Back to the future: investigating new treatments for type 1 diabetes using old inexpensive drugs

"Great disappointments in medicine frequently give rise to great innovation – so the saying goes – but who expected a 20-year detour?" Denise Faustman and her team were disappointed by their findings from human islet cell transplantation trials and felt compelled to return to the bench for 20 years to understand why the trials had been less successful than had been hoped. They first turned to an animal model of type 1 diabetes, which,
just as in people, features an autoimmune assault on the insulin-producing islet cells in the pancreas. The animal model provided an opportunity to tease apart the immune system that triggers the disease. Over the next 10 years, they turned to studying the blood of large numbers of people with type 1 diabetes, hoping that the promising mouse data could be replicated in people. Those years-long and human and mouse studies suggested a new trigger for diabetes and, thus, a new approach to designing a clinical trial to test a vaccine – a vaccine we all hope will be an advance in treatment for people with type 1 diabetes, and if successful, a remarkably affordable one.