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.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
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 people living with cystic fibrosis and their families face complicated issues related to getting the care they need. Cystic Fibrosis Foundation Compass makes sure that no one has to do it alone.
CF Foundation Compass is a service that helps people with CF and their families with navigating insurance options, connecting to legal information and experts, finding available financial resources, and tackling other life issues.
CF care team members are paramount in providing highly specialized care to people living with CF. CF Foundation Compass can help by serving as a strategic ally for care teams, so team members can focus on their patients’ care.
CF Foundation Compass can help you navigate insurance, financial, legal, and other issues you are facing. Use this online form to start your conversation with a Compass case manager today.
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.
There's a lot of buzz around personalized medicine, but what does it really mean? In the first plenary at NACFC, John P. Clancy, M.D., outlined the history of personalized medicine, where we are now and what the future holds. Read on for a quick recap.
Katherine Tuggle, Ph.D.
October 8, 2015
Opening Doors to CF Clinical Research
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Personalized medicine -- also called precision medicine -- has received a lot of attention in the past year, including a mention by President Obama during the annual State of the Union address in January. But what does it really mean for people with CF? In the opening plenary session at NACFC, John P. Clancy, M.D., professor and director of CF Clinical and Translational Research at Cincinnati Children's Hospital Medical Center, discussed just that.
The idea of personalized medicine is nothing new. It is practiced at CF care centers every day. Based on their specific needs, individuals with CF are prescribed medications and therapies, such as antibiotics, pancreatic enzymes and airway clearance therapies. But more and more, personalized medicine is beginning to have a new meaning.
With the recent development and approval of the first drugs that target the basic defect in CF, an abnormal CFTR protein, the idea of personalized medicine means understanding an individual's underlying genetic problem and treating it directly.
Clinical trials of ivacaftor (Kalydeco™) in people with G551D and other rare mutations clearly showed that drugs that target the malfunctioning CFTR protein at the cell surface have the potential to greatly improve health outcomes, including increased FEV1 and decreased exacerbations. Major clinical trials of the ivacaftor/lumacaftor combination (Orkambi™) also showed significant improvements in people with two copies of the F508del mutation, with the drug helping the CFTR protein get to the cell surface and function properly.
However, we have also learned that drugs targeting CFTR protein can result in a range of responses, with some individuals showing significant clinical improvement while others have little to no response.
The range of responses highlights the need to find ways to determine who will benefit from a specific therapy. Dr. Clancy explained how we've already started to do this, and where we can go from here.
Researchers have developed ways of creating so-called model systems, essentially cell samples taken from the nose or other area of the body of an individual with CF. The hope is that doctors will be able to take a cell sample from an individual with CF, expose it to different drug compounds and then decide which drug would benefit that person the most.
These model systems are still being tested, but they represent just how customized care could become once a broader range of drugs becomes available. Dr. Clancy emphasized that once therapies are prescribed, it is important to monitor their effectiveness to ensure they are delivering the best results for the individual. It is also important to remember that CF is a multi-organ disease, and the effectiveness of treatments in parts of the body besides the respiratory system needs to be evaluated to understand their true impact.
In the coming years, we hope that doctors will have an arsenal of new therapies at their disposal to help provide the best care and treatment options for individuals with CF so that they can live healthy and fulfilling lives. In this new era, doctors will be able to use information about a person, including his or her specific CF mutations, lung function and bacterial infections, to select from a number of therapies to determine the best treatment options.
It is encouraging to see how the progress of drugs that target the underlying cause of CF has changed the lives of so many living with the disease. Walking out of this plenary, I am even more excited to think about the potential that these therapies and others in development have for improving the lives of individuals with CF in the near future. Personalized medicine at its finest.
If you did not have the chance to watch this plenary live, watch a recording of the first plenary here.
Katherine Tuggle, Ph.D.
Director of Research, Cystic Fibrosis Foundation
Katherine has been involved with CF research since 2009 as both a graduate student and postdoctoral fellow at the University of Alabama at Birmingham. In 2014, she joined the Foundation to oversee its basic science research programs, including the 11 basic science research centers around the country. She is also involved in a number of Foundation initiatives focused on developing and validating model systems that may be used for personalized medicine. In her free time, Katherine enjoys traveling with her husband and spending time with her German shepherd.
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This site contains general information about cystic fibrosis, as well as personal insight from the CF community. It is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
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