Promising new cell-based therapy for rare autoimmune kidney disease
Dr Peter Eggenhuizen
A new study published in the Kidney International journal from researchers at the School of Clinical Sciences at Monash Health has revealed a promising new T regulatory cell-based therapy for anti-GBM disease, a rare autoimmune kidney disease.
Anti-GBM disease, also known as Goodpasture syndrome, affects approximately 1 in a million people and is considered a rare disease. While therapies have improved over the years such that the 5 year survival rate for these patients is about 80%, their quality of life remains low, with many patients eventually developing kidney failure which requires ongoing dialysis.
There is a serious need for more effective treatments for these patients, and this new study represents an exciting step in the right direction.
Lead author Dr Peter Eggenhuizen, who completed this work under the supervision of A/Professor Joshua Ooi in the Centre for Inflammatory Diseases, said that the new therapy was based on bringing back balance to the immune system, and preventing autoimmune-mediated damage to the kidneys.
This work is based on an earlier study published in Nature by the group that used patient samples to identify that the autoimmune response in Goodpasture syndrome was caused by T cells attacking a specific part of the collagen found in the kidney.
In this most recent study, the team created T regulatory cells that can also target that specific part of the collagen and suppress the surrounding immune response. Dr Eggenhuizen and the team then tested this concept in vitro - in cells in the lab - and showed that their engineered Treg cells were far more effective at suppressing the autoimmune response than non-modified Tregs.
"This potential therapy restores immune balance in anti-GBM disease by introducing engineered T regulatory cells (Tregs) that specifically target the disease-causing autoimmune response,” he explained.
“These precision-engineered Tregs act like specialised peacekeepers, suppressing harmful autoimmune cells while also promoting a more tolerant immune environment through their effects on multiple immune cell types."
A major hurdle in the development of therapies for rare diseases is the struggle to find funding for research. With such a small potential population of people who will use the therapy, it can be difficult to gain financial support from the pharmaceutical industry to develop and translate these therapies.
In addition, since rare diseases affect so few people, researchers need a high percentage of patients from that small community to participate in studies in order to collect enough samples to properly understand the disease.
“We’ve been very lucky here at the School of Clinical Sciences to collaborate with world-leading nephrologists like Professors Stephen Holdsworth and Richard Kitching who have worked over many years to engage with the Goodpasture syndrome community across Australia.”
This innovative Treg cell therapy represents a significant step forward in treating anti-GBM disease, building on decades of dedicated research and invaluable contributions from patients across Australia. While further studies are needed to move this treatment toward clinical use, the findings provide a promising foundation for developing more effective therapies for patients with this challenging autoimmune condition.
About Monash University
Monash University is Australia’s largest university with more than 80,000 students. In the 60 years since its foundation, it has developed a reputation for world-leading high-impact research, quality teaching, and inspiring innovation.
With four campuses in Australia and a presence in Malaysia, China, India, Indonesia and Italy, it is one of the most internationalised Australian universities.
As a leading international medical research university with the largest medical faculty in Australia and integration with leading Australian teaching hospitals, we consistently rank in the top 50 universities worldwide for clinical, pre-clinical and health sciences.
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