Insulin Gene Therapy

Diabetes is a condition that affects millions of people worldwide. This single condition costs the NHS over £3.5 billion each year and uses up over 10% of all NHS hospital beds, with the vast majority of spending being on type 2 diabetes. Type 1 diabetes occurs due to a lack of β-cells that would normally secrete insulin and type 2 diabetes occurs as a result of ineffective insulin production or insulin resistance, when the body does not respond to the insulin that is being produced. Type 1 diabetes is managed by regular insulin injections, while type 2 diabetes is managed initially with lifestyle modification and typically followed by the addition of metformin or a sulphonylurea and subsequently with newer agents including thiazolidinedione. These treatments work well in the initial stages of the disease but with time it becomes increasingly difficult to maintain adequate blood sugar levels. Despite combination therapy, a significant proportion of type 2 affected individuals will go on to require regular insulin injections and many will develop severe long term complications. We are currently testing whether the insulin gene itself can be the target of a novel class of therapeutic compounds, which can then be further developed into a treatment for both type 1 and type 2 diabetes. Our drug development approach lies in the synthesis of unique small molecules that can specifically regulate the human insulin gene in order to increase rates of insulin expression and/or allow insulin to be expressed in other cell types such as human stem cells.

Project Team: Dr John Barrow, Prof Kevin Docherty, Prof Matteo Zanda

Funding: Roemex, Knowledge Exchange and Transfer Fund