living with the disease for years to come. These include studies conducted using patient data in the CF Foundation's Patient Registry, which are helping us identify trends and track the effectiveness of treatments.
From bench to bedside, the Foundation is supporting the best research here and abroad to improve the quality of life of people with CF today and increase the speed of innovative research and drug development to add tomorrows. Two major initiatives have launched recently that will help us in this mission.
In 2018, the Foundation announced that it was committing $100 million to the Infection Research Initiative, a comprehensive approach to improve outcomes associated with infections through enhanced detection, diagnosis, prevention, and treatment. The Foundation also is actively pursuing and funding a broad portfolio of new treatments for other complications of the disease, such as inflammation, excessive mucus, gastrointestinal problems, and cystic fibrosis-related diabetes.
The second major initiative concentrates on people with nonsense and rare mutations who will not benefit solely from CFTR modulators and need an effective treatment for the underlying cause of their disease. The Foundation has thus far committed over $72 million to the Nonsense and Rare Mutations Research and Therapeutics Initiative, a multifaceted effort that already has funded more than 60 projects over the past several years at both academic institutions and pharmaceutical companies.
By pursuing these bold strategies and others, the CF Foundation continues to build a robust pipeline of potential new therapies that fight the disease from every angle. Learn more about the CF Foundation's key research programs:
- Research Overview: A broad look at how the Foundation supports basic science, clinical research, and real-world research to expand our knowledge of cystic fibrosis and translate discoveries and insights into vital new treatments and clinical care practices for people living with CF.
- Research We Fund: See a snapshot of how the CF Foundation is funding cystic fibrosis research.
- CF Foundation Therapeutics Laboratory: Based in Lexington, Mass., the CF Foundation Therapeutics Laboratory identifies and tests potential groundbreaking therapies for CF, readying them for further development.
- Therapeutics Development Network: The Therapeutics Development Network is the largest CF clinical trials network in the world. It provides the resources and support for studies that are leading to important new therapies and better treatments.
- New Developments in Clinical Research: Watch interviews with leading CF researchers to learn about the latest cutting-edge studies into issues such as infections, nontuberculous mycobacteria (NTM), and nutrition and gastrointestinal health.
- Drug Development Pipeline: Discoveries from the laboratory are being turned into potential drugs that attack both the symptoms of CF and the cause -- a faulty gene that makes a defective protein.
- Research Centers: These CF "think tanks" are located at top universities and medical schools across North America, where scientists from many disciplines are brought together to combine their expertise to find a cure for CF.