Foundation and Vertex Announce Positive Early Results for VX-770 March 27, 2008 The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated. Patients who took the drug for 14 days showed significant improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. The findings suggest that VX-770 improves function of what is known as the faulty CFTR protein. This early data is promising and could have important implications for studies of other drugs in development. This is the first time that any potential therapy has improved the abnormal sweat chloride (salt) levels in a person with CF. Excessive sweat chloride is a key clinical indicator of cystic fibrosis. The “sweat test” is the traditional diagnostic test for CF. “These early results are an extraordinary endorsement of our hypothesis—that small molecules can correct the basic defect and affect the clinical indicators of cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Foundation. “The emerging data for VX-770 represents the most exciting results we’ve seen from a Phase 2 trial and increase our confidence that we’re on the right track.”   Secondhand Smoke Harms People With CF January 30, 2008 Secondhand smoke worsens lung function in people with cystic fibrosis, especially those with a specific gene, according to researchers at Johns Hopkins University School of Medicine. The new research, which was reported by Reuters and an article in MedScape, shows how genetic and environmental factors can interact to harm lung function in CF patients. The study of 812 people included 188 who were exposed to secondhand cigarette smoke at home. The average age of the participants was 19. Dr. Gary Cutting is the study’s lead author. Lung function in those exposed to secondhand smoke was reduced by about 10 percent compared to those not exposed, the researchers found. Lung function was determined by how much air a person could breathe out in the first second of expiration. Preston W. Campbell, III, M.D., executive vice president for medical affairs of the Cystic Fibrosis Foundation, was quoted in Medscape: "The Cystic Fibrosis Foundation has focused on secondhand smoke for some time, and Dr. Cutting's study is the best study to date. This study gives the CF Foundation evidence-based data for their work against smoking."   CF Research Leads to 2007 Nobel Prize in Medicine October 8, 2007 The 2007 Nobel prize in medicine, announced today, has been awarded to three scientists for their work in creating mouse models of disease.  All three of the scientists—Dr. Oliver Smithies of University of North Carolina, Sir Martin Evans of Cardiff University, and Dr. Mario Capecchi—have performed CF research that contributed to their award.  The initial steps in this work began when Sir Evans isolated embryonic stem cells, making it possible to manipulate their genes to produce a "designer mouse." In work supported in part by the CF Foundation, Dr. Smithies at University of North Carolina used the stem cells to create one of the first animal disease models by disrupting the mouse CF gene.  The resulting "CF mouse" has been widely used throughout the cystic fibrosis research community, resulting in a significant impact in understanding how CF affects the various organs. Because the mouse model is so useful, development of other CF mouse strains has followed. Teaming up with researchers at the University of Iowa, Dr. Capecchi engineered a mouse to carry the mutation that is the most common in people with cystic fibrosis.  The altered mice are used by Sir Evans and many other CF researchers to study the effects of potential treatments that are geared to repair specific mutations. These studies have led directly to active clinical trials today. Sir Evans has also utilized the cystic fibrosis mouse for extensive studies in gene therapy, becoming the first to repair the CF deficit in a whole animal. The genetic manipulation techniques developed by these scientists for creation of the CF mouse model have since been used for many other disease systems, revolutionizing the study of disease processes and drug development.   Florida to Begin Newborn Screening for Cystic Fibrosis June 28, 2007 State Joins 36 Others in Screening for Life-Threatening Genetic Disease (Washington, D.C.) – The state of Florida will begin screening all newborns for cystic fibrosis (CF) starting July 1. Florida joins 36 other states in the country, plus the District of Columbia, to routinely screen for CF at birth. “This is the right thing to do,” said Peter Hodge, a Boca Raton, Fla. father of two daughters with CF. Hodge’s 14-year-old daughter wasn’t diagnosed with the disease until she was a teenager. “We wish our daughter had been diagnosed at birth. That may have prevented some of the health damage she’s already suffered. I’m a big believer that knowledge is power and that newborn screening gives families that power.” Research shows that screening for CF will likely improve and extend the lives of those born with the disease. Early diagnosis allows for affected infants to begin therapeutic interventions immediately. These interventions have been shown to help people with CF to maintain or improve lung function, as well as increase life expectancy and reduce hospitalizations. Early intervention has been shown to improve height, weight, and cognitive function as well. The CF Foundation strongly urges all states to implement comprehensive programs for routine newborn screening for cystic fibrosis.   Investigational Therapy That Seeks to Correct Basic Defect in CF Enters Phase 2 Trial June 15, 2007 Vertex Pharmaceuticals Incorporated recently announced the beginning of a Phase 2 clinical trial of an investigational therapy known as VX-770. The drug, which is given orally, is designed to address a core defect in CF. The defect causes thick mucus to accumulate in the lungs and causes a host of serious problems including inflammation and infections. This potential therapy is aimed at correcting the defect and may benefit other organs in addition to the lungs. The trial will involve approximately 36 adult volunteers with cystic fibrosis from different CF care centers across the country. A Phase 1 clinical trial of VX-770, which was completed in 2006, demonstrated that the drug could achieve desired levels in the blood. The Phase 2 clinical trial will study the safety and tolerability of the drug compared with a placebo, and how well the body absorbs the drug. Currently, the drug is being studied only in individuals with CF who carry at least one G551D mutation in their CFTR genes.   New Therapy for Cystic Fibrosis Patients with Pancreatic Insufficiency Enters Phase 3 Clinical Trials May 31, 2007 Altus Pharmaceuticals, headquartered in Cambridge, Mass., announced the start of their Phase 3 clinical trials to evaluate the safety and efficacy of ALTU-135, a new oral pancreatic enzyme replacement therapy. Pancreatic insufficiency is found in approximately 90 percent of individuals with cystic fibrosis and is treated through the administration of enzymes that help patients to enhance digestion, improve growth, reduce gastrointestinal problems, and contribute to general nutritional health. Enzyme products for pancreatic insufficiency in use today are porcine-derived and require patients to take multiple capsules—sometimes 4 or 5—with every meal or snack. ALTU-135, in contrast, is microbially derived and requires taking only one smaller capsule with every meal or snack. In a previous Phase 2 study, ALTU-135 was found to be well-tolerated and achieved statistically significant improvements in the absorption of fat and protein, as well as an overall improvement in the absorption of carbohydrates. The Phase 3 trial, named DIGEST, will recruit approximately 300 patients in nearly 50 sites in the U.S. and abroad. Altus expects the trial to be completed in 2009 and, if the results are positive, will file for regulatory clearance in the U.S. during the first half of that year.

Traveling with Cystic Fibrosis  If you are a CFer traveling by plane  below you will find some helpful information to get you there worry free. Have these things on hand when flying 1. A list of your medications (including both the trade and generic names) with a description of why you need each one 2. A letter explaining your diagnosis, medicine, and equipment can be useful when going though customs, especially if you are traveling with a compressor, insulin, or EpiPen needles 3. A simple summary of your CF care from your doctor or clinic outlining your status, usual antibiotic combinations for an exacerbation, medication summary, and anything else that a doctor may require to provide treatment in a timely manner, in case you become unwell 4. Your CF clinic's contact information, including names, telephone numbers, and email addresses. Make photocopies of these documents, so you can give a copy to any official who requests it. Keep the originals on your person or in a safe place at all times. 5. If you are traveling outside the country, find out about the voltage and frequency of electricity and the type of plugs that are used in the country you are visiting. You may need plug adapters and/or voltage converters to avoid damaging your electrical equipment. 6. Before you travel to another country, find out if there is a list of CF clinics or hospitals in the areas you will visit, in case you need to seek medical attention. Other things to consider when flying 1. All medicines and medical devices need special consideration on flights. Notify the airline ahead of time of all medications, needles, and devices you are bringing. If you bring injection devices such as insulin or EpiPens on an airplane, you must have a note from your doctor explaining why you have them. You may also be required to bring your own needle disposal unit. Keep all medications and equipment in your carry-on baggage in case you need them on your flight or in case your checked baggage gets lost, and because temperature- sensitive medications can freeze in the baggage hold. Call the airline ahead to discuss storage solutions. You may be allowed to bring a large carry-on bag to accommodate your needs. Another solution may be "at the gate baggage stow", which is generally for medical items that must be used until boarding. The airline will tell you if any of your medical supplies may be boarded in this manner. 2. On the smaller planes the vest machine will not fit in the overhead bins. You can ask for at the gate baggage stow for them so you know they will be the first to be unloaded out of the plane. 3. Oxygen levels in airplanes are lower than oxygen levels on land. This usually doesn't pose a problem unless you have low lung function. If you plan to fly to your vacation destination, your doctor can give you a flight assessment to test your blood gases under normal conditions and then under conditions similar to those on an airplane. He or she will then advise whether it is safe for you to fly without extra oxygen. Some airlines provide oxygen at no cost, some charge a fee, and some do not provide oxygen at all. Most airlines will not let you use oxygen unless you book ahead. Extra travel tips 1. Pack extra enzymes for high-fat meals you may encounter 2. Take alcohol based waterless hand sanitizer with you 3. If you are going to a hot climate, take electrolyte supplementation- such as re hydration packets or salt tablets-with plenty of water 4. Many amusement parks or tourist sights have "short line" entrances, and many others offer free or reduced rate entrance passes to people (especially kids) with certain medical conditions. Inquire about them ahead of time and remember to bring a letter from your doctor indicating that you have CF.

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