Treating all of the symptoms of CF throughout the body

Cystic fibrosis can prevent the pancreas from producing insulin properly. Blood sugar levels are controlled by the release of insulin from a specific part of the pancreas. When the pancreas can’t produce enough insulin, blood sugar levels can rise and this can lead to CF diabetes (CFD). The underlying cause of CF diabetes is unknown. We want to prevent CF diabetes and improve how it is treated and managed. 

Professor James Shaw at the University of Newcastle led a Strategic Research Centre to investigate how signals from the rest of the pancreas affect how and whether CFD develops. Understanding more about these signals could lead to entirely new approaches to treating CF diabetes, avoiding the need for insulin injections.

Read about our SRC on how disease in the digestive-juice-producing part of the pancreas leads to CFD

People with CF experience a range of gut symptoms, including small and large intestinal blockages (DIOS and constipation), bloating, nausea and diarrhoea. These symptoms can prevent people with CF getting the calories they need, be extremely painful, embarrassing and disrupt day-to-day life. 

Treatments for gut symptoms of CF are often ineffective because doctors do not fully understand why symptoms occur. By recruiting people with CF with gut symptoms in our GRAMPUS CF SRC, the researchers aim to link specific gut symptoms to changes seen within the intestines. At the same time other colleagues within the SRC will conduct lab studies to understand what triggers these changes to take place and investigate possible symptom-specific medicines that may be effective to treat them. The SRC is led by Professor Alan Smyth at Queen’s University Belfast and the University of Nottingham, alongside a multidisciplinary team of researchers.

Read about the GRAMPUS CF SRC to develop more symptom-specific treatments for CF gut symptoms

We know that CFTR modulators such as Kaftrio can help improve the lung health of people with CF, but their effects outside of the lungs are less well understood. Through a VIA award the Trust supported the RECOVER study to understand the long-term impacts of Kaftrio throughout the body in people with CF as they first gained access to it. 

Professor Paul McNally and Prof Jane Davies, who led the RECOVER study, have now been awarded funding from Cystic Fibrosis Foundation to monitor the impacts of Kaftrio throughout the body in children as they start to take these medicines. Additional funding from the Trust through a VIA award has supported this study known as ENHANCE.