PTC Therapeutics, Inc. announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine. The published data show that treatment with ataluren, an investigational new drug, resulted in statistically significant improvements in the production and function of cystic fibrosis transmembrane conductance regulator (CFTR), a critical protein lacking in CF patients. "We are encouraged by the results of this study, which show that ataluren is pharmacologically active and generally well tolerated in children with nonsense mutation cystic fibrosis, " said Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'Hopital Necker-Enfants Malade. "There is a great need for new cystic fibrosis treatments to help prevent disease manifestations, particularly in younger patients.
Data Mining Method Helps Hospitals Better Identify Needs Of Their Sickest Pediatric Patients And Could Reduce Health Care Costs
Children with chronic health conditions such as cystic fibrosis, type 1 diabetes, sickle cell diseases and cerebral palsy represent less than two percent of the population but can consume more than 50 percent of resources at children's hospitals throughout the country. Coordinating care for these children has historically been difficult because hospitals have varying methods to identify them in their systems. In a new study led by John Neff, MD, of Seattle Children's Research Institute, researchers developed a unique method to identify children with serious lifelong chronic conditions using hospital discharge data that will enable children's hospitals to improve the quality of care for these patients and reduce costs. The study, "Identifying Children with Lifelong Chronic Conditions for Care Coordination Using Hospital Discharge Data, " published online November 15 in Academic Pediatrics.
An investigational drug targeting a defective protein that causes cystic fibrosis has been shown to improve lung function in a small study of CF patients, according to findings published Nov. 18, 2010, in the New England Journal of Medicine. The investigational drug, VX-770, appeared to improve function of what is known as CFTR--the faulty protein responsible for CF. It is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis. Patients who took VX-770 for 28 days showed improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. Excessive sweat chloride is a key clinical indicator of CF. The sweat test is the traditional diagnostic test for CF.
Common Strain Of Bacteria Found In Patients With Cystic Fibrosis In Canada; Associated With Greater Risk Of Death
A common transmissible strain of the bacteria Pseudomonas aeruginosa has been identified among cystic fibrosis (CF) patients in Canada, suggesting that cross-infection has occurred widely between CF centers in the United Kingdom and Canada, according to a study in the November 17 issue of JAMA. Infection with this strain among Canadian CF patients has been associated with an increased risk of death or lung transplantation. There is variability in the type and timing of outcome among CF patients who are infected with P aeruginosa; some patients experience a rapid decline in pulmonary function after infection and others harbor the organism for extended periods without any obvious adverse effects. The marked difference in prognosis among patients with P aeruginosa has not been adequately explained, but it may be due in part to differences among infecting strains, according to background information in the article.
New England Journal Of Medicine Publishes Phase 2 Study Of VX-770 As A New Approach To Treat The Underlying Cause Of Cystic Fibrosis
In a study published in this week's New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase 2 clinical trial of 39 people with cystic fibrosis (CF). There were no discontinuations due to adverse events in the study, and the frequency of adverse events was similar across the study groups. VX-770 is an oral (tablet) medicine that is being developed by Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) to directly target the defective protein known to cause CF. An accompanying editorial on this study was also published in this week's New England Journal of Medicine. "This study marks a significant step in the development of innovative CF therapies that target the defective protein known to be the underlying cause of CF, " said Frank Accurso, M.