Cystic Fibrosis Foundation

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Chronology of CF Research


Dorothy Andersen, M.D., writes the first comprehensive medical report on cystic  fibrosis.


During a heat wave in New York City, Paul di Sant'Agnese, M.D., and others connect the extra loss of salt by people with CF to the disease's underlying cellular problem.


The Cystic Fibrosis Foundation becomes incorporated as the National CF Research Foundation and awards the first research grants to Dorothy Andersen, M.D., Paul di Sant'Agnese, M.D., and Harry Shwachman, M.D.


The Foundation establishes the accredited care center network by creating two centers devoted to treating CF.


The CF predicted median survival age reaches 10 years.


A total of 30 Foundation-accredited care centers are in operation.


To investigate CF at the cellular level and find answers about this complex disease, the first basic science committee is established.


Patient Registry launched to collect health information of patients seen at Foundation-accredited care centers.


The number of Foundation-accredited care centers totals more than 100.


The Research Development Program, a network of research centers at leading universities and medical schools nationwide, is established.


A team of Foundation-supported scientists discovers the defective CF gene and its protein product (CFTR), opening the door to understanding the disease at its most basic level.


CF researchers achieve "proof of concept" that gene therapy (in the laboratory) is possible.


The U.S. Food and Drug Administration (FDA) approves dornase alfa (Pulmozyme®), which is proven to thin the thick mucus in the lungs and is the first drug developed specifically for CF.


The Foundation establishes the Therapeutics Development Program.


The FDA approves inhaled tobramycin (TOBI®), the first aerosolized antibiotic designed for CF, which is proven to reduce hospital stays and improve lung function.


Specialized clinical research centers are designated as the Foundation's Therapeutics Development Network.


Cystic Fibrosis Foundation Therapeutics (CFFT), a nonprofit research affiliate of the Foundation, is established to govern drug discovery and development efforts.


Foundation-supported scientists map the entire genetic structure of the most common cause of CF lung infections, the bacteria Pseudomonas aeruginosa.


A CFFT-supported study shows the antibiotic azithromycin improves CF lung health.


CFFT-supported scientists at Structural GenomiX Inc., determine the three-dimensional structure of a portion of the CFTR protein, opening the door to more drug discovery opportunities.


CFFT-supported studies in Australia and at the University of North Carolina show that inhaled hypertonic saline helps clear CF mucus and improve lung health. It becomes a therapeutic option.


Ivacaftor (formerly VX-770), an oral drug in development that targets the faulty CFTR protein, enters clinical trials. Ivacaftor is designed to open chloride channels that do not function correctly in people with the disease.


Phase 2 studies of ivacaftor in people with the G551D mutation of CF show unprecedented improvements in key signs of the disease. The studies achieve "proof of concept" that it is possible to treat the root cause of CF.


The FDA approves a new antibiotic, aztreonam for inhalation solution (Cayston®), to treat CF lung infections. The drug offers an alternative for people with CF who battle recurrent infections and develop resistance to existing antibiotics.


The FDA approves ivacaftor (Kalydeco®) for people with the G551D mutation of CF ages 6 and older. The drug is the first to address the underlying cause of CF and opens exciting new doors to research and development that may lead to a cure for all people living with the disease.


The FDA approves ivacaftor as a single therapy to treat people ages 6 and older with one of nine other rare CF mutations in addition to G551D, and later extends approval to children ages 2 to 5 with any of these 10 mutations -- representing about 8 percent of the U.S. CF population.


The FDA approves the lumacaftor/ivacaftor combination drug (Orkambi®) for people with CF ages 12 and older who have two copies of the most common CF mutation, F508del -- representing about a third of those with CF in the United States.


The FDA approves lumacaftor/ivacaftor (Orkambi®) for children with CF ages 6 to 11 who have two copies of the F508del mutation. The decision means that about 2,400 additional children in the U.S. are eligible to receive the drug, bringing the total number of those eligible for the treatment in the U.S. to nearly 11,000.


The Foundation maintains a robust pipeline of potential therapies that target the disease from every angle. The more drugs in the pipeline, the greater the odds of producing successful therapies and a cure for all people with CF.