MELBOURNE, Australia — Potentially monumental new research details how scientists discovered how to correct a defect that causes the currently incurable autoimmune disease lupus. Researchers from Monash University say their discovery may pave the way for effective long-term lupus treatment.
More specifically, the new report explains how study authors uncovered a way to reprogram defective cells in lupus patients using protective molecules taken from healthy individuals. Thanks to those healthy cells, the new treatment effectively restores the protective side of the immune system responsible for preventing autoimmunity, or the immune system attacking itself. Lupus, meanwhile, is an autoimmune disease that’s hard to diagnose and even tougher to treat. No two cases of lupus are identical, but the disease is identifiable by the facial rash resembling a butterfly that often occurs among patients.
The research team hopes their new method, developed in test tubes and proven in pre-clinical models, can also apply to additional autoimmune diseases like diabetes, rheumatoid arthritis, and multiple sclerosis.
All humans have proteins that the immune system is capable of attacking, but this doesn’t happen in healthy individuals due to special cells called regulatory T cells or T-regs that stop autoimmune disease. People who develop lupus and other autoimmune conditions do not have T-regs.
Co-senior study author Associate Professor Joshua Ooi, who heads Monash University’s Regulatory T Cell Therapies Group based at Monash Health, explains the therapeutic effect of the new method occurs via the identification of specific protective molecules from healthy people and the subsequent reprogramming of ineffective lupus patient T-regs to restore their ability to switch off unhealthy immune responses.
“We showed the effectiveness of this approach using human lupus patient cells, both in the test tube and in an experimental model of lupus kidney inflammation,” Associate Professor Ooi says in a media release.
“We were able to completely arrest the development of lupus kidney disease, without the use of the usual non-specific and harmful immunosuppressant drugs. It’s like a reset of the abnormal immune system back to a healthy state – kind of like a major software upgrade. That it uses the patient’s own cells is a very special part of this.”
Co-senior study author Professor Eric Morand, Dean of Monash University’s Sub Faculty of Clinical & Molecular Medicine and founder of the Monash Lupus Clinic, describes the new treatment’s effectiveness as both “profound” and a “game-changer.”
Researchers assessed study patients at Monash Health, where Prof. Morand also serves as the Director of Rheumatology. He adds the research team is now designing clinical trials, expected to start around 2026, aimed at investigating whether this method may represent a long-term cure for people with lupus.
“The ability to target, specifically, the disease-causing immune defect, without the need to suppress the entire immune system, is a game-changer,” Morand explains. “Even if the effects are only medium term, we are confident the treatment can be easily repeated as needed.”
Associate Professor Ooi previously discovered that a dearth of specific T-regs capable of stopping the immune system from targeting the body can lead to the development of autoimmune disease. This new treatment, however, would involve taking blood cells from lupus patients, modifying them in a lab setting to restore the protective effect, and then giving them back.
“This project relied on the generous involvement of patients, which enabled us to use human lupus cells every step of the way,” Ooi notes. “This allows us to work as close to the human disease as possible in the lab.
“This is a unique characteristic of Monash University — state-of-the-art research labs side-by-side with clinicians and patients, in this case at Monash Health.”
Co-first study authors Peter Eggenhuizen, a PhD candidate and Research Fellow with the Centre for Inflammatory Disease Monash University, and Dr. Rachel Cheong, former PhD candidate at the Centre for Inflammatory Disease Monash University, are both very confident the new method can prove useful for up to 100 additional autoimmune diseases including diabetes, rheumatoid arthritis, multiple sclerosis, Sjögren’s syndrome, scleroderma, and myasthenia gravis.
“This breakthrough offers huge hope not only in lupus but across the spectrum of autoimmune diseases,” Eggenhuizen says. “There is a huge range of autoimmune diseases that could be targeted with this approach.”
“The great thing is that because the treatment is very specific, it doesn’t harm the rest of the immune system. However, this means that the treatment needs to be carefully developed disease-by-disease, as each one is distinct,” Dr. Cheong adds.
New treatment is giving 30-year lupus patient new hope
Vu Nguyen, 39, was originally diagnosed with lupus in 1995 at the age of nine after she developed swelling and pain in her joints. Fast forward almost 30 years, and Vu has dealt with a wide range of symptoms, kidney biopsies, and hospital stays. She had a stroke when she was 22 and has also been diagnosed with epilepsy, which is secondary to lupus.
It took many years to stabilize Vu’s condition, inspiring her to establish Lupus Victoria. A Masters of Marketing graduate, Vu is currently completing psychological science post-graduate qualifications so she can counsel others who have lupus.
“My main symptoms are now bad functioning kidneys and my epilepsy,” Vu explains. “With Lupus, it always changes. I’m currently in remission but I’ve had a lot of ups and downs with this disease. I think the lupus has made my body more prone to having the stroke and subsequent epilepsy. I am good at the moment. It’s just the epilepsy that’s currently attacking my body.”
“This new treatment will really help people living with lupus; if the treatment was around 30 years ago it would have made a real difference for me. It could really cut down the many different types of medicines we take. With this procedure, we could possibly need just one treatment.”
The study is published in the journal Nature Communications.