Drug Repurposing in Neonatal Diabetes Study

Investigating the effect of an existing medicine on people with neonatal diabetes



Neonatal diabetes is a rare form of genetic diabetes that occurs in the first six months of a baby’s life. About 20% of babies born with the condition also experience delays in development, muscle weakness, learning difficulties and epilepsy. While current medications help to manage diabetes, they fail to fully address the developmental problems that often persist into adulthood.

But a medicine currently used to treat other conditions affecting the brain offers new hope. Our year long study will investigate whether this drug has any effects on patients’ glucose levels or mental functioning. The results will lay the foundation for a larger clinical trial to find a potentially  life-changing treatment for the developmental problems in patients with neonatal diabetes.

What are we doing?

Neonatal diabetes is a genetic condition caused by a single gene alteration, which means the body struggles to regulate insulin levels in response to changes in blood glucose. Medicines called sulphonylureas help to stabilise patients’ glucose levels by helping patients release their own insulin. However, sulphonylureas fail to treat the developmental problems due to the effects of the gene alteration on the brain. This is because the body moves the medicine out of the brain too quickly, before it can take effect. The team will investigate whether a different drug, already used in other conditions affecting the brain, has the potential to help the developmental problems in neonatal diabetes.

How are we doing it?

Over three months a group of adults with the condition will be given the drug alongside their existing sulphonylurea medication. The team will monitor patients’ sugar levels in the blood and use standardised tests to monitor any changes in brain function. The results from this study will offer crucial insights for a more expansive clinical trial to find a life-changing medicine for individuals affected by neonatal diabetes.

What happens next?

The data collected in this research will guide the design of a larger international trial, assessing the efficacy and safety of the drug in treating neonatal diabetes-related brain and development delays.

BRC Colleagues

Dr Craig Beall

“We know that medicines currently on offer don’t improve the developmental and behavioural problems experienced by patients with neonatal diabetes. We want to change that by finding a medicine that can stay in the brain for longer and overcome the problems caused by the genetic alteration.” 

Dr Pamela Bowman