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   PID: hpt/lnx 1.9.0-cur 2019-01-08   
   TID: hpt/lnx 1.9.0-cur 2019-01-08   
    Triple combination therapy brings lasting improvement in cystic fibrosis   
      
      
     Date:   
         July 7, 2023   
     Source:   
         Charite' - Universita"tsmedizin Berlin   
     Summary:   
         The mucus in the airways is not as sticky, inflammation in the lungs   
         significantly reduced: Triple combination therapy can achieve these   
         positive, lasting effects in patients with cystic fibrosis (CF).   
      
         According to new research, this form of medication improves the   
         symptoms of CF in many patients.   
      
      
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   The mucus in the airways is not as sticky, inflammation in the   
   lungs significantly reduced: Triple combination therapy can achieve   
   these positive, lasting effects in patients with cystic fibrosis   
   (CF). Researchers from Charite' -- Universita"tsmedizin Berlin and the   
   Max Delbru"ck Center have just recently published their findings in the   
   European Respiratory Journal.* According to their research, this form   
   of medication improves the symptoms of CF in many patients.   
      
   Two years ago, a research group headed by Charite' showed that combination   
   therapy involving three drugs -- elexacaftor, tezacaftor, and ivacaftor   
   -- is effective in a large portion of patients with cystic fibrosis, a   
   hereditary disease, meaning that the treatment noticeably improves both   
   lung function and quality of life. Now, the team headed by Prof. Marcus   
   Mall, who has been the lead researcher in both studies, has investigated   
   for the first time whether this form of treatment is also helpful in the   
   long term, meaning over a period of 12 months or more. To examine this,   
   the researchers took a closer look at the sputum, the secretions from   
   patients' respiratory tracts. "In patients with cystic fibrosis, the   
   mucus in the airways is very sticky because it doesn't contain enough   
   water and the mucins, the molecules that form mucus, adhere too much due   
   to their chemical properties. This results in thick, sticky mucus, which   
   clogs the airways, making it harder for patients to breathe and leading   
   to chronic bacterial infection and inflammation of the lungs," explains   
   Mall, Director of the Department of Pediatric Respiratory Medicine,   
   Immunology and Critical Care Medicine and the Christiane Herzog Cystic   
   Fibrosis Center at Charite'.   
      
   In the current study, the researchers show that a combination of   
   elexacaftor, tezacaftor, and ivacaftor results in less viscous respiratory   
   secretions and decreasing inflammation and bacterial infection in the   
   lungs of cystic fibrosis patients. "What's more, the effects lasted   
   over the entire one-year study period. This is really important because   
   previous medications caused a rebound in the bacterial load in the   
   airways," explains Dr. Simon Gra"ber, who also works in the Department   
   of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine   
   at Charite' and was one of the co-leaders of the study.   
      
   79 adolescents and adults with cystic fibrosis and chronic lung disease   
   participated in the trial.   
      
   A major step in treating cystic fibrosis, further research important   
   "This is a major step forward in treating cystic fibrosis," Mall   
   says. "At the same time, it would be premature to say that patients have   
   been normalized, let alone cured. Chronic lung changes arising over many   
   years of living with the disease cannot be reversed, unfortunately." This   
   means patients with advanced lung disease will still need to rely on   
   established treatments involving inhaling mucus-thinning medications,   
   taking antibiotics, and physical therapy.   
      
   "We plan to forge ahead with our research on how to make treatments   
   that address cystic fibrosis via the molecular defects that cause the   
   disease - - like the triple medication combination studied here --   
   even more effective.   
      
   This includes starting treatment in early childhood with the goal of   
   preventing chronic lung changes wherever possible," Mall notes. "Aside   
   from that, this therapy is not available to about ten percent of our   
   patients right now due to their genetic conditions," Gra"ber adds. "That's   
   why we are also hard at work on research involving new molecular   
   treatments so we can treat all people with cystic fibrosis effectively."   
   The researchers are also working to advance their understanding of mucus   
   defects in cystic fibrosis and develop new mucolytics, drugs that thin   
   and loosen the mucus. This research could also benefit patients with   
   common chronic inflammatory lung diseases such as asthma and COPD.   
      
   Cystic fibrosis Cystic fibrosis is one of the most common fatal hereditary   
   diseases worldwide.   
      
   As many as 8,000 children, teens, and adults are living with the   
   disease in Germany today. An imbalance in salt and water transport   
   across mucosal surfaces of the body causes people with cystic fibrosis   
   to produce thick, sticky secretions that harm organs such as the lungs,   
   intestine and pancreas. This leads to progressive loss of lung function   
   and shortness of breath, which still significantly lowers life expectancy   
   despite advances in treatment. Some 150 to 200 children are born with   
   this rare disease in Germany each year.   
      
   About the triple combination therapy A combination of three drugs --   
   elexacaftor, tezacaftor, and ivacaftor - - became available in Europe in   
   August 2020. The therapy noticeably improves lung function and quality   
   of life in patients with the most common genetic defect involved in CF,   
   F508del. This means the treatment is an option for nearly 90 percent of   
   those living with cystic fibrosis. The combination therapy was approved   
   for children starting at the age of six years in early 2022.   
      
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   Worthy_of_a_Toast Story Source: Materials provided by   
   Charite'_-_Universita"tsmedizin_Berlin. Note: Content may be edited for   
   style and length.   
      
      
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   Journal Reference:   
      1. Laura Schaupp, Annalisa Addante, Mirjam Vo"ller, Kerstin Fentker,   
      Aditi   
         Kuppe, Markus Bardua, Julia Duerr, Linus Piehler, Jobst Ro"hmel,   
         Stephanie Thee, Marieluise Kirchner, Matthias Ziehm, Daniel Lauster,   
         Rainer Haag, Michael Gradzielski, Mirjam Stahl, Philipp Mertins,   
         Se'bastien Boutin, Simon Y. Graeber, Marcus A. Mall. Longitudinal   
         Effects of Elexacaftor/Tezacaftor/Ivacaftor on Sputum Viscoelastic   
         Properties, Airway Infection and Inflammation in Patients with   
         Cystic Fibrosis.   
      
         European Respiratory Journal, 2023; 2202153 DOI:   
         10.1183/13993003.02153- 2022   
   ==========================================================================   
      
   Link to news story:   
   https://www.sciencedaily.com/releases/2023/07/230707111622.htm   
      
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