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Dr Katherine Hisert spoke at the European Respiratory Society Congress in Milan about addressing the needs of people with CF after Kaftrio.
Needs of people with CF on and off modulators
Dr Hisert gave a focused explanation breaking down the needs of people with CF, whether they are on modulators or not, and the research opportunities that exist.
Since modulators remain a treatment and not a cure for the disease, two questions arise for those who take them:
- What is the new status of the disease?
- What challenges lie ahead for ageing patients?
However, for the rest of the people with CF who do not take them, we must continue to treat the disease as we have been doing so far and make progress in research, based on modulators or other types of therapies, to provide them with a similar quality of life to those who take modulators.
Surprising statistics
There are several reasons why people with CF cannot be treated with modulators: because of their mutations, because of intolerance or because of economic issues.
We talk about 10-30% of people with CF not receiving modulators in developed countries, but the reality is that ONLY 12% OF PEOPLE WITH CF WORLDWIDE ARE RECEIVING MODULATOR TREATMENT.
Increased survival in people with CF
The results of the model by Lopez et al, JCF 2023, suggest that treatment with triple therapy (Kaftrio: ELX/TEZ/IVA) can substantially increase survival in people with CF.
In addition, early initiation of this treatment may allow them to achieve a near-normal life expectancy (source: https://www.cysticfibrosisjournal.com/article/S1569-1993(23)00048-6/fulltext).
Research challenges
Many research projects are currently underway, aimed at solving the following challenges:
- Use of animal or pre-clinical models to characterise the effects of modulators during development (in utero and during infancy).
- Development of methodologies to monitor early lung disease, either with inflammatory and infection markers, imaging and X-ray technologies, etc.
- Personalisation of the dose of modulators through changes in the sweat test, mini-organoids or spheroids, biomarkers…
- Reduced treatment burden for people on modulator treatment.
- Changes in long-term patterns of inflammation and infection in people treated with modulators.
- Modification of other CF complications with modulators and possible new complications with age.
- Epidemiological studies and implementation of resources for the whole world, i.e. global neonatal screening, as well as diagnostic services, specialist care, etc.
- Development of gene therapies that restore CFTR gene function in all people with CF.
Progress is being made in all these areas, but there is still a long way to go.
#TogetheragainstCF
