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P. aeruginosa Infection Differs Between Cystic Fibrosis and Non-CF Bronchiectasis, Study Shows

Pseudomonas aeruginosa, one of the most common types of bacteria present in severe lung infections, infects cystic fibrosis (CF) patients differently than those with non-cystic fibrosis bronchiectasis, according to a long-term study., Researchers observed that these bacteria are more stable in CF patients than in non-CF bronchiectasis patients, and that the appearance of new strains was not necessarily associated with bronchiectasis exacerbations, as opposed to CF. The study, “Epidemiology and natural history of Pseudomonas aeruginosa airway infections in non-cystic fibrosis bronchiectasis,” was published in the journal ERJ Open Research. Infection with P. aeruginosa has been thoroughly stud

Triclosan, often maligned, may have a good side — treating cystic fibrosis infections

Maybe you’ve had the experience of wading in a stream and struggling to keep your balance on the slick rocks, or forgetting to brush your teeth in the morning and feeling a slimy coating in your mouth. These are examples of bacterial biofilms that are found anywhere a surface is exposed to bacteria in a moist environment. Besides leading to falls in streams or creating unhealthy teeth, biofilms can cause large problems when they infect people. Biofilms, multicellular communities of bacteria that can grow on a surface encased in their own self-produced matrix of slime, can block immune cells from engulfing and killing the bacteria or prevent antibodies from binding to their surface. On top of

Vertex, NHS England no closer to Orkambi settlement

Vertex and NHS England remain locked in a battle over the provision of cystic fibrosis drug Orkambi, seemingly no closer to a settlement that would allow patients access to the drug on the NHS. In a statement, Vertex said it had made the “best offer in the world” to NHS England that includes Orkambi (lumacaftor/ivacaftor) as well as Kalydeco (ivacaftor) and future medicines still in development. It also provides “budget certainty to NHS England and assurance that patients will have rapid access to advances in medicines soon to come, just like agreements made in other countries,” the firm noted. However, it seems NHS England has rejected the offer, as Vertex said it was now calling on the gov

Proof that Mycobacterium Abscessus Doesn’t Always Lead to Transplant Failure

Part two of a series. Read part one. Last week, I wrote about how disheartened I felt that many lung transplant centers refuse to help cystic fibrosis patients with Mycobacterium abscessus complex (Myco Ab), which is a species of nontuberculous mycobacteria (NTM). Myco Ab makes transplant difficult, but not impossible. I aim to establish both that Myco Ab patients can be successfully transplanted, and that transplant centers need to figure out their policies regarding transplanting patients with the bug. Today, I want to shift from my perspective to those of researchers in the NTM and lung transplant fields. I’m going to make this simple, straight to the point. Below, I summarize a few studi

Azithromycin Reduced Pulmonary Exacerbation Risk in Cystic Fibrosis

Azithromycin significantly reduced the risk for pulmonary exacerbations and resulted in sustained improvement in weight but had no effect on microbiologic outcomes in children with cystic fibrosis with early Pseudomonas aeruginosa infection, according to the results of a study published in the American Journal of Respiratory and Critical Care Medicine. Nicole Mayer-Hamblett, PhD, professor, Department of Pediatrics and adjunct professor, Department of Biostatistics at the University of Washington and the Seattle Children's Hospital in Seattle, and colleagues analyzed data from the OPTIMIZE trial (Optimizing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis; ClinicalTria

Health Canada Approves Symdeko to Treat CF Patients 12 and Up with Certain Gene Mutations

People ages 12 and older with cystic fibrosis (CF) who live in Canada can now be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). This follows Health Canada‘s approval of Symdeko to treat people who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), or who have one copy of F508del mutation in the CFTR gene and at least one other variant in the gene that is responsive to the treatment. Defects in the CFTR gene are the underlying cause of cystic fibrosis. Some of treatment-responsive mutations are P67L, D110H, R117C, L206W, R352Q, A455E, D579G, S945L, S977F, R1070W, and D1152H. CF patients who don’t know their gene mu

Omega-3 Essential Fatty Acids May Benefit Cystic Fibrosis Patients, Mouse Study Suggests

Dietary supplementation with a type of omega-3 essential fatty acid during pregnancy and in offspring for 60 days after birth improved the growth defects and reduced lung damage in a mouse model of cystic fibrosis (CF), a study reports. The study, “Long-term dietary (n-3) polyunsaturated fatty acids show benefits to the lungs of Cftr F508del mice,” was published in the journal Plos One. Accumulation of excess mucus due to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene facilitates the growth of bacterial pathogens like Pseudomonas aeruginosa in CF patients. Pseudomonas infection promotes the loss of lung function, and remains the leading cause of death and co

Supplemental antioxidants may reduce exacerbations in cystic fibrosis

An antioxidant-enriched vitamin may decrease respiratory exacerbations in people with cystic fibrosis (CF), according to new research published online in April in the American Journal of Respiratory and Critical Care Medicine. In "Effects of an Antioxidant-Enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial," Scott D. Sagel, MD, Ph.D., a professor of pediatrics at Children's Hospital Colorado and director of the University of Colorado Cystic Fibrosis Center, and coauthors report a 50 percent reduced risk of time to the first exacerbation requiring antibiotics in those receiving the supplemental antioxidants. During the 16-week study of 73 patients (36 received

AzurRx BioPharma reports positive results in its Phase 2a trial of pancreatitis drug

MS1819-SD was developed to treat exocrine pancreatic insufficiency caused by chronic pancreatitis AzurRx BioPharma Inc (NASDAQ:AZRX) announced a successful Phase 2a trial study of its pancreatitis drug. MS1819-SD is designed to treat exocrine pancreatic insufficiency caused by chronic pancreatitis, a disease characterized by inflammation of the pancreas. READ: Trevena shares pop on successful completion of Phase 1 study of acute migraine treatment Initial data from the study shows a “very strong safety and efficacy profile”, according to the company’s press statement. The Brooklyn-based biopharma will look into applying the drug to the treatment of cystic fibrosis as well. “Based on these da

Nanoparticles Therapy May Be ‘One-Size-Fits-All’ Approach for CF, Study Suggests

A potential therapy delivered through nanoparticles enables production of the normal, healthy form of the key CFTR protein in cystic fibrosis (CF), a new study suggests. The research, “Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis,” was published in the journal Molecular Therapy. Although CF is a monogenic disorder, which means it is caused by alterations in a single gene (the cystic fibrosis transmembrane conductance regulator, or CFTR), more than 300 different disease-related mutations in CFTR have been discovered. This means significant variance in disease progression from patient to patient. Patients with CF may be treated with appro

Pancreatic Cell Transplant Program Offers New Hope to Some CF Patients

The University of North Carolina (UNC) is among only a few institutions in the United States that offers patients with inflamed pancreas (pancreatitis) or pancreatic cystosis a surgical treatment option that allows a better outcome. It is relatively common for patients with cystic fibrosis (CF) to develop small cysts in the pancreas (pancreatic cystosis), as a result of impaired hydration of the organ, high protein content secretions, and inflammation. There is no treatment for this condition, and only management of the symptoms or surgical removal of the cysts are possible therapeutic options. The Chronic Pancreatitis and Autologous Islet Cell Transplant Program in UNC was established in 20

Vertex Pharmaceuticals opens expanded San Diego research center with focus on cystic fibrosis

Vertex Pharmaceuticals opened its new San Diego research center Monday, starting a new chapter in a decades-long quest to not only treat but cure cystic fibrosis. In 18 years, three drugs for the lung-ravaging disease have emerged from Vertex’s San Diego center and more are in the pipeline. The first, Kalydeco, was approved in 2012. It is the first drug that treats the underlying cause of the disease. The second, Orkambi, was approved three years later. And the third, Symdeko, was approved in February. These drugs can benefit about half of all patients with the incurable disease. In the next several years, Boston-based Vertex hopes its drugs can help nearly all patients live longer, healthie

WVU Medicine Granted CFF Accreditation for its Adult Cystic Fibrosis Program

The Adult Cystic Fibrosis Program at West Virginia University Medicine’s Mountain State Cystic Fibrosis Center was recently awarded accreditation from the Cystic Fibrosis Foundation (CFF) for its work in the cystic fibrosis (CF) community. The accreditation is granted to institutions after a thorough application process that includes a site visit, interviews with the team, and a review of metrics (such as lung function, body mass index, and others) impacting patient health. Quality improvement initiatives, clinical trials research, and community outreach are also assessed. “Gaining separate adult program accreditation through the CFF is recognition of the dedicated and high quality care we h

Immunosuppressive drug therapy for preventing rejection following lung transplantation in cystic fib

Abstract Background For people with cystic fibrosis and advanced pulmonary damage, lung transplantation is an available and viable option. However, graft rejection is an important potential consequence after lung transplantation. Immunosuppressive therapy is needed to prevent episodes of graft rejection and thus subsequently reduce morbidity and mortality in this population. There are a number of classes of immunosuppressive drugs which act on different components of the immune system. There is considerable variability in the use of immunosuppressive agents after lung transplantation in cystic fibrosis. While much of the research in immunosuppressive drug therapy has focused on the general p

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