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.

* RELATED_TOPICS
o Health_&_Medicine
# Cystic_Fibrosis # Lung_Disease # COPD #
Diseases_and_Conditions # Lung_Cancer # Wounds_and_Healing
# Patient_Education_and_Counseling # Alzheimer's_Research
* RELATED_TERMS
o Cystic_fibrosis o West_Nile_virus o Pulmonary_embolism o
Mucous_membrane o Epilepsy o Insulin o Bronchitis o Therapy_dog

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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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