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The long road of multiple sclerosis research

Monday, September 26, 2016

The Pulse on Global Trials by Matthew Howes

Multiple sclerosis (MS), a chronic, autoimmune, inflammatory neurological disease of the central nervous system, is the largest neuro disease affecting young people. In MS, the immune system attacks the protective sheath, called myelin, that covers nerve fibers. Communication between the brain and the body becomes disrupted and nerves become damaged.

Clinical development in MS has been a big business for drugmakers for decades, with lots of advances in recent years. Biogen Idec brought to market both natalizumab, a monoclonal antibody, and dimethyl fumarate (DMF), an immunomodulator. Novartis’ compound, fingolimod, the first sphingosine 1-phosphate receptor modulator, has become the leading oral therapy for MS in the EU. Many analysts speculate that inflammation and immunology products for a wide array of diseases, including MS, may be the next big thing for drugmakers.


But with all the advances, large gaps in care still remain for MS patients. In 2015, there were 868,240 diagnosed cases of MS in the U.S., Japan and five major EU markets (France, Germany, Italy, Spain and the U.K.). The number of diagnosed prevalent cases is predicted to increase to 887,330 cases by 2035. There is an urgent need to identify biomarkers, delay disability, reduce symptoms and prevent side effects.

MS is a disease characterized by a number of paradoxes. With decades of study and a plethora of treatment options, there are no rigid guidelines for a treatment paradigm, like NCCN in oncology. An autoimmune condition, it is treated by neurologists rather than immunologists. First-line therapy for MS is usually an injection. Oral medicines, often thought of as more effective, are saved for second-line.

MS patients are often referred to as snowflakes. From a distance, they all look the same, but on closer examination, each one is unique. Finding a single therapeutic option to treat such a heterogeneous audience is a real challenge, which may explain why there are so many agents available, yet none that comprehensively satisfy the many unmet needs of MS. The history of MS is a story with characters from around the globe. In 1868, Jean-Martin Charcot, a professor at the University of Paris articulated the first complete description of the disease. MS was later recognized in England by Dr. Walter Moxon in 1873, and in the U.S. by Dr. Edward Seguin in 1878.

In 1906, the Nobel Prize for Medicine was awarded to Spanish physicians Dr. Camillo Golgi and Dr. Santiago Ramón y Cajal, who perfected new chemicals to enhance the visibility of nerve cells under the microscope.

In 1935, Dr. Thomas Rivers at the Rockefeller Institute in New York City demonstrated that immune cells, not viruses, produced the MS-like illness. This would change the trajectory for how the disease was approached through clinical investigation. It wasn’t until the 1990s that viable treatments for controlling symptoms and slowing progression become available.

Fast forward to 2016. The clinical research landscape for MS looks very different and very promising. Current clinical studies can be categorized in three groups: high-efficacy antibodies, S1P modulators and early experimental approaches to repair and regenerate.

Clinical investigation of the next generation of antibodies, led by major players Biogen, AbbVie and Roche, have produced promising new thereapies like daclizumab and ocrelizumab that deliver high-efficacy with a better safety profile. These therapies, delivered through injections or infused under close medical supervision, tend to serve the needs of patients with severe symptoms and disease progression became available.

For patients with more moderate forms of the disease, improvements in oral S1P therapies may be more interesting treatment advancements. Novartis, Actelion and Celgene are all developing next-generation S1P receptor agonists, aiming to improve upon fingolimod’s poor safety profile. 

Current treatments limit the recurrence of relapses but none currently reverse the damage already done. Repairing damage—the Holy Grail of MS research—while likely a long way off, is perhaps some of the most exciting research for investigators.

Researches at Queen’s University Belfast aim to understand how damage done to the myelin can be repaired. Their innovative study looks at the way the immune system and the central nervous system interact.

Five thousand miles away, on the other side of the globe at Stanford University, researchers are hoping to make breakthroughs in bone marrow transplants, which today are associated with many side effects and high death rates. New science could effectively eliminate these complications and usher in a new era in regenerative medicine. Because bone marrow generates the body’s immune cells, diseases like MS, where rogue immune responses wreak havoc, could potentially be cured through transplant. 


Matthew Howes is senior vice president, marketing innovation for PALIO, an inVentiv Health company. A leader in digital strategy, he has provided the fuel for digital businesses visited by more than 100 million people every month. Email matthew.howes@inventivhealth.com.

This article was reprinted from CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »

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