A promising treatment for autoimmune disease lupus: Sylviane Muller named European Inventor Award 2017 finalist
- French immunologist is nominated for EPO prize for developing new targeted medication against the autoimmune disease lupus
- Muller's treatment of systemic lupus erythematosus (SLE) specifically targets "T-cells" associated with disease symptoms
- First medicine to stop the progression of lupus without impeding healthy immune function
- EPO President Battistelli: "Muller's invention is a breakthrough in treating an incurable autoimmune disease that affects quality of life for millions."
Munich, 26 April 2017 - Lupus (Systemic lupus erythematosus or SLE) is a disease in which the immune system of the body mistakenly attacks healthy tissue. It affects over 5 million people worldwide, many of whom live with severe lifestyle restrictions. Until now there was no treatment available to stop the progression of lupus, but that is about to change. Developed by French immunologist Sylviane Muller (64) at the National Centre for Scientific Research (CNRS) in Paris, a new medication is being heralded as the world's first "off switch" for lupus. Slated for a 2018 release, the treatment disrupts the cellular causes of the disease without the negative side effects associated with previous drugs.
For this achievement, Sylviane Muller has been nominated for the European Inventor Award 2017 as one of three finalists in the category "Research". The winners of the 12th edition of the European Patent Office (EPO)'s annual innovation prize will be announced at a ceremony in Venice on 15 June.
"Sylviane Muller's invention is a breakthrough in treating an incurable disease that affects the quality of life of millions of people. They have a hope for a targeted treatment that switches off lupus," said EPO President Benoît Battistelli, announcing the European Inventor Award 2017 finalists. "The successful start-up company she co-founded to market her invention is also a superb illustration of the economic potential of patented inventions."
An unexpected discovery
Muller achieved her breakthrough by analysing the root cellular causes of lupus in her laboratory at the Institute of Molecular and Cellular Biology in Strasbourg in 2001. "At that time, we weren't actually planning to develop a drug; we were only looking for a way to diagnose lupus. But then we discovered that a certain peptide (a short chain of amino acids) slowed down the lupus disease enormously," says Muller. Her research revealed a common pattern. As the main trigger behind all cases of lupus, she identified so-called CD4 T-cells and successfully formulated a medication (based on the molecule they had discovered) to shut them down. "We synthesised these peptides and discovered that they reacted with the immune cells of lupus patients."
Taking aim at an elusive disease
The invention solves a long-standing problem in the treatment of autoimmune diseases. Lupus occurs when the immune system - tasked with defending the body from outside threats - turns inwards, attacking the body's own healthy tissues and processes. Existing medications for lupus suppress the immune system in its entirety, which opens the door to infections. Another major form of treatment, steroids, fails to target lupus specifically and causes significant side effects during long-term use.
The synthetic peptide at the heart of Muller's invention - chemical name: P140 - changes the paradigm. Instead of acting as an immunosuppressant, it acts as an immunomodulator, modifying the body's immune response to stop lupus in its tracks, leaving the rest of the immune system intact.
New hope for patients
The prevalence of SLE worldwide ranges between 40 and 70 people per 100 000, and 90% of those affected are women, especially young women between the ages of 15 and 45. Lupus is not only very difficult to treat, but also extremely hard to diagnose. Symptoms such as joint pain, fatigue, hair loss and rashes can flare up periodically and vary from patient to patient. Experts estimate that over 60% of lupus patients do not receive adequate treatment, which causes further suffering. Untreated SLE can have detrimental effects on heart, bone, blood, lung and kidney functions. While 80-90% of SLE patients enjoy the same life expectancy as those without the disease, lupus can still cause organ failure or large-scale infections and reduce longevity.
Muller's invention promises to make a big impact for lupus victims worldwide. Its action mechanism has been proven to work for broad patient populations. This puts an end to individually tailored drug regimens that increase costs for insurance providers and lupus sufferers.
From laboratory to market
After Muller and her team filed the pivotal patent for the treatment in 2001, she co-founded two companies in order to make the move to clinical practice: Neosystem (now Polypeptide France) in 1986 and ImmuPharma in 2002. Asked about the importance of patenting her inventions, Muller says: "It's very important, because without a patent, neither the pharmaceutical industry nor any large companies will be interested in you, and your work won't be appreciated."
The drug is currently being brought to market under the name Lupuzor by ImmuPharma, a process in which co-founder and president Robert Zimmer has played a major role. Poised for a 2018 market release in the US and five EU countries, Lupuzor is heralded as a potential game-changer in the global market. ImmuPharma estimates that annual sales of Lupuzor could reach EUR 940 million. Analysts estimate that the market for drugs to treat SLE and lupus nephritis (LN) - inflammation of the kidneys caused by SLE - in the world's seven largest pharmaceutical markets could reach EUR 3 billion by 2025. The mechanism behind Muller's invention could also be leveraged to produce highly targeted drugs against other illnesses - including both autoimmune and non-autoimmune diseases.
A champion of multidisciplinary research
Having worked in lupus research for more than three decades, Muller is recognised as an international authority on immune-inflammatory diseases, and she has filed two dozen patents (including 16 at the EPO) and published more than 330 papers and reviews. After obtaining her doctorate in Sciences from the University of Strasbourg, she worked as a postdoctoral researcher at the Max-Planck Institute for Immunobiology in Freiburg, Germany. Today, Muller is a research director at CNRS and supervises over 50 researchers at the CNRS Laboratory of Therapeutic Immunology and Chemistry at the Institute of Molecular and Cellular Biology in Strasbourg, which she has been heading since 2001.
Muller attributes her laboratory's success to bringing together the brightest minds in diverse fields, from medicine and physiology to biochemistry and chemo-informatics. The formula appears to be working. Muller's discoveries have received numerous awards, including the Apollo-B award from Roche (2007), Silver medal of CNRS (2010), the CNRS Innovation Medal (2015) and the French Académie des Sciences Grand Prix Léon Velluz (2016). Asked whether she could see herself leaving the laboratory to play a larger role in her successful company, the inventor says: "I think everybody should stay where he or she is strongest. I decided to stay at the lab doing research."
Additional resources
- Video and photo material
- Read more about the inventor
- View the patent: EP1425295
Europe's universities and public research institutes: inventing a better tomorrow
Sylviane Muller made her breakthroughs at her lab at the University of Strasbourg, which is part of France's National Centre for Scientific Research (CNRS). The CNRS, founded in 1939, is one of Europe's leading public research agencies. Other CNRS scientists to be honoured with a European Inventor Award in the Research category in recent years include Ludwik Leibler (winner, 2015) for creating a new class of plastics known as "vitrimers", and Gilles Gosselin and team (winner, 2012), for developing a revolutionary drug against Hepatitis B. Read more about European research institutes.
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