Developing autologous stem-cell derived therapies for CF diabetes Development Award
CF diabetes is a difficult illness to manage alongside other CF symptoms, increasing the burden of treatment and risk of long term complications. In this Development Award, Dr Ildem Akerman and Prof Victoria Salem are exploring a new way to treat it.
Summary: Could beta cell transplants work for CF diabetes?
Diabetes occurs when the body can’t regulate the level of sugar in the blood. There are different types of diabetes, and people with CF develop a specific form of diabetes known as CF diabetes or CF-related diabetes. In people who develop this condition, the pancreas can’t release enough insulin and the body may respond differently to the insulin that is released.
Beta cells are the cells in the pancreas that produce insulin, a hormone important for regulating blood sugars. The signs and symptoms of CF diabetes share similarities to both Type 1 and Type 2 diabetes, but the underlying cause is thought to be different.
Elsewhere, researchers are exploring whether lab-grown beta cell transplants could be used as a treatment for Type 1 diabetes. Research in this Development Award will explore whether this could be a feasible approach to treat CF diabetes. If it works, it could mean that people with CF might not have to take insulin in the future.
The research programme in more detail
Dr Akerman from the University of Birmingham and Prof Victoria Salem from King’s College London have been pioneering how to grow new insulin-producing cells from stem cells in the lab, and how to transplant these new cells into people with Type 1 diabetes. The aim of these transplants would be to remove the need for insulin therapy.
Dr Akerman and Prof Salem have been awarded a Development Award to extend their research to find out if a similar approach could work for CF diabetes.
This is an early stage research study. Before this type of treatment is ready to be tested in people with CF, there are two questions that need to be answered in the lab.
- Is it possible to grow beta cells from people with CF?
- Would a beta cell transplant be successful?
More detail about each of these questions is given below.
Is it possible to grow beta cells from people with CF?
For a CF diabetes beta cell transplant in the future, the cells used for each individual’s treatment would originally have come from them. This is known as an ‘autologous transplant’. The reason for doing this is to avoid the need for immunosuppressive treatment.
Every cell from someone with CF will contain CFTR variants. The first step in this research is to test whether the presence of CFTR variants affects the conversion of blood cells into stem cells, the first step to create new beta cells. The researchers also need to find out if these new beta cells can produce insulin and function normally in the lab.
Would a beta cell transplant be successful?
The researchers plan to transplant the new beta cells just under the skin of the donor. To do this they need to ensure that the beta cells are in an environment that allows them to carry out their function and survive. They also need to ensure that the transplant doesn’t cause any side effects or harm to the person being treated.
Two specific areas of investigation are:
1) Finding the best material for the cells to grow on.
2) How to ensure that the beta cells are connected to a good blood supply. Insulin is transported in the blood, so a good blood supply is important to allow the insulin produced by the new beta cells to be circulated through the rest of the body.
Who's involved?
Principal investigator: Dr Ildem Akerman, University of Birmingham
Co-investigators:
Professor Victoria Salem, King's College London
Dr Rebecca Stott, Chelsea and Westminster Hospital, London