Invention: Treating lupus by targeting T-cells
Patients suffering from the autoimmune disease known as systemic lupus erythematosus (SLE) could soon benefit from much better treatment outcomes. A new medication invented by French immunologist Sylviane Muller and her team at the National Centre for Scientific Research (CNRS) in Paris not only alleviates symptoms, but is the first treatment of its kind to halt the progression of the disease.
Lupus is an incurable disease in which the immune system -
tasked with defending the body from outside threats - turns inwards, attacking
healthy tissues and processes. Until now, treatments have consisted of
steroids, associated with significant side-effects when used over the long term,
and so-called immunosuppressants that suppress the entire immune system and
leave the body vulnerable to infection. Muller's new medication, marketed as Lupuzor and expected to be
launched in 2018, silences only so-called "T-cells" associated with
lupus while leaving healthy defences intact. Muller and her team at CNRS achieved
their breakthrough in the early 2000s as they were researching the effects of
synthetic peptides on immune responses. A peptide (a short chain of amino
acids) known as P140 showed promising characteristics: instead of acting as an
immunosuppressant, it acted as an immunomodulator,
modifying the body's immune response
to stop lupus in its tracks. Research showed that the peptide shut down the
process of cell autophagy (from a Greek word meaning "self-devouring")
that is behind lupus.
Societal benefit
SLE affects 5 million people worldwide, 90% of whom are women. The prevalence of a symptomatic version of the disease ranges between 40 and 70 per 100 000 people around the world. The Latin name lupus means "wolf", as it resembles a wolf bite and reddens the skin of those affected with the disease. Symptoms such as fatigue, joint pain and hair loss prevent many patients from leading functional lives. And while 80-90% of those diagnosed with SLE have the same lifespan as those living without the disease, SLE can overwhelm the body with organ failure or large-scale infections and reduce longevity.
Untreated, SLE can have detrimental effects on muscles and bones, blood (anaemia) and the heart, lungs and kidneys. Yet it is estimated that over 60% of lupus patients do not receive adequate treatment. That's because lupus is extremely difficult to diagnose, since symptoms vary from patient to patient, and has until now been impossible to target with universal treatments. Muller's invention introduces a significant improvement. In the bigger picture, the mechanism behind her invention could be leveraged in drugs against other diseases, both autoimmune and non-autoimmune.
Economic benefit
Muller has co-founded two companies based on her discoveries: Neosystem (now Polypeptide France) in 1986 and ImmuPharma in 2002. The inventor credits a close co-operation with researcher Robert Zimmer, currently President of ImmuPharma, with the successful transition from patented invention to a viable pharmaceutical company. A co-founder of ImmuPharma, Zimmer earned his MD at Strasbourg Medical School and a PhD at the University of Aix-Marseille, and he worked on drug development at companies such as Roche.
Marketed by ImmuPharma, Lupuzor (also known as Rigerimod, IPP-201101 and P140) is slated to launch in the US and five EU countries in 2018. Muller's breakthrough is heralded as a potential blockbuster drug for treating SLE. Based on revenues from current lupus medicines on the market, ImmuPharma conservatively estimates annual sales of Lupuzor could reach over EUR 940 million. The US market, where the drug is currently fast-tracked for FDA approval, is expected to contribute 80% of sales. The latest industry forecasts by GlobalData estimate that revenues from drugs to treat SLE and lupus nephritis (LN) - inflammation of the kidneys caused by SLE - in the world's seven largest pharmaceutical markets will reach EUR 3 billion by 2025.
