Researchers at the University of Queensland have launched the world’s first clinical trial of an innovative immunotherapy designed to treat type 1 diabetes (T1D), a chronic autoimmune condition in which the immune system attacks insulin-producing beta cells in the pancreas. The investigational therapy, known as ASITI-201, aims to retrain the immune system to stop this harmful attack and preserve the body’s remaining ability to produce insulin naturally.
Developed under the leadership of Professor Ranjeny Thomas, ASITI-201 combines a pancreatic protein with a form of vitamin D and is delivered through a targeted nanoparticle system. The approach is designed to selectively suppress the autoimmune response without weakening the overall immune system—a major goal in autoimmune disease treatment. Preclinical studies have shown promising results, paving the way for this first-in-human trial.
The Phase 1 trial will involve five adult participants who were recently diagnosed with type 1 diabetes. The primary goal is to evaluate the safety and tolerability of ASITI-201, as well as its potential to preserve beta cell function. If successful, the therapy could delay or prevent full insulin dependence and offer a new direction in diabetes care.
This research is supported by funding from the Australian Government’s Medical Research Future Fund, Breakthrough T1D (formerly JDRF), and the Helmsley Charitable Trust. The team hopes that positive results will lead to larger clinical trials, including those focused on pediatric patients.
If proven effective, ASITI-201 could represent a paradigm shift in the treatment of T1D—from managing symptoms to addressing the root cause of the disease. This trial marks a major milestone in the search for immune-based therapies that stop or slow the progression of autoimmune conditions while preserving patients’ long-term health and quality of life.
