Claim: Trial Shows Cystic Fibrosis Drug Helped Ease Breathing
Verdict: SUPPORTS

Retrieved passages:
title: Denufosol
text: with the compound. The drug was also investigated for the treatment of retinal detachment and other retinal diseases, but trials were terminated in 2006. In Phase III studies, denufosol was orally inhaled by patients with cystic fibrosis three times a day using a jet nebulizer. To be effective, it had to reach the deeper parts of the lung (bronchioles), making it unsuitable for children under five years of age. Cystic fibrosis is characterised by a defect of the chloride channel CFTR (cystic fibrosis transmembrane conductance regulator) on epithelial cells in the lungs. The CFTR regulates the components of sweat, digestive

title: Cystic fibrosis
text: viscosity. Denufosol, an investigational drug, opens an alternative chloride channel, helping to liquefy mucus. Whether inhaled corticosteroids are useful is unclear, but stopping inhaled corticosteroid therapy is safe. There is weak evidence that corticosteroid treatment may cause harm by interfering with growth. Pneumococcal vaccination has not been studied . , there is no clear evidence from randomized controlled trials that the influenza vaccine is beneficial for people with cystic fibrosis. Ivacaftor is a medication taken by mouth for the treatment of CF due to a number of specific mutations responsive to ivacaftor-induced CFTR protein enhancement. It improves lung function by

title: Ivacaftor
text: to a mutation called G551D. The safety and efficacy of ivacaftor for the treatment of cystic fibrosis in patients with this mutation was examined in 2 clinical trials. The first trial was performed in adults having baseline respiratory function (FEV1) between 32% and 98% of normal for persons of similar age, height, and weight. The baseline average was 64%. Improvement in FEV1 was rapid and sustained. At the end of 48 weeks, people treated with ivacaftor had on average an absolute increase in FEV1 of 10.4%, vs. a decline of 0.2% in the placebo group. Pulmonary exacerbations were reduced by

title: Gene therapy
text: immune response) removed using CRISPR-Cas9. A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy. In February Kite Pharma announced results from a clinical trial of

title: Cystic fibrosis
text: help push air into their lungs. These machines, known as bilevel positive airway pressure (BiPAP) ventilators, help prevent low blood oxygen levels during sleep. Non-invasive ventilators may be used during physical therapy to improve sputum clearance. It is not known if this type of therapy has an impact on pulmonary exacerbations or disease progression. It is not known what role non-invasive ventilation therapy has for improving exercise capacity in people with cystic fibrosis. During severe illness, a tube may be placed in the throat (a procedure known as a tracheostomy) to enable breathing supported by a ventilator. For children, preliminary

title: Cystic fibrosis
text: full-length protein. The aminoglycoside gentamicin has been used to treat lung cells from CF patients in the laboratory to induce the cells to grow full-length proteins. Another drug targeting nonsense mutations is ataluren, which is undergoing Phase III clinical trials . Cystic fibrosis Cystic fibrosis (CF) is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in

title: Orla Tinsley
text: campaign to get Kalydeco into Ireland after experts rejected making the drug available because it was too expensive. The drug is the first to treat the cause of cystic fibrosis and not just the symptoms. In February 2013 the Minister for Health announced that the drug would be made available. Since late 2013 Ms Tinsley is one of the first people in the world to be treated by the second new cystic fibrosis drug lumacaftor/ivacaftor which treats the most common mutation of CF. The drug has proven to improve lung function and health for people with CF. In 2014 Ms

title: Denufosol
text: Denufosol Denufosol (INN) is an inhaled drug for the treatment of cystic fibrosis, being developed by Inspire Pharmaceuticals and sponsored by the Cystic Fibrosis Foundation. It was tested in two Phase III clinical trials, TIGER-1 and TIGER-2. Initially, in the first Phase III trial, TIGER-1, the compound showed significant results as compared with placebo. In the second Phase III trial, TIGER-2, the compound did not meet the primary endpoint, a significant change in baseline FEV1 (forced expiratory volume in one second) at the week 48 endpoint as compared to placebo. As of 2011, no additional clinical studies are being conducted

title: Cystic fibrosis
text: to improve lung function by impeding the growth of colonized bacteria. Inhaled antibiotic therapy helps lung function by fighting infection, but also has significant drawbacks such as development of antibiotic resistance, tinnitus, and changes in the voice. Inhaled levofloxacin may be used to treat "Pseudomonas aeruginosa" in people with cystic fibrosis who are infected. The early management of Pseudomonas aeruginosa infection is easier and better, using nebulised antibiotics with or without oral antibiotics may sustain its eradication up to 2 years. Antibiotics by mouth such as ciprofloxacin or azithromycin are given to help prevent infection or to control ongoing infection.

title: Cystic fibrosis
text: can significantly improve QOL. No definitive cure for CF is known, but diverse medications are used, such as mucolytics, bronchodilators, steroids, and antibiotics, that have the purpose of loosening mucus, expanding airways, decreasing inflammation, and fighting lung infections, respectively. Cystic fibrosis is the most common life-limiting autosomal recessive disease among people of European heritage. In the United States, about 30,000 individuals have CF; most are diagnosed by six months of age. In Canada, about 4,000 people have CF. Around 1 in 25 people of European descent, and one in 30 of Caucasian Americans, is a carrier of a CF mutation.

title: Pulmonary heart disease
text: resolve the shortness of breath. Additionally, oxygen to the lungs also helps relax the blood vessels and eases right heart failure. When wheezing is present, the majority of individuals require a bronchodilator. A variety of medications have been developed to relax the blood vessels in the lung, calcium channel blockers are used but only work in few cases and according to NICE are not recommended for use at all. Anticoagulants are used when venous thromboembolism is present. Venesection is used in severe secondary polycythemia (because of hypoxia), which improves symptoms though survival rate has not been proven to increase.Finally, transplantation

title: Cystic fibrosis
text: is positive expiratory pressure physiotherapy that consists of providing a back pressure to the airways during expiration. This effect is provided by devices that consists of a mask or a mouthpiece in which a resistance is applied only on the expiration phase. Operating principles of this technique seems to be the increase of gas pressure behind mucus through collateral ventilation along with a temporary increase in functional residual capacity preventing the early collapse of small airways during exhalation. As lung disease worsens, mechanical breathing support may become necessary. Individuals with CF may need to wear special masks at night to

title: Vertex Pharmaceuticals
text: Vertex gained FDA approval of the first drug, Kalydeco, to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation. In the US, 30,000 people have cystic fibrosis. About 4% of those, or 1,200, have the G551D gene mutation. Vertex is marketing the drug at $307,000 a year per patient. Vertex also is studying ivacaftor in combination with another drug (lumacaftor) for the most common mutation in CF, known as F508del, and expects the first set of results in 2012. Vertex worked for 13 years with the

title: Ivacaftor
text: about half in the ivacaftor group relative to the placebo group. In a second trial conducted in children age 6 to 11, the average improvement in FEV1 was an absolute increase of 12.5% in the ivacaftor group at 48 weeks, compared to a very slight decline in the placebo group. A third clinical trial examined the efficacy of ivacaftor in people with cystic fibrosis due to G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R mutations. This trial, which included 39 people of age greater than 6 years, used a crossover design. The people in the trial had FEV1 averaging

title: Cystic fibrosis transmembrane conductance regulator
text: a clinically important sweat test for cystic fibrosis often used diagnostically with genetic screening. Cystic fibrosis transmembrane conductance regulator has been shown to interact with: It is inhibited by the anti-diarrhoea drug crofelemer. CFTR has been a drug target in efforts to find treatments for related conditions. Ivacaftor (trade name Kalydeco, developed as VX-770) is a drug approved by the FDA in 2012 for people with cystic fibrosis who have specific CFTR mutations Ivacaftor was developed by Vertex Pharmaceuticals in conjunction with the Cystic Fibrosis Foundation and is the first drug that treats the underlying cause rather than the symptoms

title: Ivacaftor
text: scientific research performed by the National Institutes of Health and relied upon by Vertex in its cystic fibrosis drug discovery programs. The company responded in an email that "while publicly funded academic research provided important early understanding of the cause of cystic fibrosis, it took Vertex scientists 14 years of their own research, funded mostly by the company, before the drug won approval." The Cystic Fibrosis Foundation, a non-profit organization dedicated to improving healthcare for people with cystic fibrosis, provided $150 million of the funding for the development for ivacaftor in exchange for royalty rights in the event that the

title: Venture philanthropy
text: which include a drug candidate that could treat many more people with CF. It also pointed out the strong efficacy of the drug, and laid out the costs of managing CF that would be saved for people that the drug could treat; those costs include repeated hospitalizations and lung transplants. Nonetheless the high price led to sharp criticism of Vertex and the CFF. Twenty-nine physicians and scientists working with people with cystic fibrosis (CF) wrote to the CEO of Vertex Pharmaceuticals to plead for lower prices. CFF made the investment in exchange for a promise of royalties paid on sales

title: Joshua Boger
text: Administration as Incivek in May 2011. A third product, an orally administered pill for the treatment of cystic fibrosis, grew out of the acquisition of Aurora Biosciences Corporation by Vertex in 2001. The San Diego research company had a contract with the Cystic Fibrosis Foundation, a nonprofit for patient advocacy and research, to create an assay for the disease. After the acquisition, the Cystic Fibrosis Foundation approached Vertex about expanding the contract to search for a drug treatment. The potential market for such a drug was small, unlikely to return a profit. The problem required a new approach: finding a

title: Bronchiectasis
text: One commonly used therapy is beclometasone dipropionate. Although not approved for use in any country, mannitol dry inhalation powder, has been granted orphan drug status by the FDA for use in people with bronchiectasis and with cystic fibrosis. The disease affects between 1 per 1000 and 1 per 250,000 adults. The disease is more common in women and increases as people age. It became less common since the 1950s, with the introduction of antibiotics. It is more common among certain ethnic groups such as indigenous people. The exact rates of bronchiectasis are often unclear as the symptoms are variable. Rates

title: Icos
text: to develop severe respiratory problems. Icos also tested Pafase for acute respiratory distress syndrome (ARDS). In phase II trials for ARDS, Pafase reduced the death rate after 28 days and reduced the chance that the lungs of the patient would fail. However, Icos halted development in December 2002 when interim data from phase III trials for sepsis showed that the drug did not help patients survive. Scientists at Northwestern University later studied Pafase for necrotizing enterocolitis, and there is ongoing research on the enzyme for atherosclerosis at the University of Utah. In June 2000, Icos and Texas Biotechnology formed a