Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
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Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
Published on July 18, 2017
Promising early stage clinical trial results for three next-generation CFTR modulator therapies that target the underlying defect in cystic fibrosis were announced today. The findings are the first to demonstrate the potential of next-generation modulators to treat the underlying cause of CF in people with a single F508del CFTR mutation. The therapies included in the studies combine tezacaftor, ivacaftor, and a next-generation modulator.
The studies, led by Vertex Pharmaceuticals Inc., highlight the potential of next-generation combination therapies to be significantly more effective than current FDA-approved modulators and to also benefit more people with CF than ever before. Data from the trials, along with results from another study expected to begin later this year, will help Vertex decide which next-generation modulator candidate or candidates to move forward into Phase 3 studies in early 2018.
The two Phase 2 studies and one Phase 1 study examined the safety and effectiveness of three different compounds -- VX-440, VX-152, and VX-659 -- in combination with ivacaftor and tezacaftor (VX-661) in individuals with one F508del CFTR mutation and one minimal function mutation. (“Minimal function” refers to mutations that do not make meaningful CFTR protein. These mutations include nonsense mutations, also known as “stop” or “x” mutations.) Participants in the trials showed striking improvements in lung function, comparable to ivacaftor (Kalydeco®) in the original G551D trials. They also experienced improvements in their sweat chloride, and the triple combination was generally well tolerated by patients in the studies.
Data also showed significant improvement in lung function in participants with two copies of the F508del mutation when VX-440 and VX-152 were added to tezacaftor and ivacaftor, compared to treatment with only tezacaftor and ivacaftor.
“We have been working for many years to develop therapies that could benefit people with CF who have a single F508del mutation, and the results announced by Vertex today are a significant step toward that goal,” said Michael Boyle, M.D., senior vice president for therapeutics development for the Cystic Fibrosis Foundation. “This is an exciting moment for the CF community, and we are pleased to play a role supporting the upcoming studies through our clinical trials network and look forward to this next stage which we hope will confirm these very promising results.”
He continued, “Clinical trials play a critical role in continuing to advance these therapies, and we commend all of the participants who volunteer, without whom new advances, such as the ones announced today, simply wouldn't occur.”
Cystic Fibrosis Foundation Therapeutics Inc., the CF Foundation's nonprofit drug discovery and development affiliate, provided $150 million in research funding to Vertex to support its pursuit of treatments that address the underlying defect in CF, including $75 million to develop next-generation candidates currently in clinical development.
“Today's announcement brings us even closer to reaching our mission, and our community should feel great pride in the tremendous role they have played in achieving this milestone. The dreams of the parents who first started the CF Foundation more than 60 years ago are being realized step by step, and we are inspired to continue our work until all people with CF have the chance they deserve for longer, healthier lives,” said Preston W. Campbell, III, M.D., president and CEO of the Cystic Fibrosis Foundation. “We are grateful to the many people with CF and their families who participated in these clinical studies, as well as to the Vertex team. Based on these results we have more hope than ever that disease-modifying combination treatments will benefit more than 90 percent of the CF community. We will not rest until 100 percent of our population has the cure they need.”
Summary of Results:
For additional information regarding the results of these clinical trials, including the safety and efficacy outcomes, please see the Vertex press release.
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