CYSTIC FIBROSIS FOUNDATION …ADDING TOMORROWS EVERYDAY

ABOUT CYSTIC FIBROSIS FOUNDATION  Established in 1955  CFF discovered that children with the disease rarely lived long enough to attend elementary school.  This is why CFF is more determined than any other foundation in the world, to find a cure for this deadly disease.  The organization funds more research than any other organization and created nearly every CF drug that is available today.  a non-profit donor-supported organization, but it is also dedicated to fighting and curing CF.  Their focus is to:  “support development of new drugs to fight the disease, improve the quality of life for those with CF, and ultimately to find a cure.

WHAT IS CYSTIC FIBROSIS?  CF is an inherited chronic disease that affects the lungs and digestive system.  It is caused by a defective gene and its protein product that produces unusually thick, sticky mucus that :  Clogs the lungs and leads to life-threatening lung infections  Obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food

SYMPTOMS OF CYSTIC FIBROSIS  Very salty-tasting skin  Persistent coughing, at times with phlegm  Frequent lung infections  Wheezing or shortness of breath  Poor growth/weight gain in spite of good appetite  Frequent greasy, bulky stools or difficulty in bowel movement

IMPORTANT INFORMATION  30,000 children and adults have CF in the United States (70,000 worldwide)  1950s-few children with CF lived to attend elementary school  Today advances in research and medical treatments have further enhanced and extended life for children and adults with CF  Many people now expect to live into their 30s, 40s and beyond

STATISTICS  About 1,000 new cases of CF are diagnosed each year.  More than 70% of patients are diagnosed by age 2  More than 45% of the CF patient population is age 18 or older  The predicted median age of survival for a person with CF is in the late 30s  No 2 people with CF can be in the same room because they will get each other sick

PERSONAL STORY  “I’ve lived with CF my whole life so I’m pretty used to it but sometimes it gets annoying if I’m coughing and people always ask if I’m sick.”  “I have to take medicine everyday and stay active to keep myself healthy.”  “I have done cheer and tumbling since 4 th grade and sometimes I go skateboarding with my friends”  “I get scared when I go to the doctors because it makes me realize that I’m not healthy like everyone else.”

THE CYSTIC FIBROSIS FOUNDATION IS… HOPE IN ACTION Although there is no cure for this disease, the foundation is working long hours to extend and enhance life for those who have this disease. Research pioneers are blazing new trails in drug development. Fundraisers are being held, securing the money needed to support the fight. Many advocates make sure CF is a top priority in government industry and research. Stewards are using donations to help fuel the creation of new drugs. There are also caregivers that link patients and families to specialized CF care and helping hands that offer support, information and resources.

TIMELINE Research Milestones 1938 Dorothy Andersen, M.D., writes the first comprehensive medical report on cystic fibrosis (CF) 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 Drs. di Sant’Agnese, and Andersen and Harry Shwachman, M.D The Cystic Fibrosis Foundation-accredited care center network begins by establishing two centers devoted to treating CF The CF predicted median survival age is 10 years A total of 30 Foundation-accredited care centers are now in operation To investigate CF at the cellular level and find answers about this complex disease, the Foundation establishes the first basic science committee The Cystic Fibrosis Foundation launches a patient data registry that collects health information of patients seen at Foundation-accredited care centers The number of Foundation-accredited care centers totals more than The Foundation creates the Research Development Program, a network of research centers at leading universities and medical schools nationwide The Foundation launches the Cystic Fibrosis Services Pharmacy 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 lab dish) is possible Landmark gene therapy trial begins in people with CF.

TIMELINE CONT’D 1993 Landmark gene therapy trial begins in people with CF 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 The Foundation establishes the Therapeutics Development Program The FDA approves 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 Pseudomonas aeruginosa bacterium. Researchers can identify the function of specific genes and find ways to turn off the bad ones A CFFT-supported study shows 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 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 Pseudomonas aeruginosa bacterium. Researchers can identify the function of specific genes and find ways to turn off the bad ones A CFFT-supported study shows 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.

TIMELINE CONT’D 2004 CFFT-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 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 Vertex 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 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 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 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™ 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 F 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 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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