Lupus anticoagulants

A new study, led by Australian researchers at Monash University, demonstrates a method to reprogram defective cells that could lead to a new cell therapy to provide an effective long-term treatment for lupus. Using human cells from healthy people, the research team developed a treatment that restores the protective aspects of the immune system that prevents autoimmunity.

While the new method, reported today in the journal Nature Communications, was developed specifically to create this protective effect for lupus, the researchers believe it can also be used to create cell therapies for other autoimmune diseases such as diabetes, rheumatoid arthritis, and multiple sclerosis.

As described by Joshua Ooi, PhD, an associate professor at Monash who leads the Regulatory T Cell Therapies Group at Monash Health, the team produced the positive therapeutic effect in vitro and in preclinical models by identifying specific protective molecules in healthy people, the reprogramming the defective regulatory T cells (T-regs) in lupus patients to restore their ability to turn off unwanted immune response.

“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,” Ooi said. “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.”

Using these findings, the Monash researchers are designing clinical trials, set to commence in 2026, to find whether this cell therapy can provide a long-term cure for people with lupus. According to co-senior author of the paper, Eric Morand, PhD, dean of Monash University’s Sub Faculty of Clinical & Molecular Medicine and founder of the Monash Lupus Clinic: “The ability to target, specifically, the disease-causing immune defect, without the need to suppress the entire immune system, is a game-changer. Even if the effects are only medium term, we are confident the treatment can be easily repeated as needed.”

The new discovery was built on previous research by Ooi that showed a lack of specific T-regs to stop the immune system from attacking the human body can lead to the development of an autoimmune disease. The potential new therapy would take blood cells from the lupus patient, modify them in the lab to restore their protective properties, and then reinfuse them to the patient.

Ooi noted that the research was aided by the involvement of patients at Monash allowing the investigators to use human lupus cells throughout the entire study which allowed them to “work as close as possible to the human disease as possible.”

While the researchers believe this approach can be applied to as many as 100 other autoimmune disorders, each treatment will need to be developed individually on a disease-by-disease basis in order to provide the disease-specific effect while not affecting other aspects of the immune system.

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