Cystic fibrosis (CF) is an inherited and chronic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which provides instructions for making a protein that transports salts across cellular membranes. Mutations in the CFTR gene prevent the transport protein from functioning properly, resulting in the production of a thick and sticky mucus surrounding the lungs, pancreas, liver, intestines, sinuses, and reproductive organs.
This thick mucus is particularly problematic in the lungs as it blocks the airways, making it difficult for patients to breathe. It also facilitates the growth of bacteria and infection by viruses.
CF is a progressive disease that currently has no cure, but a community of patients, families, friends, physicians, researchers, advocates, and organizations work together to improve the lives of patients with CF. The Cystic Fibrosis Foundation (CFF) is currently the largest organization in the U.S. focusing on that goal.
History of the CFF
In 1955, when the life expectancy of patients with cystic fibrosis was fewer than 10 years, a group of concerned parents joined forces to fight the disease. The first years of the CFF were filled with obstacles because little was known about the disease. Still, families continued to work, and six years after the CFF was established, the group had already created two centers for the treatment of CF and an accredited care center network.
Throughout the years, thousands of people have joined the cause, and the CFF now has a volunteer base that spans the whole country. It is heralded as the world’s leader in the search for a cure for CF.
Some milestones of the foundation include the launching, in 1966, of a patient registry that collects health information from patients seen at foundation-accredited care centers, the landmark increase of CFF-accredited care centers to more than 100 in 1978, the approval of the first treatment for CF patients in 1993, and the creation, in 2000, of the Cystic Fibrosis Foundation Therapeutics Lab, a nonprofit research affiliate of the organization focused on governing drug discovery and development efforts.
In addition, the CFF has participated in the development of Kalydeco (ivacaftor), the first treatment approved by the U.S. Food and Drug Administration that addresses the underlying cause of the disease. CFF also assisted in the development of Orkambi, the lumacaftor/ivacaftor combination for people ages 12 and older who have two copies of the most common CF mutation, F508del.
The CFF in research
The CFF is currently supporting multiple studies on treatments including CFTR modulators, which are designed to correct the defective CFTR protein, anti-infection therapies that fight acute and chronic lung infections, and anti-inflammatory treatments designed to reduce inflammation in the lungs.
The CFF offers many resources not only for CF patients, but also for their friends and families, and medical professionals working in the field. The CFF provides funding and accreditation for more than 120 CF care centers and 55 affiliate programs nationwide, including 96 programs for treating adults with CF.
In addition to CF care centers and affiliate programs, 70 CFF chapters and branch offices across the U.S. offer the CF community information and support on topics that include diagnosis, treatment, research, legal rights, insurance, clinical care guidelines, and emotional wellness.
Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.