Thursday, July 25, 2013

We are in the home stretch...

The light at the end of the tunnel is no longer a train. Watch what CF Researchers are most excited about and view the Cystic Fibrosis Foundation Timeline below. The CFF is dedicated to the search for the cure and committed to CF families - the fight will not end until CF stands for Cure Found. If you have 30 minutes, 30 hours, or 30 days - we need you! Your time and energy can help fuel the pipeline that is finding the cure for CF. Ask me how.


1938Dorothy Andersen, M.D., writes the first comprehensive medical report on cystic fibrosis (CF).
1953During 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.
1955The 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.
1961The Cystic Fibrosis Foundation-accredited care center network begins by establishing two centers devoted to treating CF.
1962The CF predicted median survival age is 10 years.
1962A total of 30 Foundation-accredited care centers are now in operation.
1964To investigate CF at the cellular level and find answers about this complex disease, the Foundation establishes the first basic science committee.
1966The Cystic Fibrosis Foundation launches a patient data registry that collects health information of patients seen at Foundation-accredited care centers.
1978The number of Foundation-accredited care centers totals more than 100.
1982The Foundation creates the Research Development Program, a network of research centers at leading universities and medical schools nationwide.
1988The Foundation launches the Cystic Fibrosis Services Pharmacy.
1989A 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.
1990CF researchers achieve “proof of concept” that gene therapy (in the lab dish) is possible.
1993Landmark gene therapy trial begins in people with CF.
1993The 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.
1997The Foundation establishes the Therapeutics Development Program.
1997The FDA approves TOBI®, the first aerosolized antibiotic designed for CF, which is proven to reduce hospital stays and improve lung function.
1998Specialized clinical research centers are designated as the Foundation’s Therapeutics Development Network.
2000Cystic Fibrosis Foundation Therapeutics (CFFT), a nonprofit research affiliate of the Foundation, is established to govern drug discovery and development efforts.
2000Foundation-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.
2002A CFFT-supported study shows azithromycin improves CF lung health.
2003CFFT-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.
2004CFFT-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.
2006VX-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.
2007Vertex selects a second potential drug known as VX-809 for development. Like VX-770, VX-809 addresses the root cause of CF, but it works by helping the defective CF protein move to its proper place in the cell.
2008The 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.
2010The 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.
2011The 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™.
2011 Results from the first part of an ongoing Phase 2 trial testing Kalydeco in combination with VX-809 show promising results in people with the most common CF mutation, Delta F508.
2012The 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.
2012Results from a Phase 2 trial of Kalydeco in combination with VX-809 show a significant improvement in lung function in people with two copies of the most common CF mutation, Delta F508.
2012The 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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