Emily’s Entourage reveals the results of their Final 10% Survey
It also found that knowing there are life-changing medications available but that they don’t work for everyone is creating further distress. However, the findings do show that many not eligible for modulators have or would take part in a clinical trial.
Carried out in 2022, the charity surveyed over 300 people affected by cystic fibrosis from 29 countries across the world, including the UK, who are part of the CF population that do not currently benefit from CFTR modulator therapies. The survey asked recipients questions about how the condition affects their life, their concerns, their opinions on treatment options, research studies and clinical.
Some of the key findings included:
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The most frequently reported symptom people living with CF experienced was an excessive cough (70%), followed by fatigue (61%), and pulmonary exacerbation and chronic lung infections (55%)
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70% of people with CF reported spending 1-4 hours a day on treatments
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22 respondents were eligible or had a family member eligible for therapies but 68% cited the side effects were the reason they were not taking them. 3% reported that they or their family member were taking a modulator “off-label”
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50% of people with CF responded saying that they had taken part in a clinical trial before and 83% expressed interest participating in a future clinical trial
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People with CF expressed that while a cure for the disease as a whole is needed, treatments to improve lung function (76%) and address lung infections and exacerbations (59%) are the most important aspects of CF for new therapies to address
The survey also highlights the thoughts and fears of being left behind by those who can’t access the treatments. A number of these were also from countries where modulator therapies are not yet available. You can read The Final 10% Survey Report here
What we’re doing to help
You’ve told us that your number one research priority is: What options are available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)? That’s why last year we announced a funding partnership with Emily’s Entourage to develop novel ways to deliver a genetic therapy for CF in the lab. If successful this research could lead to a treatment for everyone with CF in the future.
Cystic Fibrosis Trust are funding a number of lab-based research studies that could benefit people with rare mutations. We’re also working actively to improve the day-to-day symptoms of everyone with CF. This includes diagnosing and developing better treatments for infections that are becoming resistant to current medicines.
Early phase genetic therapy clinical trials are now recruiting a few people with CF in the UK. The treatments being tested are designed to improve the lung function of CF. They’re in the very early stages, but they hold a lot of promise for the future. You can take a look at genetic therapy clinical trials for CF on our Trials Tracker.
We’re dedicated to funding research that will benefit everyone with CF, regardless of their CFTR mutation. We won’t stop until everyone can live a life unlimited.
Here to help
If you’re struggling with any aspect of cystic fibrosis, we’re here to help. Contact our Helpline for support, a listening ear or just someone to talk to.