By Dr. Jo Sahhar

A ground-breaking new treatment strategy which has been developed by researchers at Monash University for the treatment of lupus may offer a new and safe approach for treating patients with Systemic sclerosis or scleroderma.

A study published in Nature Communications by a team led by Assoc Prof Joshua Ooi at Monash University demonstrated that his team were able to reprogram the defective cells of patients with lupus using protective molecules from healthy people in the laboratory.

Lupus and SSc are both multisystem autoimmune diseases, which predominantly affect women, for which there is no cure and limited therapies. In people with autoimmune disease, organ damage occurs when the immune system attacks the host’s own cells. Whilst immunosuppressive therapies can help, they can be associated with harmful side effects such as infections. 

Humans have many proteins in their bodies which can be targeted and attacked by their immune system, but this doesn’t happen in healthy people because of special cells called ‘regulatory T cells’ or ‘T-regs’ that protect the body from autoimmune disease.  Assoc Prof Ooi discovered that people with lupus and other immune conditions have reduced T reg function, making them more vulnerable to autoimmune disease.

Using human cells, this new treatment restores the protective capacity of the immune system by enhancing the body’s own T- reg function. The investigating team have identified specific protective molecules from healthy people and used these to reprogramme ineffective T-reg cells in lupus patients to restore their ability to switch off harmful immune responses. This technique has been developed in the laboratory in test tubes using cells of patients with lupus. It has been proven to be effective in experimental mouse models of lupus kidney disease with trials demonstrating that the development of kidney disease was stopped completely without the need for potentially harmful immunosuppressive drugs. 

Co-senior author Professor Eric Morand, who heads Monash Rheumatology and is Dean of Monash University’s Sub Faculty of Clinical & Molecular Medicine stated “The ability to target, specifically, the disease-causing immune defect, without the need to suppress the entire immune system, is a game-changer.” The research team hopes to start clinical trials of this therapy in patients with lupus 2026 to determine whether this reprogramming of T- reg cells translates into a long-term and safe cure for people with lupus. The treatment would involve taking cells from the lupus patients, modifying them in the laboratory to restore their protective function, then giving them back to the patients to treat their disease. Assoc Professor Ooi describes this new therapeutic strategy as being “like a reset of the abnormal immune system back to a healthy state – kind of like a major software upgrade”.

Professor Morand and Assoc Prof Ooi believe that this novel treatment strategy could also be effective in treating a range of other autoimmune diseases including Scleroderma, Sjogren’s syndrome and diabetes.

They have recruited a team of clinician/researchers and consumer advocates in these disease areas, including Professor Mandana Nikpour, Assoc Professor  Joanne Sahhar from the Australian Scleroderma Interest Group  (ASIG) and Amanda Lawrie-Jones, President of Scleroderma Victoria and Chair of the Board for Scleroderma Australia and applied for a 25 million dollar Medical Research Futures Fund grant to enable development of this this  treatment strategy to target diseases like Scleroderma.

If this funding application is successful, further development and trials of this novel therapeutic strategy may provide a safer, new treatment for patients with Scleroderma.