Media Release: 14 August 2009
Garvan scientists have welcomed recent news that a late-stage clinical trial of the lupus drug belimumab has shown highly promising results. If approved, it will be the first new treatment for lupus, or systemic lupus erythematosus (SLE), in decades.
Belimumab is a monoclonal antibody which targets and inhibits the activity of BLyS (B cell lymphocyte stimulator). BLyS is a naturally occurring protein which is normally involved in the survival of antibody producing B cells. When present in excess it can lead to the development of autoimmune diseases like lupus.
In previous research conducted at the Garvan, Professor Fabienne Mackay* laid the ground work for conducting trials like this by demonstrating the potential of BLyS, known also as BAFF, as a therapeutic target for lupus in mice.
“BLyS is a very important survival factor for immune cells and careful regulation of its production is key to preventing autoimmunity. We found that in a mouse model, over production of this one molecule led to the same kind of tissue damage seen in human lupus and related diseases,” said Prof Mackay.
“We and others have shown there is a correlation between elevated BLyS levels in the patients’ serum and autoimmune disease activity. These observations have led to multiple drug development programs targeting BLyS/BAFF for human autoimmune diseases,” she added.
In the phase III trial, run by GlaxoSmithKline and Human Genome Sciences, belimumab produced statistically significant improvement in patient symptoms compared to placebo and reduced the number of patients reliant on cortisone to control their symptoms.
Lupus is an autoimmune disease in which various tissues in the body become chronically inflamed. A healthy immune system makes proteins called antibodies that protect the body against invading bacteria, viruses and other foreign invaders. Lupus is the result of mistaken identity: the immune system produces antibodies, called auto-antibodies that mistakenly attack the body’s own healthy tissue, in particular the joints, skin, blood, brain and kidneys.
Research into other ways of preventing inflammatory autoimmune diseases continues at the Garvan given that these conditions collectively remain the third commonest cause of morbidity and mortality after heart disease and cancer.
* Professor Fabienne Mackay is now Chair of the Department of Immunology at Monash University, Melbourne.