RESEARCH

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 Kalydeco® (ivacaftor), Orkambi®  (lumacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor), Trikafta® (elexacaftor/tezacaftor/ivacaftor), Cayston® (aztreonam), and TOBI® (tobramycin).

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.

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 are helping with this mission.

To make meaningful progress against infections, we established the five-year Infection Research Initiative to help improve the detection, diagnosis, prevention, and treatment of infections. From 2018 through 2023, we invested more than $170 million to fund research and the development of new treatments. We also conducted a comprehensive review of our research portfolio, identifying and filling gaps, evaluating our investments, and recalibrating our infection research strategy. This initiative helped us set the agenda going forward for a robust infection research program so that we can continue to meet the needs of the CF community.

The second major initiative is the Path to a Cure, an ambitious research agenda to deliver treatments for the underlying cause of the disease and a cure for every person with CF. The Foundation is challenging potential collaborators to submit proposals that will accelerate the pace of progress in CF drug discovery and development and intends to allocate $500 million to the effort through 2025. The Path to a Cure centers around two core strategies to address the underlying cause of CF: restoring CFTR protein when none exists and fixing or replacing the underlying genetic mutation to address the root cause of CF. 

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 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.

  • 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.

*Information used with permission from The Cystic Fibrosis Foundation