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Timeframe for influencing the biological processes in the lung

Dear Ecorn-CF team,
in this article it says:
"Also for the treatment of CF the study gives an important hint: while treatment of older CF-patients is only possible to a small extent, there is obviously a timeframe in smaller children, in order to influence the biological processes in the lungs favourably in order to improve the further course of the illness."
As we have a 2-year-old child with CF, I would like to know how this process can be influenced in a positive way.
Many thanks and best regards,
Dear parents,
Thank you for this quite important question!
The research of the lung initiated by Prof. Tuemmler, is based on a list of questions:
- Is the lung of a CF patient at birth totally healthy?
- Is there a colonization with illness-causing germs like bacteria, viruses and fungi already at birth?
- Is a regenration of certain defects in the lungs of a small child possible?
- When is the maturation of the lungs finished?

Already before birth, morphological changes of the structures of the airways are assumed due to the pahtology of the CFTR-channel. This is only visible after birth in the form of overinflation of the lungs and widening of the bronchi. However, clinically the CF-child is totally free of symptoms, except probably an increased frequency of breathing.
The studies done by Prof. Marcus Mall, PD Olaf Sommerburg and Prof. Matthias Kopp (universities of Heidelberg and Lübeck), could show even at the age of 3-4 months in young children with CF changes of the bronchi measured by MRI investigations of the lungs, without clinical symptoms of the chidren.
Due to the dysfunction of the CFTR-Chanel, there are early infections with consecutive inflammation and increased mucus production. The consequence is an early beginning remodelling of the lung structures.
The lacking CFTR-function worsens the muco-ciliary transport. The important cilia do not have the fluid film for performing the necessary clearance of the bronchial system. Therefore other problems result. The pH-value is decreased in the fluid film. This has a negative influence on the inhibition of the anti-microbial activity. Therefore an increased growth of bacteria resulting in a chronic bacterial colonization. This might answer your first two questions.
In case of an early starting therapy, regenration could be possible, this is therefore the basis for a good lung function.
The maturation of the lung is nearly finished at the age of 3-4 years, no later than in the 7th year of life. Until then, regenerations could be successful. The lung research of Prof. Tuemmler will for sure be able to show more important facts.
Now to the question, what can be proposed in the first years of life, apart from the know therapy modules. Helpfuls are the AWMF guidelines for children in the first years of life, partly this is also true for the following years.
The thickened mucus on the bronchi can be made more fluid by the inhalation of certain substances: hypertonic saline and dornase-alpha, also addition of beta-2 mimetics have a positive influence.
At this early age, the following therapy modules have to be taken into account:
- physiotherapy
- inhalative therapy
- strict and frequent control of the microbial status (throat swab)
- early treatment with an antibiotic, if needed
- surveillance with imaging (x-ray of the thorax, MRI of the lungs)
- substitution with pancreatic enzymes
- surveillance of the inflammation activity, the breathing frequence, the vitamin D-, E- and A- level, the blood cell count, the length and weight
- early beginning of sports/physical activities
- climatic stays, e.g. at the North Sea, the Canary Islands, The Cape Verd Islands
- complete vaccination status and also vaccination against the flu, in the future also vaccination against Covid 19

In general, a huge influence can be expected by the intake of CFTR-modulators. In case of certain CFTR-mutations it can be taken from the 6 th month of life, in case of others from the 2 nd year of life on.
Please discuss such a treatment with your CF centre according to the CFTR-mutation status of your child.
Alone the initiation of the newborn screening led to a siginificant improvement concerning the body mass index, the lung function and a decreased infection with Pseudomonas. With this, the survival rate increased markedly. The consequence of the newborn screening war to initiate a targeted therapy very early.
In my opinion an early starting physiotherapy and a consequent inhalation therapy with hypertonic saline and dornase-alpha is a key issue in the possible regeneration of the lung. Those therapies should not be started when the first symptoms occur, but early after birth for prevention.
A very new and effective treatment in order to influence the course of the illness early, is the usage of CFTR-modulators, that can (at least partially) restore the function of the impaired CFTR-chanel. Certain modulators can already be used in the first year of life, and in the future, further modulators will be developed that can be used in a more and more larger and younger population of patients. The treatment with those drugs as early as possibel in addition to the standard therapy will lead to an early intervention in the course of the illness and will lead to milder courses of the illnesses and an increased life expectancy.
The time frame is open in the first years of life. During this time, biological processes can be influenced favourably with those therapeutic options.
Best regards,
Dr. H.-E- Heuer