News & Articles

New Test of Mucus Properties May Help with Coughing and Airway Clearance in CF

A new test was able to assess various properties of the mucus that accumulates in the lungs of people with cystic fibrosis and other pulmonary diseases, and may help to find treatments — or a combination of treatments — for CF patients that can make coughing a more viable way of clearing mucus from their airways. Developed by researchers at University of North Carolina at Chapel Hill, the assay was described in the study “Roles of mucus adhesion and cohesion in cough clearance,” published in the Proceedings of the National Academy of Sciences. Pulmonary mucus clearance is a critical mechanism in protecting the body from potentially harmful microorganisms and particulates — mucus works to tra

Avycaz May Be Effective for Multidrug-resistant P. aeruginosa Infections in CF Patients, Study Sugge

Antibacterial Avycaz (ceftazidime-avibactam) showed significant in vitroefficacy against multidrug-resistant Pseudomonas aeruginosa collected from the sputum of cystic fibrosis (CF) patients, according to a recent study. The study, “Multidrug-resistant Pseudomonas aeruginosa from sputum of patients with cystic fibrosis demonstrates a high rate of susceptibility to ceftazidime–avibactam,” was published in the journal Infection and Drug Resistance. In the U.S., most adults with CF are infected with P. aeruginosa, and approximately a third of these bacteria are multidrug-resistant. P. aeruginosa is associated with frequent pulmonary exacerbations in CF patients, presenting various mechanisms of

Ultrasound Predicts Advanced Liver Disease in CF Patients, According to Clinical Trial Data

Ultrasound may predict liver disease in young cystic fibrosis (CF) patients, according to the results of a clinical trial. Researchers compared liver patterns from young CF patients, and found that those with non-uniform patterns on ultrasound are more likely to develop liver scar tissue. Results from the the Cystic Fibrosis Liver Disease Network PUSH study (NCT01144507). PUSH stands for Prospective Study of Ultrasound to Predict Hepatic Cirrhosis in Cystic Fibrosis, which is a collaboration between several health clinics in North America. The results were presented at the North American Cystic Fibrosis Conference (Oct. 18-20 in Denver, Colorado). The PUSH study was funded by the Cystic Fibr

New cystic fibrosis drugs significantly improve lung function

A triple drug combination for treating the underlying cause of cystic fibrosis is currently under study. The preliminary results show a significant improvement in lung function. Cystic fibrosis is a genetic disease affecting the chloride channels in the body. These chloride channels are called cystic fibrosis transmembrane conductance regulator (CFTR). The major body systems affected by this mutation are the respiratory system and the gastrointestinal system. Cystic fibrosis mainly manifests itself as chronic lung disease and a deficiency in digestive enzymes. Chronic lung disease in people with cystic fibrosis is characterized by an increase in thick and sticky mucus and chronic lower respi

Law Student returned to life with lung transplantation

Law student came back to life with lung transplantation, 20 years of law student struggles with genetic disease ISTANBUL – For many years, Law School Ece Sapancalı, who is struggling with cystic fibrosis disease, has come back to life with a successful lung transplant. The patient was donated for Ece Sapanca and was transferred to the lung transplant list in April 2018. She was operated on October 17 with the discovery of the right donor. Dr. Ahmet Erdal Tasci Thoracic Surgery Training officer Lung Transportofficier: "I do not do anything alone, we're a team Transplants are not personal, but the success of teams She had problems with the airways for a long time He applied to our hospital We

The Bittersweet Downside of CFTR Modulators

I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three years now. My lung function has remained stable during that time. CFTR modulators are medications that “fix” the defect that causes cystic fibrosis. “Fixing” the defect means improving CFTR function. A person without cystic fibrosis (CF) has at least 50 percent CFTR function. People with CF can have any percentage under that, and clinical severity is likely to be inversely related to CFTR function percentage. CF is a very complicated disease. The existence of medications that can improve CFTR function gives me a reason to be hopeful. With Kalydeco (ivacafto

#NACFC2018 – RPL554 Shows Promising Results in CF Studies

Positive data from preclinical and Phase 2a studies evaluating the investigational cystic fibrosis (CF) treatment RPL554 were presented at the 2018 North American Cystic Fibrosis Conference (NACFC) in Denver, Colorado (Oct. 18-20). RPL554, developed by Verona Pharma, is an inhaled potential therapy for CF and chronic obstructive pulmonary disease (COPD). It is the first drug of its kind designed to inhibit both phosphodiesterase 3 and 4, which results in strong bronchodilatory and anti-inflammatory actions. In CF, mutations in the CF transmembrane conductance regulator (CFTR) gene results in a defective CFTR ion channel causing the accumulation of mucus. Previous studies have shown that RPL5

Worse CF Lung Outcomes Linked with Mutations Causing Iron Overload Disorder, Study Finds

Patients with cystic fibrosis (CF) who carry certain mutations in the HFE gene, which are also behind an iron overload disorder, have poorer lung function that declines faster and a higher risk of CF-related diabetes (CFRD) and intestinal blockage, a study reports. These findings support the idea that HFE variations can be genetic modifiers of CF, that is, genetic factors that change the clinical manifestations and outcomes of the disease. The study, “Mutations in the HFE gene can be associated with increased lung disease severity in cystic fibrosis,” was published in the journal Gene. Several gene variations other than those affecting the CFTR gene, the defective gene in CF, have been ident

Cystic Fibrosis Lung Function Improved With Triple Therapy

Patients with cystic fibrosis with either 1 or 2 Phe508del alleles who were treated with a drug combination of VX-659, tezacaftor, and ivacaftor showed improved forced expiratory volume in 1 second (FEV1), sweat chloride concentrations, and questionnaire scores, according to the results of a study published in the New England Journal of Medicine. The absolute change from baseline in the percentage of predicted FEV1 was compared between patients with cystic fibrosis who received either the triple drug combination (tezacaftor-ivacaftor-VX-659) or tezacaftor-ivacaftor-placebo. Patients who were homozygous or heterozygous for Phe508del alleles were included. Secondary end points consisted of the

Mucus, cough and chronic lung disease: New discoveries

As a cold ends, a severe mucus cough starts. Sound familiar? Two studies now give explanations: First, crucial mechanisms of the mucus in both diseased and healthy airways; second, what happens in such chronic lung diseases as cystic fibrosis and chronic obstructive pulmonary disease (COPD). "This is new knowledge, but there are no instant cures to be found yet. But to make progress, it's important to understand how the mucus clearance works," says Gunnar C. Hansson, Professor of Medical Biochemistry at Sahlgrenska Academy, University of Gothenburg. The studies, published in European Respiratory Journal and Journal of Clinical Investigation Insight, describe how the normal lung is kept clean

AzurRx BioPharma Receives Sanction of Cystic Fibrosis Therapeutics Development Network for the Study

On track to initiate Phase 2 trial in cystic fibrosis patients in the fourth quarter of 2018 and conclude in 2019 NEW YORK, Nov. 01, 2018 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ:AZRX) (“AzurRx” or the “Company”), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today announced that the Company's Phase 2 study protocol to investigate MS1819-SD in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) has received sanction from the Therapeutics Development Network (TDN), a collaborative network of CF clinical trial specialists supported by the Cystic Fibrosis Foundation (CFF). The Phase 2 multi-center

Santhera Starting Phase 1b/2a Trial Testing Potential CF therapy POL6014

A Phase 1b/2a, dose-escalating clinical trial evaluating the effects of POL6014 in patients with cystic fibrosis (CF) was recently launched, according to Santhera Pharmaceuticals, developer of the investigational therapy. The Phase 1b/2a (EudraCT 2016-005110-22) will evaluate the safety, tolerability, pharmacokinetics (drug absorption, distribution, and elimination by the body), and pharmacodynamics (the drug’s effects) of orally inhaled POL6014 in patients with CF. Researchers will also assess the highest tolerated dose of POL6014 and associated dose regimen. Other primary objectives are the effect of the treatment on patients’ lung function, assessed by spirometry tests, and blood oxygen l

#WeCare and #MECFA advance CF care in the Middle East

“to improve and extend the lives of Cystic Fibrosis patients through disease awareness, advocacy and advanced care." MECFA The Middle East Cystic Fibrosis Association (MECFA) is a non-governmental organization dedicated to improving and extending the lives of Cystic Fibrosis (CF) patients in the Middle East. Established in 2015 by a group of dedicated clinicians, MECFA member countries committed to CF care include: Armenia, Azerbaijan, Bahrain, Oman, Egypt, Iran, Iraq, Jordan, Kuwait, Lebanon, Pakistan, Qatar, Saudi Arabia, Syria, Turkey, UAE, Yemen and Sudan. MECFA strives to bring disease awareness, diagnostic tools, access to necessary medications and clinician education to its member co

#NACFC2018 — Vertex Presents Latest Clinical Data on CFTR Modulators

Vertex Pharmaceuticals’ VX-659 or VX-445 in combination with Kalydeco(ivacaftor) and tezacaftor (VX-661) can improve the lung function of patients with cystic fibrosis (CF) with one or two copies of the CFTR F508del mutation, Phase 2 data shows. Updated results of the studies were discussed in two oral presentations at the 32nd North American Cystic Fibrosis Conference held recently in Denver, and were also published in the The New England Journal of Medicine. The reports are titled “VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles” and “VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.” The Phase 2 tri

PTI-808 and PTI-801 Combo Improves Lung Function in CF Patients, Phase 1 Data Show

Treatment with a combo of Proteostasis Therapeutics’ investigational CFTR modulator therapies — PTI-808, a potentiator, plus PTI-801, a corrector — can promote significant improvements in lung function and reduction of sweat chloride in patients with cystic fibrosis (CF), Phase 1 data show. So far in the Phase 1 trial (NCT03140527), 21 patients carrying two copies of the F508del mutation in the CFTR gene — known to cause CF — were randomized and treated with either a placebo or a doublet combination of 100 mg, 200 mg, or 300 mg of PTI-801 plus 50 mg, 100 mg, or 300 mg of PTI-808. The collected data revealed that after seven and 14 days of treatment with the combo therapy, patients experienc

#NACFC2018 — Celtaxsys’ Acebilustat Delays Onset, Reduces Rate of Pulmonary Exacerbations in CF, Pha

Once-daily acebilustat, an investigational oral therapy for cystic fibrosis (CF) under development by Celtaxsys, reduces the frequency and delays the onset of pulmonary exacerbations in CF patients, results from a Phase 2 study show. Patients with better lung function benefited most, with a 96% higher chance of being free of exacerbations after 48 weeks of treatment, compared with a placebo. These topline results, previously announced by Celtaxsys, were presented at the 2018 North American Cystic Fibrosis Conference (NACFC) Oct. 18-20 in Denver, Colorado. Pulmonary exacerbations (the acute worsening of lung symptoms) are common throughout the life of a CF patient. Episodes generally include

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