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Research Milestones

1938 Dorothy Andersen, M.D., writes the first comprehensive medical report on cystic fibrosis (CF).
1953 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.
1955 The Cystic Fibrosis Foundation becomes incorporated as the National CF Research Foundation and awards the first research grants to Drs. di Sant’Agnese, and Andersen and Harry Shwachman, M.D.
1961 The Foundation establishes the accredited care center network by creating two centers devoted to treating CF.
1962 The CF predicted median survival age reaches 10 years.
1962 A total of 30 Foundation-accredited care centers are now in operation.
1964 To investigate CF at the cellular level and find answers about this complex disease, the Foundation establishes the first basic science committee.
1966 The Cystic Fibrosis Foundation launches a patient data registry that collects health information of patients seen at Foundation-accredited care centers.
1978 The number of Foundation-accredited care centers totals more than 100.
1982 The Foundation creates the Research Development Program, a network of research centers at leading universities and medical schools nationwide.
1988 The Foundation launches the Cystic Fibrosis Services Pharmacy.
1989 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.
1990 CF researchers achieve “proof of concept” that gene therapy (in the lab dish) is possible.
1993 Landmark gene therapy trial begins in people with CF.
1993 The Food and Drug Administration (FDA) approves Pulmozyme®, which is proven to thin the tenacious, sticky mucus in the lungs and is the first drug developed specifically for CF. The time taken to develop Pulmozyme is less than half of the industry average.
1997 The Foundation establishes the Therapeutics Development Program.
1997 The FDA approves TOBI®, the first aerosolized antibiotic designed for CF, which is proven to reduce hospital stays and improve lung function.
1998 Specialized clinical research centers are designated as the Foundation’s Therapeutics Development Network.
2000 Cystic Fibrosis Foundation Therapeutics (CFFT), a nonprofit research affiliate of the Foundation, is established to govern drug discovery and development efforts.
2000 Foundation-supported scientists map the entire genetic structure of the most common cause of CF lung infections — the Pseudomonas aeruginosa bacterium. Researchers can identify the function of specific genes and find ways to turn off the bad ones.
2002 A Foundation-supported study shows azithromycin improves CF lung health.
2003 Foundation-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.
2004 Foundation-supported studies in Australia and at the University of North Carolina show that hypertonic saline helps clear CF mucus. It is proven to improve lung function and reduce hospital stays, and becomes a therapeutic option.
2006 VX-770, an oral drug in development by Vertex Pharmaceuticals, Inc., with support from the Foundation, enters clinical trials. VX-770 is one of the first compounds to attack the root cause of CF, and works at the cellular level to open chloride channels that do not function correctly in people with the disease.
2007 Vertex selects a second potential drug known as VX-809 for development. Like VX-770, VX-809 addresses the underlying cause of CF, but it works by helping the defective CF protein move to its proper place in the cell.
2008 The Foundation and Vertex achieve a “proof of concept,” showing that it is possible to treat the root cause of CF. During Phase 2 studies of VX-770, trial participants, all of whom carry the G551D mutation of CF, show unprecedented improvements in key signs of the disease.
2010 The FDA approves a new inhaled antibiotic called Cayston® (aztreonam for inhalation solution) for the treatment of CF. Developed by Gilead Sciences, Inc., Cayston offers a much-needed antibiotic alternative for CF patients who battle recurrent lung infections and develop resistance to existing antibiotics.
2011 The Foundation announces that Phase 3 clinical trials of VX-770 showed profound results. Those receiving the drug demonstrated the highest increase on a lung function test seen in any clinical trial of a CF drug. Vertex submits a New Drug Application to the FDA for VX-770 under the trade name Kalydeco™.
2012  The FDA approves 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.
2013 Vertex begins enrollment in two large international Phase 3 trials of Kalydeco in combination with VX-809 in people with two copies of the most common CF mutation, Delta F508. Results from a Phase 2 trial testing the combination treatment showed promising results in people with two copies of Delta F508.
2013 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 CF. 

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Updated 11/13/13

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