Where are we now?
Cystic fibrosis is caused by mutations in the CFTR gene. Some effective treatments for people with specific mutations, known as 'precision medicines', have now been developed, and more drugs are being tested in clinical trials. By 2021 there could be an effective treatment that would work for around 95% of people with CF in the UK.
If they are licenced, these treatments could have a dramatic impact on the lives of people with CF (and have begun doing so already) by improving their life expectancy, reducing the number of lung infections and improving their quality of life.
However, the era of precision medicines brings with it new challenges which the research strategy aims to help solve.
What are the challenges and how are we solving them?
The best treatment for you
If and when access to new precision medicines is approved, people with CF of different ages will be affected by their condition in different ways. For example, older people with CF may have more lung damage, or complications such as CF-related diabetes (CFRD). This means that the way that their CF is managed will be unique to them, and treatments will need to reflect this.
Waiting for the right drugs
The development of precision medicines for people with CF is a huge leap forwards, but there are a number of reasons we shouldn’t be complacent. Not only are we still campaigning tirelessly with the community for access to these medicines, but there is also room for improvement in the drugs that are licenced now – by tweaking the drug design we could improve how they work and reduce the side-effects, creating the ‘next generation’ of precision medicines. It’s also important to keep the interest of the drug companies involved, as they may decide to work on easier (and more profitable) disease areas if we don’t. We are also still looking to develop drugs for all that would work for anyone with CF, whatever their genotype. This would be of particular benefit to people with rare mutations or those for whom the current drugs don’t work or cause side effects.
CF and diseases of older age
As the general population gets older, they start to become affected by a range of different diseases and conditions. The same will be true for people with cystic fibrosis. It isn’t known how things like cancer, diabetes, heart disease and social wellbeing will affect people with CF and how to reduce their impact.
Altering the CFTR gene
Rather than using drugs to correct the function of the CFTR protein, another area of research underway is looking to correct the CFTR gene itself, either by delivering healthy, non-mutated copies of the gene using gene therapy, or by permanently correcting the existing damaged copy of the gene using gene editing.
Treating lung infections
People with CF can have a range of different lung infections, and it’s important to know which infections are present and how to treat them. The earlier the infections are detected, the better chance there is of effectively treating them. Understanding how bacteria work can lead researchers to spot weaknesses that can be targeted with drugs.
Repairing damaged lungs
As the lungs get more damaged, for many with CF a lung transplant may become the only treatment option, but there are sadly not enough donor lungs readily available. Researchers are investigating whether a combination of stem cell technology and gene editing
could replace damaged lung cells, regenerating the lungs and providing an alternative to lung transplant.
How will we do it?
Balancing our funding
Over the last five years, we have principally supported a balance between:
- response-mode research - funding proposals based on the research areas proposed by researchers
- strategic research - identifying the research areas to be funded, in consultation with our strategy advisory board
New strategic investments growing out of the previous research strategy include the Clinical Trials Accelerator Platform, SmartCareCF and the Innovation Hub based in Cambridge.
Over the coming five years, we will continue this balanced approach but we are now well-positioned to grow our strategic investments through the development of strong partnerships with external funders and the biopharmaceutical, biotechnology and HealthTech industrial sectors.
Using existing funding schemes
Our 2013-2018 research strategy created new approaches for conducting research, which included setting up multidisciplinary, collaborative and international research programmes called Strategic Research Centres (SRCs) and co-investing with external funding bodies to progress translational research in CF through our Venture and Innovation Awards (VIAs). We will continue to fund research using these funding schemes to fund both response and strategic mode funding.
Integrating our flagship programmes
The Trust is continually developing a number of flagship programmes, including the UK CF Registry, the Clinical Trials Accelerator Platform, SmartCareCF and the Innovation Hub. Integrating the activity across these programmes helps us to move towards personalised medicine for people with cystic fibrosis.
One example of this is our SmartCareCF digital healthcare programme. Machine-learning algorithms for Registry data could inform which data people with CF collect on the programme. In turn, data collected as part of the SmartCareCF programme may be efficient at predicting the benefits of a drug in a CTAP co-ordinated clinical trial.
Working with you
The CF community is at the heart of everything we do, and we will ensure they are able to continue to help shape our research agenda by ensuring they are represented on our funding panels. We will also continue to improve how we communicate the research we’re funding, progress made and how this investment makes a difference to people affected by cystic fibrosis and their families.
Taking opportunities to participate in wider programmes
Areas of research that are important for managing and treating cystic fibrosis are also priorities in wider strategy and policy initiatives. For example, antimicrobial resistance, accelerated access to medicines and personalised healthcare, which are all of importance in cystic fibrosis, are also the focus of Government and NHS initiatives in a wide range of health conditions. The research strategy aims to harness these initiatives to accelerate progress in CF research.
Read about our research strategy and the achievements from our last strategy.
Read our new strategy