Researchers discover disease-causing stem cells in lungs of cystic fibrosis patients

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.

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.

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

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.

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.

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.

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.

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.

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.