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Oral therapy as safe, effective as insulin in cystic fibrosis-related diabetes

Patients with cystic fibrosis-related diabetes treated with oral repaglinide for 24 months experienced improvements in HbA1c and pulmonary function that were similar to those in patients treated with human insulin therapy, according to findings from an open-label, comparative intervention trial. “If cystic-fibrosis-related diabetes is diagnosed, treatment guidelines recommend insulin therapy with multiple daily injections, although there are no data to assess whether this is the optimum treatment,” Manfred Ballmann, MD, of the pediatric clinic at University Medicine Rostock, Germany, and colleagues wrote in the study background. “Oral antidiabetic therapies might be as effective as insulin f

Garlic Compound Could Kill Superbugs

Garlic can do more than ward off vampires. It appears an active sulphurous compound, called ajoene, in the allium can help fight superbugs in patients with chronic infections. Researchers at the University of Copenhagen have shown that the compound is able to destroy important components in the bacteria’s communication systems, which involve regulatory RNA molecules. So while ajoene doesn’t outright kill the bacteria, it prevents them from communicating with other microbes, which allows them to grow. “We really believe this method can lead to treatment of patients, who otherwise have poor prospects. Because chronic infections like cystic fibrosis can be very robust. But now we, together with

Kalydeco Seen as Safe in Treating Young Children, Ages 1 to 2, in Phase 3 Trial, Vertex Reports

Kalydeco (ivacaftor) is safe and effective in 1- and 2-year-olds with cystic fibrosis (CF), Vertex Pharmaceuticals reported based on data from its ongoing Phase 3 clinical trial in infants and toddlers. The treatment’s safety was similar to that seen in studies of older children, the company said. Moreover, the treatment improved sweat chloride levels — indicating its effectiveness in treating CF also in children as young as one year. The results prompted Vertex to start preparing regulatory requests in the U.S. and E.U. for Kalydeco to also be approved to treat children between the ages of 1 and 2 years. The company plans to submit its applications in early 2018. “We know that cystic fibros

New Simple Test Could Help Cystic Fibrosis Patients Find Best Treatment

These are nasospheroids that developed from a CF patient's nasal tissue in a dish. UNC researchers are using them to screen the effectiveness of CF treatments. Credit: UNC Several cutting-edge treatments have become available in recent years to correct the debilitating chronic lung congestion associated with cystic fibrosis. While the new drugs are life-changing for some patients, they do not work for everyone. In a study published in the Journal of Clinical Investigation Insight, a team led by UNC researchers presented a simple test that aims to predict which treatment is most likely to work for each patient, an approach known as personalized or precision medicine. “Any given drug may not

GLPG2222 Plus Kalydeco Shows Positive Clinical Results in Phase 2 Trial, Galapagos Reports

Adding investigational CFTR corrector GLPG2222 safely improved disease parameters in cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor), reported the therapy’s Belgian developer, Galapagos, citing Phase 2 clinical trial data. The benefit of adding GLPG2222 was similar to that seen in an earlier study of a tezacaftor-Kalydeco combination, researchers said. “The addition of the novel CFTR corrector molecule GLPG2222 on top of highly efficacious CFTR modulator treatment already given for years in patients with gating mutations was well tolerated and may bring additional benefit to patients,” Scott Bell of Prince Charles Hospital in Brisbane, Australia, and principal investigator o

Galapagos Cystic Fibrosis Drug Enters Phase I, Triggering €8.5M Payout

Galapagos will receive a $10M (€8.5M) milestone payment from its collaborator, AbbVie, after their cystic fibrosis candidate started a Phase I trial. Galapagos is a clinical-stage biotech developing small molecule medicines for a range of indications. It partnered up with the global pharma company, AbbVie, back in 2013, to develop a number of cystic fibrosis (CF) candidates for a combination therapy. Galapagos has pocketed $10M (€8.5M) from AbbVie on the back of the news that its CF corrector, GLPG3221, will enter Phase I to test its safety, tolerability and pharmacokinetics in healthy patients. Around 70,000 people are living with CF worldwide, a progressive disease that sees patients suffe

Supine posture changes lung volumes and increases ventilation heterogeneity in cystic fibrosis

Abstract Introduction Lung Clearance Index (LCI) is recognised as an early marker of cystic fibrosis (CF) lung disease. The effect of posture on LCI however is important when considering longitudinal measurements from infancy and when comparing LCI to imaging studies. Methods 35 children with CF and 28 healthy controls (HC) were assessed. Multiple breath washout (MBW) was performed both sitting and supine in triplicate and analysed for LCI, Scond, Sacin, and lung volumes. These values were also corrected for the Fowler dead-space to create ‘alveolar’ indices. Results From sitting to supine there was a significant increase in LCI and a significant decrease in FRC for both CF and HC (p<0.01).

Advances in the Prevention and Treatment of MRSA in Patients with Cystic Fibrosis

Active research and special expertise at Cleveland Clinic’s Adult CF Center Methicillin-resistant Staphylococcus aureus (MRSA) increases the risk of death for people with cystic fibrosis (CF). Studies reveal a dramatic increase in the number of individuals with CF living with MRSA infection, now comprising 25 percent of the CF population in the United States. These findings have led to active research by my group and others, as well as enhanced infection control practices to minimize transmission and optimize CF patient outcomes. Methicillin-resistant Staphylococcus aureus (MRSA) increases the risk of death for people with cystic fibrosis (CF). Studies reveal a dramatic increase in the numbe

Galapagos Initiates Phase 1 Trial to Evaluate CF Corrector GLP3221 in Healthy Volunteers

Galapagos has initiated a Phase 1 clinical trial in healthy volunteers to evaluate a new C2 corrector, called GLPG3221, developed for the treatment of cystic fibrosis (CF). The Phase 1 study, being conducted in Belgium, will evaluate the safety and tolerability of GLPG3221, and seeks to understand how the investigational therapy is processed once inside the body. Healthy volunteers will be randomized to receive either the C2 corrector or a placebo. The study is part of a collaborative agreement between Galapagos and AbbVie, established in September 2013. The aim of the collaboration is to discover, develop, and commercialize potentiator and corrector molecules for the treatment of CF. In 201

Spyryx Presents Data Ably Supporting SPX-101 as Potential Treatment for All CF Patients

Spyryx Biosciences recently presented preclinical data supporting the clinical benefits of its cystic fibrosis (CF) treatment candidate SPX-101, intended to improve lung function in all patients regardless of their underlying disease-causing mutation. The data, presented in four posters at the recent North American Cystic Fibrosis Conference (NACFC) in Indianapolis, demonstrated how the inhaled drug works — data that support the HOPE-1 Phase 2 clinical trial of SPX-101 in adults with CF, which is currently recruiting participants. SPX-101 targets epithelial sodium channels (ENaC) in the lungs, making them leave the cell surface. One of the consequences of a faulty CFTR channel (the cause of

Sclerosis medicine can fight multi-resistant bacteria

A surprising discovery shows that a widely used and 20-year-old medicine used to treat multiple sclerosis can also beat a type of multi-resistant bacteria for which there are currently only a few effective drugs Encountering bacteria with innocent names such as Pseudomonas aeruginosa and Enterobacteriaceae can lead to hospitalisation and - in a worst-case scenario - can also be life-threatening. The bacteria, which cause infections such as pneumonia, frequently develop multi-resistance towards classic antibiotics. Researchers from Aarhus University have discovered that a drug known as glatiramer acetate, which is normally used for treating the disease multiple sclerosis, has a hitherto unkno

Virtual reality used during cystic fibrosis treatment

Virtual reality headsets like this one allow the viewer to immerse themselves in another setting Virtual reality headsets are being used to help people going through treatment for cystic fibrosis. Patients are immersed in safari experiences and can explore their surroundings as a distraction therapy. Beth Clarke from Cardiff, who trialled it, said: "I was really pleasantly surprised. Any distraction from being in hospital is welcome." The first trial of its kind in the UK is being carried out at Llandough Hospital in Vale of Glamorgan. Ms Clarke said: "It really does take you to somewhere else for a few minutes. A hospital stay is never going to be enjoyable, the staff here are brilliant, bu

Tezacaftor-ivacaftor improves pulmonary function in cystic fibrosis patients: The EVOLVE trial

1. Tezacaftor-Ivacaftor significantly improves lung function and reduces the frequency of pulmonary exacerbations in cystic fibrosis (CF) patients homozygous for the Phe508 deletion (Phe508del). 2. Unlike safety outcomes reported for lumacaftor-invacaftor, there was no increased incidence of adverse respiratory events in the tezacaftor-ivacaftor group compared to placebo. Evidence Rating Level: 1 (Excellent) Study Rundown: Cystic fibrosis is caused by a loss of function in the cystic fibrosis transmembrane conductance regulator (CFTR) protein which results in thick mucus secretions negatively impacting various organ systems. For individuals with CF found to be homozygous for the Phe508del mu

CF Patients Increase Bacterial Load But Lose Bacterial Diversity As They Age, Study Finds

Cystic fibrosis (CF) patients have more bacteria but lower bacterial diversity in their lungs than people without CF — yet bacterial diversity diminishs with age in CF patients, a large multicenter study found. The study, “Airway microbiota across age and disease spectrum in cystic fibrosis,” appeared in the European Respiratory journal. CF is characterized by recurrent airway infection, inflammation and progressive decline in lung function. Genetic mutations in these patients induce a salt imbalance, leading to airway surface dehydration and the buildup of thick and sticky mucus. This creates an ideal habitat for bacteria, which is why chronic bacterial lung infections are the leading cause

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