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Professor Marcus Mall from the Christiane Herzog CF Center in Berlin spoke at ERS Congress 2023 about the future of CF treatment being a new therapeutic approach.
Challenges ahead for new therapies for cystic fibrosis
Dr Mall delved into the research being conducted in people with CF, with a special focus on people with CF who are not on modulators. He listed the following challenges that science has ahead of it:
- Establish pharmacological approaches to restore CFTR function in people with CF ineligible for modulators.
- People with CF with mutations that synthesise some protein and may respond to modulators.
- Study of the response to modulators in vitro using human-derived models such as nasal tests or gut miniorganoids.
- Through compassionate use clinical therapy. France, with people with CF with FEV1<40% without deltaF508 is testing modulatory therapy: up to 54% are responders, thus improving their lung function, sweat test, etc. (Source: Burgel et al, ERJ 2023).
- For people with CF who do not synthesise protein (class I). Suppressors of premature termination codons (e.g. ELX-02) are being developed in mini-organoids and are being combined with modulators.
- For people with mutations that cannot be repaired pharmacologically, gene therapy and gene editing may be used.
- To increase the degree of restoration of CFTR function in modulator-eligible patients for optimal clinical benefit.
- Despite the use of modulators only a maximum of 40-50% is restored, i.e. there is improvement, but many abnormalities remain, and strategies must be employed to better restore this CFTR function.
- With gene editing, which makes it possible to correct individual CFTR mutations in the genome of the CF person’s cells, effective treatment for all people with CF is now on the horizon.
- Improving symptomatic therapies remains important, especially for people with chronic lung disease, and may be applicable to address mucus plugging, chronic airway inflammation and infection in other muco-obstructive lung diseases.
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