Finding a new purpose

Posted: 01st May 2019

Developing a new medicine is a fraught process.
On average, bringing a new drug to market costs $2-3 billion and takes more than 13 years of intense study and clinical trials. Many drugs fall at one or other of the hurdles put before them – they have unintended side effects, or are toxic, or patients don’t show the benefits that have been seen in animal models of disease.
Because of the great investment of time, money and resources spent on getting a drug to the clinic, researchers are increasingly combing through the medicine chest to identify approved drugs that might work for a different indication.
This is why, when researchers in SVI’s Immunology & Diabetes Unit learnt that a drug had been approved to treat the autoimmune disease rheumatoid arthritis, they were excited.
Head of the Unit, Professor Helen Thomas, says that they knew that this drug inhibited a family of proteins that her group had been investigating for some years. They had shown that the proteins played a role in type 1 diabetes, in which they amplified the body’s immune attack against insulinproducing cells.
“When we tested this drug in a type of mouse that develops type 1 diabetes, we found that not only did it prevent the disease but, to our delight, it even reversed it in newly diagnosed mice, effectively curing them.”
Helen says that when type 1 diabetes is first diagnosed in both mice and humans, there are still a substantial number of insulin-producing cells left. If protected from attack by the immune system, at least in Helen’s mice, these cells can make sufficient insulin for normal health.
“We are now planning a clinical trial to test this drug in people with newly diagnosed type 1 diabetes, to see if we can reproduce the benefits we saw in the mice.”
Helen adds that if the trial proceeds as planned, it is likely that in the long term the drug will need to be combined with other treatments to stop the immune attack.
Insulin – first used almost 100 years ago – remains the mainstay of treatment for type 1 diabetes.
Helen and her group hope to soon change that.

For more information please see: Human Immunology