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.
Phase 3 clinical trials of ivacaftor (Kalydeco™) in combination with lumacaftor (VX-809) for people with two copies of the F508del mutation of cystic fibrosis showed significant improvements in lung function and other key measures of the disease, according to Vertex Pharmaceuticals Inc.
Published on June 24, 2014
Based on these results, Vertex plans to submit a New Drug Application (NDA) by the end of 2014 to the U.S. Food and Drug Administration (FDA) for review, with possible approval in 2015.
Results from the two 24-week clinical trials mark an important milestone for nearly 50 percent of people with cystic fibrosis - specifically those with two copies of F508del, the most common CF mutation. The potential treatment is the first to combine two pills to address the underlying genetic cause of CF in people with the F508del mutation.
“This is a proud day for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Many people with CF and their families have been eagerly awaiting these results, and we are thrilled with the outcome. These studies further validate that we are on the right track to getting new and effective treatment into the hands of people with CF who so desperately need them.”
The Phase 3 trials were conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, more than 1,100 people with two copies of the F508del mutation, ages 12 and older, participated in the studies.
The trials tested two different doses of the ivacaftor and lumacaftor combination therapy. Compared with those on placebo, participants who took the combination treatment showed significant and consistent improvement in lung function and in other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations.
Repairing the defective protein in people with the F508del mutation is a particularly challenging and intricate process. In this mutation, a series of problems prevents the protein from reaching the surface of the cell. Lumacaftor is designed to move the F508del CFTR protein to the cell surface where ivacaftor can improve its function and help increase the normal flow of salt and fluids in and out of the cell.
These trials are the latest example of the CF Foundation's innovative venture philanthropy model, in which the Foundation raises and invests millions of dollars in research toward new treatments. This model has led to tremendous advances in the health and quality of life for people with CF. In fact, the life expectancy of a child with CF has doubled in the last 30 years.
Ivacaftor and lumacaftor were discovered by Vertex in collaboration with the CF Foundation, which provided substantial clinical and financial support in the development process, including approximately $75 million in research funding. The Foundation has since committed another $75 million with Vertex for the discovery of additional CF drugs.
Ivacaftor was approved by the FDA as a stand-alone drug for people with the G551D mutation in 2012, and eligibility for the drug was expanded to people with several other closely related mutations in February 2014.
Dr. Beall continued: “Kalydeco's approval was the essential first step in treating the underlying defect in CF; now, with this promising drug combination, we are taking another critical step forward in making sure that all people with CF have effective treatments, regardless of their mutation. We will not rest until we reach that goal.”
The Foundation continues to pursue the development of other promising compounds that address the basic defect in CF through collaborative efforts with leading biotech and pharmaceutical companies, including Pfizer, Genzyme and others. The exciting gains in CF drug discovery and development have attracted considerable interest from pharmaceutical companies new to the CF field, spurring research and innovative strategies that could speed development of therapies for all people with CF, including those with rare mutations.
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