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Lovelace Biomedical Talks About Developing Novel Therapies for Cystic Fibrosis Month

May is Cystic Fibrosis (CF) month. Lovelace has been working in respiratory research for over 70 years, and has been using gene therapy studies to develop drugs to treat CF and other similar diseases for over 10 years. As a part of CF month, Lovelace’s expert scientists, Ted Barrett and Philip Kuehl are here to answer common questions related to drug development for CF treatment. We also get a look into the clinician’s perspective on the current state of CF, its therapies, and where it may be headed. From the Clinician: Carolyn Cannon, PhD, who is an Associate Professor at the Texas A&M College of Medicine, and is in the Microbial Pathogenesis & Immunology department, has been heavily involv

Phages, Viruses that Attack Bacteria, Are a Potential Therapy Against Infections, Case Report Sugges

The case report of a cystic fibrosis (CF) patient suggests that phages — viruses that naturally infect and kill bacteria — are a potential personalized therapy against infections caused by antibiotic-resistant bacteria. The study, “Engineered bacteriophages for treatment of a patient with a disseminated drug-resistant Mycobacterium abscessus,” was published in the journal Nature Medicine. Patients with CF frequently have long-lasting bacterial infections in the airways. Sometimes, these bacteria are resistant to antibiotics, which makes them difficult to manage. Antibiotic-resistant bacteria pose a particular risk to CF patients undergoing lung transplant, because they can flare during the i

SNSP113 (Previously SYGN113)

SNSP113 is an inhaled therapy being developed by Synspira Therapeutics that aims to ease pulmonary infections, airway congestion, and inflammation. SNSP113 was designated an orphan drug as a potential treatment of pulmonary decline in cystic fibrosis (CF) orphan medicinal product How SNSP113 works SNSP113 contains an active ingredient called poly N (acetyl, arginyl) glucosamine, which is a type of complex sugar molecule. The treatment is reported to have a multi-faceted mechanism of action that might protect pulmonary function in CF patients: Targeting and breaking apart bacterial biofilms. These are communities of bacteria that are attached to the airway epithelium and are usually resistant

Incidence of CF-related Diabetes Stable in Pediatric CF, Canadian Study Finds

The incidence rate of cystic fibrosis-related diabetes (CFRD) in children with cystic fibrosis (CF) has remained stable over time, according to a Canadian multi-center registry study. According to the researchers, these findings contrast with increasing incidence rates observed for type 1 and type 2 diabetes in the general population. The study “Incidence and risk factors of paediatric cystic fibrosis-related diabetes” was published in the Journal of Cystic Fibrosis. Nearly half of all CF patients develop CFRD, and the condition typically becomes more prevalent as patients age. Although CFRD shares some features with both type 1 (impaired insulin production) and type 2 (insulin resistance) d

Phage viruses tailored in Pitt lab used to treat patient with drug-resistant infection Phage viruses

A cocktail of viruses developed at the University of Pittsburgh to precisely attack life-threatening bacteria resistant to antibiotics was successfully used to treat a young double-lung transplant patient in London, according to research reported Wednesday. It appears to be the first use of engineered viruses known as bacteriophages to fight a Mycobacterium infection in a human — in this case, a 15-year-old girl with cystic fibrosis and other serious conditions, including diabetes and liver disease. “Phage-hunting” college students were behind the discovery of the phages that could kill the Mycobacterium abscessus that had spread throughout the girl’s body after her transplant. A more widesp

Early, Rapid BMI Increase May Affect Overall Growth of CF Children, Study Suggests

Rapid weight gain early in life may have a long-term negative impact on the growth of children with cystic fibrosis (CF), a study has found. This finding suggests that nutritional interventions aimed at supporting the child’s normal growth should be implemented early on, taking into consideration their weight and overall body mass index. The study, “Rapid early increase in BMI is associated with impaired longitudinal growth in children with cystic fibrosis,” was published in the journal Pediatric Pulmonology. Many patients with CF struggle with low body weight due to poor nutrient absorption related to their disease. To overcome this, high-fat, high-protein diets are often recommended for pa

Poor Oxygen Conditions May Promote P. aeruginosa Infection Over Other Pathogen in CF, Study Suggests

Poor oxygen conditions, a common trait in the lungs of cystic fibrosis (CF) patients, may help Pseudomonas aeruginosa bacteria outcompete other infectious agents, namely Staphylococcus aureus, a study suggests. The study, “Anaerobiosis influences virulence properties of Pseudomonas aeruginosa cystic fibrosis isolates and the interaction with Staphylococcus aureus,” was published in the journal Nature Scientific Reports. Opportunistic pathogens often infect CF patients. Staphylococcus aureus is one of the first bacteria to colonize the lungs of a child with CF, and the most common infection in the first decade of their life. Pseudomonas aeruginosa, another common bacteria in CF, tend to colon

Incidence of Non-CF Bronchiectasis is Rising in Germany, Study Reports

The incidence of non-cystic fibrosis bronchiectasis is rising in Germany, which necessitates a better understanding of the economic burden of the disease, a new study reports. The study, “Incidence of patients with non-cystic fibrosis bronchiectasis in Germany – A healthcare insurance claims data analysis,” was published in the journal Respiratory Medicine. Bronchiectasis is a chronic pulmonary disease that is characterized by permanently dilated airways due to chronic bronchial inflammation. A common and well-known cause of bronchiectasis is cystic fibrosis (CF). However, bronchiectasis can also be caused by conditions other than CF, which is referred to as non-cystic fibrosis bronchiectasi

Decreasing Acidity of Airway Liquid in Newborn CF Patients May Reduce Infection Risk, Study Suggests

Normalizing the acidity in the thin layer of liquid covering the airways of newborns with cystic fibrosis might make them more resistant to bacterial infections, a study has found. The study’s researchers also identified two proteins, pendrin and ATP12A, that could be targeted to regulate acidity levels of the airway liquid in these infants, which is “significantly” more acidic in CF patients than in healthy people based on the pH scale. The study, “Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis,” was published recently in the journal Nature Scientific Reports. Airway secretions show evidence of Staphylococcus aureus (S. aureus) infection in chil

FDA Approves Kalydeco for Treatment of 6- to 12-month-old Infants with Cystic Fibrosis

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for the treatment of infants from six to 12 months old, who have cystic fibrosis (CF) and at least one mutation in the CFTR gene, announced the therapy’s developer, Vertex Pharmaceuticals. With the potential to treat the underlying cause of CF, the twice-daily oral therapy is designed for CF patients with specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in the production of a defective CFTR protein and impaired movement of salts in and out of cells. “Today’s approval for Kalydeco allows physicians to begin treating the underlying cause of CF in eligible infant

Breathing space: Inhalable biologics interest prompts Intertek expansion

Interest in inhaled biologics is increasing says Intertek, which has announced plans to expand its testing facility in Melbourn, near Cambridge. Intertek will double capacity with a new 20,000 square-foot lab kitted out with technologies use to assess inhaled and nasal formulations of large molecule drugs. It also plans to hire 100 employees over the next four years. “As companies are increasingly looking at biologics for non-invasive delivery platforms like inhaled and nasal, there is expected to be an increased demand,” Chris Vernall, business development director, Intertek Pharmaceutical Services, told Bioprocess Insider. “This is something we are already seeing at Intertek Melbourn and h

How to Love Your Body, Even If It’s Genetically Abnormal

When we say “me” or “I,” what exactly are we referring to? Are we referring to our bodies, our minds, or the combination of the two? It’s both a scientific and philosophical question, who we are. In a casual manner, we often speak in the first person when referring to the being that is both the body and the mind. When we are ill, annoyed, or even just joking, we will often say, “My body has betrayed me.” But what does it mean when our bodies are working against us? Within my body, wars are raging every single day. The cells in my body — those with dysfunctional CFTR function, which causes the multitude of other problems I face — are the same ones that are part of the fight against bacteria a

Diabetes and Insulin Treatment Adds to CF Burden and Ways Are Needed to Ease It, Study Says

Having to take insulin to treat cystic fibrosis-related diabetes (CFRD) adds to patient distress and sense of a poorer quality of life, a study says, alerting experts to a need to find approaches that might ease this burden. The findings were described in the study “The impact of cystic fibrosis-related diabetes on health-related quality of life,” published in the Journal of Cystic Fibrosis. Diabetes is a common complication of CF, affecting 40% to 50% of adult patients, and one impacting lung function, weight, and survival. CFRD is caused by the accumulation of sticky, thick mucus, which is characteristic of CF, scarring and injuring the pancreas — the organ where insulin, a hormone key to

Pseudo‐Bartter syndrome in children with cystic fibrosis

Abstract Cystic fibrosis (CF) is an autosomal recessive genetic disorder. We report a case of a boy aged 4.5 years with cystic fibrosis, presenting under‐weightness, hypocalcemia, metabolic alkalosis, hypokalemia, and hyponatremia. Sweat analysis of the patients concluded pseudo‐Bartter syndrome, which was successfully treated using antibiotics, physiotherapy, fluids, vitamin supplements, and pancreatic enzyme therapy. 1 INTRODUCTION Cystic fibrosis (CF) is a genetic disorder with autosomal recessive inheritance pattern due to the mutation in CFTR (cystic fibrosis transmembrane conduction regulator), which is responsible for transferring chloride ions into the epithelial membrane of the orga

Abeona Therapeutics to Report New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 fo

NEW YORK and CLEVELAND, April 30, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today will report new preclinical data demonstrating that ABO-401, the Company’s novel gene therapy for cystic fibrosis (CF), efficiently delivered a highly-expressed, functional copy of human mini-CFTR (hCFTR) to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells. The data will be presented this evening at the American Society of Gene and Cell Therapy 22nd Annual Meeting in Washington, D.C. “These encouraging preclinical data add to the growing body of evidence suggesting ABO-401 may addre

We Must Pay Closer Attention to the Global CF Population

Based on the stringent qualifications the Cystic Fibrosis Foundation uses to determine who has cystic fibrosis, approximately 70,000 people currently have CF worldwide. That includes 30,000-35,000 in the United States. In this broad population, many subpopulations fit neatly into the CFF’s qualifications. Others do not. As much as we all wish it were different, CF is not the simple disease it was once believed to be. I wrote about this spectrum of the disease a few months ago. Essentially, the thesis of that piece was that CF is not a monolithic disease that affects everyone the same. The most common subpopulation of CFers consists of those with two copies of the ∆F508 allele. This mutation

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