Project summary
Lipodystrophy causes fat to build up in organs, leading to a rare and hard to treat form of diabetes. Many people with the condition face delayed diagnosis, and current treatments are limited and often ineffective.
Professor Justin Rochford will test whether using existing diabetes treatments alongside newer therapies could lead to better treatment options for people with lipoatrophic diabetes.
Background to research
Lipodystrophy is a rare condition where the body can’t store fat in the usual way. Instead of sitting under the skin, fat builds up in places like the liver and pancreas, where it can cause harm. This makes it much harder for the body to control blood sugar, leading to a type of diabetes called lipoatrophic diabetes. It can look similar to type 2 diabetes, but the usual treatments often don’t work as well for people with this condition.
One treatment that can help is leptin therapy. Leptin is a hormone normally made by body fat and replacing it with daily injections can improve blood sugar and reduce fat in the liver. But not everyone can access it, and daily injections aren’t easy to keep up with.
Another group of medications, called GLP‑1 agonists (GLP‑1s), are commonly used to treat type 2 diabetes. They help the body release insulin when blood sugar is high. In earlier mouse studies that mimicked lipoatrophic diabetes, Professor Justin Rochford found that GLP‑1s quickly improved blood sugar levels. More recent work showed that giving leptin through gene therapy could reduce liver fat and boost insulin levels.
Professor Rochford now wants to find out whether combining these two treatments could offer an even better way to manage this rare form of diabetes.
Research aims
Professor Justin Rochford and the team will study the safety and effectiveness of different treatments using mice that share key features of lipoatrophic diabetes seen in people living with the condition.
The study will test three treatments: a leptin gene therapy approach and two GLP-1s, ‘Semaglutide’ (Ozempic) and ‘Tirzepatide’ (Mounjaro).
First, each treatment will be tested on its own to understand how well it works individually. They’ll then test combinations of the gene therapy with each drug to see whether using treatments together improves outcomes.
Finally, the researchers will examine tissues and organs in detail to understand how these treatments affect the body and how they work to improve lipoatrophic diabetes.
Potential benefit to people with diabetes
People with lipoatrophic diabetes are often very unwell and may go years without the right diagnosis or treatment. This research could pave the way for better, easier access treatments that make daily life more manageable and reduce the emotional toll for people living with this condition.
