A pioneering immunotherapy drug aimed at treating Type 1 diabetes has entered its first clinical trial phase, marking a significant milestone in diabetes research. Researchers at the University of Queensland (UQ) have successfully administered the experimental treatment, ASITI-201, to five participants.
Led by Professor Ranjeny Thomas from UQ’s Frazer Institute, this novel drug is designed to modulate the immune system’s response, preventing it from attacking insulin-producing cells in the pancreas. Type 1 diabetes occurs when the body’s immune system mistakenly targets these cells, leaving patients reliant on insulin therapy for survival. Professor Thomas explains that the current standard treatment only addresses the symptoms, rather than the underlying cause of the disease.
ASITI-201 combines a protein derived from the pancreas with vitamin D to help restore immune balance and potentially stop the immune system’s harmful attack on pancreatic cells. “In preclinical trials, particularly in mice, this approach has shown promise in managing the disease,” said Professor Thomas.
Dr. Aakansha Zala, the lead investigator, emphasized that the goal of the trial is to preserve pancreatic function in patients recently diagnosed with Type 1 diabetes, reducing their dependency on insulin. “We are particularly focusing on individuals over the age of 18 who have been diagnosed within the past five years. Our key aim is to observe how the drug influences the immune system’s behavior,” Dr. Zala explained.
Type 1 diabetes affects over 120,000 Australians, primarily in children and young adults, and is a leading cause of lifelong health challenges. The researchers anticipate that larger-scale trials, which could include children, will be essential as the disease often progresses rapidly in younger patients.
Professor Thomas also noted that similar research in the development of treatments for rheumatoid arthritis has informed their work on ASITI-201. “The knowledge we gained from that field has been instrumental in advancing this treatment for Type 1 diabetes,” she said.
The trial is funded by Australia’s Medical Research Future Fund and supported by CUREator, a national biotech incubator. Liperate Therapeutics, a spin-out company formed through UQ’s commercialisation initiative UniQuest, is playing a crucial role in the development of the drug. The project has also received substantial backing from Breakthrough T1D (formerly JDRF), which provided over $2.5 million in grants between 2003 and 2015. Additionally, the Leona M. and Harry B. Helmsley Charitable Trust contributed $5.33 million for the final stages of preclinical development, including safety testing and manufacturing.
Dr. Ben Williams, Program Officer at the Helmsley Charitable Trust, expressed pride in supporting this vital research. “We’re thrilled to be part of this journey, which could significantly improve the lives of those affected by Type 1 diabetes,” he said.
The trial marks an exciting step forward in the quest to transform the treatment of Type 1 diabetes, offering hope for long-term management and, ultimately, a cure.
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