A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease – a serious and common complication of the condition, has shown promising results in a University of Bristol study.
One in three people with type one diabetes will develop kidney damage during their lifetime, which can develop silently over many years, often going undetected until it becomes severe.
Current treatments can slow kidney damage but there are none that act on the root cause: a tiny filter called the glomerulus. A new study published in Molecular Therapy, demonstrated a 64% reduction in a damage indicator for kidney disease, paving the way for a potential new treatment.
The study, driven by first author, Dr Aldara Martin Alonso and led by Dr Rebecca Foster, Associate Professor of Microvascular Medicine at Bristol Medical School: Translational Health Sciences, explored the potential of delivering a protein called VEGF-C directly into kidney cells.
Previous studies have shown VEGFC could protect against kidney disease as it helps keeps blood vessels in the kidney filter healthy, repairing early signs of diabetes-related kidney damage.
To test whether this new approach could be used to treat or slow down kidney disease, the team used a harmless virus to deliver VEGF-C directly into the kidney cells of diabetic mice.
Their results showed that this approach not only helped the kidneys work better, but also protected a key part of the kidney filter that normally helps prevent damage. It led to a 64% reduction in albuminuria. Importantly, this reduction is more than twice the reduction recommended by the American Diabetes Association to slow the progression of chronic kidney disease.
Dr Foster, the study’s senior author, explained: “Currently, there are no drugs specifically available to protect people with type 1 diabetes from kidney disease, despite their higher risk of developing kidney disease. This gap in treatment highlights the urgent need for new therapeutic approaches. Our goal was to investigate whether gene therapy could offer a viable solution by delivering VEGFC in a more targeted way.”
Dr Foster added: “This gene therapy approach has not been explored before in pre-clinical models and offers a long-term solution for these patients who are at risk of developing kidney disease.”
Source: University of Bristol
