Accelerating a pipeline of new medicines and diagnostics to help treat lung infections in people with Cystic Fibrosis.
People with CF experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body.
People with CF are susceptible to lung infections, which can be hard to detect and treat, as some infections are becoming resistant to antimicrobial medicines.
Many different types of bugs cause lung infections. While they are often harmless to people who don’t have CF, they can be a serious problem for people with CF.
Left untreated, these infections can trigger permanent lung damage, meaning people are more breathless and have less energy to do day-to-day activities.
This can include:
Ground-breaking treatment, known as triple-modulator therapy, is now available to help people manage their condition.
However, this treatment is not suitable for everyone and lung infections are still a problem for many.
People with CF are routinely treated with oral and inhaled antibiotics, which are necessary for two reasons:
2. In the long term, they help to suppress the growth of bacteria in the lungs.
Antimicrobials are vital to managing exacerbations and to help suppress or manage long-term chronic infections. Without these treatments, lung function and overall health, quality of life and life span of people living with CF are dramatically altered. Treatment failure due to antimicrobial resistance (AMR) is a frequent and significant challenge.
It is a continual race to develop new antimicrobial approaches where bacteria find it harder to develop resistance, and that can combat newly resistant strains.
Discovering new antimicrobials to treat the infections associated with CF is an urgent, unmet need.
These barriers affect how quickly innovators can develop and translate their research to the clinic.
Cystic Fibrosis antimicrobial development is challenging.
Here are some reasons why:
1. It can be difficult for drug developers to access insights from people with CF and therefore understand their needs for future treatments.
2. In CF, different clinical needs need to be targeted: eradication of infection, management of pulmonary exacerbations, and suppression of chronic infection.
3. New approaches to tackle the newly emerging resistant bugs are being discovered but the path to develop them into medicines is unprecedented.
4. The preclinical landscape for CF antimicrobials is rapidly evolving, with a lack of guidance and preclinical models that can accurately predict the clinical effect.
5. Researchers face difficulties accessing relevant samples on which to test and develop promising antimicrobial and diagnostic programmes.
To address this challenge, the CF AMR Syndicate was established in 2019.
The Syndicate is jointly managed by Medicines Discovery Catapult, Cystic Fibrosis Trust and LifeArc.
The CF AMR Syndicate is a cross-sector initiative that brings together leading experts in CF and AMR from industry, academia and the clinic with people living with CF.
The CF AMR Syndicate’s research agenda has been informed by extensive efforts to understand the research landscape and the needs of the CF infection research community.
To accelerate CF antimicrobial drug discovery and development, the Syndicate has catalysed collaborative R&D to build a suite of enabling tools and resources, including:
2. PIPE-CF, led by Dr Jo Fothergill from University of Liverpool, is a global consortia comprised of nine entities. Jointly funded by The Cystic Fibrosis Trust and the Cystic Fibrosis Foundation, this Strategic Research Centre is focused on developing an evidence-based preclinical framework for the development of novel antimicrobials in CF.
3. Patient-focused Target Product Profiles (TPPs) for CF-related infections, which provide guidance in CF antimicrobial and diagnostic discovery and development to innovators in industry and academia.
The CF AMR Syndicate’s newest initiative is the launch of a £3 million collaborative discovery programme.
The Syndicate is working with the community to identify and support antimicrobials R&D with the potential to meet the needs and priorities of people with CF.
The programme will be truly collaborative, providing wrap-around support throughout the project journey to enable the best chances of success for projects to attract further investment.
As a strategic partnership focused on this urgent, unmet need, the CF AMR Syndicate will continue to drive a pipeline of therapeutics and diagnostics through a collaborative drug discovery programme.
In 2022, the Patient Partnership Index awarded the CF AMR Syndicate GOLD standard in recognition of the Syndicate’s demonstration of best practice in working with people with CF.
The Syndicate’s proactive involvement of people with CF, recognising them as experts on their condition and bringing valuable knowledge, exemplifies how organisations can best engage and communicate with patients and the groups that advocate for them.
Anyone working in or interested in CF infection research can sign up to join the CF AMR Syndicate Network for free.
This includes people with CF and people from academia, NHS, industry, government and charity.
The network is open globally for anyone to join, and events are organised for, and in collaboration with, the CF AMR community on topics aimed at advancing the field.
The Syndicate aims to spark new collaboration so that, together as a community, we can bring new medicines to people who need them.
Become part of a collaborative network and help bring new treatments to people with CF faster.
Visit the CF AMR Syndicate website for more information.
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