Researchers receive funding for proof-of-principle experiments investigating potential of cell replacement therapies for cystic fibrosis-related diabetes.
Researchers from the University of Birmingham have been awarded a £300,000 Development Award from the Cystic Fibrosis Trust to explore whether stem cell derived islet cell transplants could offer a feasible future treatment option for cystic fibrosis-related diabetes (CFRD).
As a result of medical advances, people with cystic fibrosis are living much longer, however as they age around half of patients develop CFRD. This develops as a result of secretions in the pancreas which damage the insulin producing cells, located in a drop of hormone secreting cells called the “islets”. Loss of islets makes it more difficult to control blood sugar.
Like patients with type 1 diabetes, people with CFRD often need to take multiple daily insulin injections. However, managing diabetes alongside cystic fibrosis can be challenging, as problems with absorption of food can make blood sugar levels unpredictable and high blood sugar can increase the risk of lung infections. There have been recent developments in treating type 1 diabetes patients using islet cells grown in a lab made from stem cells.
Dr Ildem Akerman, Associate Professor in Functional Genomics at the University of Birmingham, working alongside Dr Victoria Salem from King’s College London, will test whether it is possible to grow healthy, functional islet cells in the lab using stem cells that carry the gene mutation underlying cystic fibrosis (CF).
By introducing the cystic fibrosis gene mutation into stem cells, researchers aim to understand how it affects the development and function of pancreatic islet cells. This research could help determine whether people with cystic fibrosis may benefit from the stem cell therapies currently being trialled for type 1 diabetes. Unlike type 1 diabetes, CFRD is not associated with autoimmunity, which means that in future, lab-grown cells could be made from a patient’s own cells — removing the need for immunosuppression.
We believe that the tools we are developing to treat people living with type 1 diabetes could also benefit those with cystic fibrosis-related diabetes. We’re excited to begin this project and to leverage the infrastructure at BetaCell Birmingham to advance personalised, cell-based therapies for CF-related diabetes.
The project does not involve transplants yet but is a proof-of-concept study to see if it is possible to make these insulin producing cells in a lab from people living with the gene mutation of Cystic Fibrosis. Collaborators at Kings College London will explore whether other useful cells made from the patient’s blood could be used to improve the function and survival of the lab-made islet cells after transplant in future treatments.
Researchers hope that the work will ensure that patients with CFRD are included in the next generation of cell-based treatments, ultimately contributing to the effort to find a long-term treatment for CFRD.
Diabetes UK RD Lawrence Fellow
School of Medical Sciences
Staff profile for Dr Ildem Akerman, Diabetes UK RD Lawrence Fellow and Associate Professor in Functional Genomics at the Department of Metabolism and Systems Science, College of Medicine and Health.
