Two nationally recognized experts in cloning and stem cell science from the University of Houston, Wa Xian and Frank McKeon, are reporting that five lung stem cell variants dominate the lungs of patients with advanced cystic fibrosis (CF), and that these variants drive key aspects of CF pathology including inflammation, fibrosis and mucin secretion.
Tag: Cystic Fibrosis
New international guidelines will improve cystic fibrosis treatment
A team of global experts have spent a decade creating a guide to clinical assessments which help inform exercise training for people living with cystic fibrosis.
Diagnosis of Cystic Fibrosis Often Missed or Delayed, Especially in Non-White Infants
Ann & Robert H. Lurie Children’s Hospital of Chicago is leading an awareness campaign that aims to reduce missed or delayed diagnosis of cystic fibrosis after newborn screening, especially in non-White infants. In its first phase, the campaign targets primary care providers and public health officials, so that treatment can start earlier, which is linked to better outcomes for people with cystic fibrosis. The general public phase is expected to follow within the year.
Starting CFTR modulator therapy in mother with cystic fibrosis associated with less severe complications in the fetus
A woman with cystic fibrosis was treated with elexacaftor-tezacaftor-ivacaftor (ETI), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, during pregnancy and gave birth to a healthy baby after experiencing complications in two previous pregnancies. The case report is published in Annals of Internal Medicine.
Cystic fibrosis drug could help treat pneumonia
Pathogens such as SARS-CoV-2 and pneumococcus can cause severe pneumonia. If the airways then fill with fluid, the patient risks developing acute respiratory distress syndrome.
Study Finds Delays in Initial Cystic Fibrosis Evaluation in Infants of Color
Infants from minoritized racial and ethnic backgrounds who have positive newborn screening tests for cystic fibrosis received their diagnostic follow-up for the disease later than recommended and later than white, non-Hispanic infants, according to a study published in the Journal of Cystic Fibrosis. The study also found that this delay in diagnosis and treatment was associated with worse early nutritional outcomes and may contribute to previously documented, considerable health disparities in people with cystic fibrosis.
Hormone Infusion Improves Pancreatic Insulin Production in Cystic Fibrosis Patients with or at Risk for Diabetes
Medication therapy based on the hormone glucagon-like peptide-1(GLP-1) may help regulate natural insulin production in cystic fibrosis, potentially offering a better way to prevent and ultimately manage diabetes than daily insulin injections
A New Treatment Approach for Cystic Fibrosis
Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells.
Unprecedented Case Series Advances Promise of Phage Therapy
An international team of researchers, led by scientists at University of California San Diego School of Medicine and the University of Pittsburgh, report promising results from the largest case series yet of patients treated with bacteriophage therapy for antibiotic-resistant infections.
Pitt researchers led the largest-ever series of phage therapy case studies
In a new paper published today in the journal Clinical Infectious Diseases, a team led by researchers from the University of Pittsburgh and the University of California San Diego report 20 new case studies on the use of the experimental treatment, showing the therapy’s success in more than half of the patients.
Stem Cell Cloning Experts Unraveling Cystic Fibrosis
Two nationally recognized experts in cloning and stem cell science from the University of Houston are taking the first step toward limiting the consequences of chronic inflammation in cystic fibrosis (CF) by identifying the source of this persistent and enigmatic inflammation in CF lungs.
16-Year-Old with Cystic Fibrosis Receives Double Lung Transplant, a First for Hassenfeld Children’s Hospital at NYU Langone
Hassenfeld Children’s Hospital at NYU Langone and the NYU Langone Transplant Institute reached a historic, first-ever milestone when surgeons replaced the lungs of a 16-year-old girl with cystic fibrosis.
Lower Airways Are Distinct in Cystic Fibrosis Even at Younger Ages
In the largest study of its kind, researchers found that the lower airways in children with cystic fibrosis (CF) have a higher burden of infection, more inflammation and lower diversity of microorganisms, compared to children with other illnesses who also have lung issues. They noted a clear divergence in these bacterial communities in toddlers, which is typically before progressive lung disease takes hold in patients with CF. Their findings, published in the journal PLOS ONE, could help providers target specific pathogens earlier, treat them and potentially prevent more severe lung disease.
Novel Insights on COVID-19 Vaccines and Virus Evolution, Artificial Intelligence in the Clinic, Miniaturization of Diagnostic Platforms, and Much More to Be Explored at the 2021 AACC Annual Scientific Meeting & Clinical Lab Expo
At the 2021 AACC Annual Scientific Meeting & Clinical Lab Expo, laboratory medicine experts will present the cutting-edge research and technology that is revolutionizing clinical testing and patient care.
Researchers Explore Potential Platform to ID Patient-specific Therapies for Cystic Fibrosis
Article title: Patient personalized translational tools in cystic fibrosis to transform data from bench to bed-side and back Authors: Kavisha Arora, Fanmuyi Yang, John Brewington, Gary McPhail, Alexander R. Cortez, Nambirajan Sundaram, Yashaswini Ramananda, Herbert Ogden, Michael Helmrath, John P. Clancy,…
Scientists Demonstrate Promising New Approach for Treating Cystic Fibrosis
Scientists created a potentially powerful new strategy for treating cystic fibrosis and potentially other diseases; it involves small, nucleic acid molecules called oligonucleotides that can correct some of the gene defects that underlie CF but are not addressed by existing modulator therapies.
Cystic fibrosis: towards better treatment and stronger lungs
In their lab, CRCHUM scientists Emmanuelle Brochiero and Damien Adam are studying ways of better predicting the efficacy of medications and helping injured lungs repair and regenerate.
‘Sweat sticker’ diagnoses cystic fibrosis on the skin in real time
A Northwestern University-led research team has developed a novel skin-mounted sticker that absorbs sweat and then changes color to provide an accurate, easy-to-read diagnosis of cystic fibrosis within minutes.
Three-Drug Therapy for Most Common Genetic Cause of Cystic Fibrosis Found Safe and Effective in 6-11-Year-Olds
An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90 percent of the cystic fibrosis population in the United States.
Understanding lung infections in patients with cystic fibrosis
Staphylococcus aureus (which includes MRSA) is the most prevalent organism isolated from the airways of children with cystic fibrosis (CF), and is treated using antibiotics, but its role in lung disease is poorly understood
Genetic Mutation Could Worsen Heart Function in Duchenne Muscular Dystrophy Patients
DALLAS – Nov. 4, 2020 – A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients’ lives.
CLEVELAND CLINIC UNVEILS TOP 10 MEDICAL INNOVATIONS FOR 2021
An up-and-coming gene therapy for blood disorders. A new class of medications for cystic fibrosis. Increased access to telemedicine. These are some of the innovations that will enhance healing and change healthcare in the coming year, according to a distinguished panel of clinicians and researchers from Cleveland Clinic.
In conjunction with the 2020 Medical Innovation Summit, Cleveland Clinic announced the Top 10 Medical Innovations for 2021.
Scientists Discover Curious Clues in the War Between CF Bacteria
This research shows that both Pseudomonas and Burkholderia use toxic weaponry, called Type VI Secretion Systems (T6SS), to compete with and establish dominance over each other. It could be possible to target or mimic this weaponry to defeat the bacteria before they cause irreparable lung damage.
Could a Urine Test Help Assess Aspects of Cystic Fibrosis?
• Studies conducted in mouse models, patients with cystic fibrosis, and different types of cells have helped to define how cystic fibrosis affects the kidneys.
• A urine test may help to assess aspects of cystic fibrosis in patients, which may be useful for testing the effectiveness of new medications.
ACADEMY OF NUTRITION AND DIETETICS RELEASES PRACTICE GUIDELINE: NUTRITIONAL NEEDS OF PEOPLE WITH CYSTIC FIBROSIS
The Academy of Nutrition and Dietetics has released a comprehensive guideline to help registered dietitian nutritionists address the needs of people with cystic fibrosis who are at risk of developing nutrition problems commonly associated with pulmonary disorders.
Compound in Red Wine, Grape Juice May Limit Inflammation in Cystic Fibrosis
Article title: Resveratrol restores intracellular transport in cystic fibrosis epithelial cells Authors: Binyu Lu, Deborah A. Corey, Thomas J. Kelley From the authors: “Though further studies are necessary, the ability of resveratrol to reverse [cystic fibrosis] cellular phenotypes in a manner not…
Henry Ford Health System Receives $25 Million Gift, Largest Single Donation in its History
Nationally-known developer Chris Jeffries and his wife Lisa have donated $25 million to Henry Ford Health System, the largest single gift from an individual in the health system’s 105-year history. This historic gift will rapidly accelerate the growth and expansion of Henry Ford’s Precision Medicine program, with the ultimate goal of creating a Precision Health Center. The efforts will have a robust focus on the advancement of cancer research and treatment, while also expanding to other medical specialties treating behavioral health, cardiovascular and metabolic diseases.
Researchers Identify Molecule Cluster That Improves Mucus Hydration in Cystic Fibrosis
Article title: Inhibition of ATP hydrolysis restores airway surface liquid production in cystic fibrosis airway epithelia Authors: Catharina van Heusden, Brian Button, Wayne H. Anderson, Agathe Ceppe, Lisa C. Morton, Wanda K. O’Neal, Hong Dang, Neil E. Alexis, Scott H.…
Modifier Gene May Explain Why Some with Cystic Fibrosis are Less Prone to Infection
People with cystic fibrosis who carry genetic variants that lower RNF5 gene expression have more mutant CFTR protein on cell surfaces. Even if the CFTR protein isn’t fully functional, it’s better than none, and may explain why some with cystic fibrosis are less prone to infection than others.
Three-drug combo improves lung function in most common genetic form of cystic fibrosis
A phase three clinical trial that UT Southwestern participated in determined that a three-drug combination improved lung function and reduced symptoms in cystic fibrosis (CF) patients who have a single copy of the most common genetic mutation for the disease.
FDA approves drug that improves lung function in most Cystic fibrosis patients.
Steve Rowe, M.D., professor of medicine at the University of Alabama at Birmingham and director of the Gregory Fleming James Cystic Fibrosis Research Center, is available to discuss the FDA approval of Trikafta, a new triple combination therapy for CF. …