MS is the most common neurological disease in young adults. There are about 2.3 million people worldwide who face a high risk of ever worsening disabilities from this disease. This year, two innovative drugs were approved in the EU, namely, cladribine, an active agent originally used in cancer therapy, and the monoclonal antibody ocrelizumab. This marks the approval of the first drug for combating primary chronic progressive MS, a form of the disease characterized by continuously worsening symptoms from the onset. In parallel to that and at the initiative of EAN and ECTRIMS, neurologists from 13 countries drew up recommendations for MS treatment in Europe. The new treatment guideline supports physicians in weighing the opportunities and risks of the available treatment options when making therapy decisions.
There are about 2.3 million people worldwide who are suffering from multiple sclerosis (MS), the most common neurological disease in young adults. Three to four times as many women as men are affected by this chronic inflammatory disease of the central nervous system and its risk of progressive disability. "Promising new drugs and the treatment guideline published this year by the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the European Academy of Neurology (EAN) will further refine and improve the treatment of MS in substantial ways," EAN President Prof Günther Deuschl (Kiel, Germany) said at the occasion of the 4th Congress of the EAN in Lisbon, where broad-based discussions of the new advances in MS therapy are conducted.
For 85 to 90 percent of the patients, MS begins as a relapsing-remitting disease. But there are also patients for whom the disease takes a primary progressive course. For them, the complaints worsen continuously from the onset. Without competent treatment, a large share of the patients with relapsing-remitting MS would enter a secondary progressive phase where existing symptoms constantly worsen.
Treatment goal: maximize prevention of relapses and disability progression
Until the mid-1990s, drug therapy was only possible for acute MS relapses. With the first disease-modifying therapies such as interferons beta or glatiramer acetate, it became possible to reduce the annual relapse rate by about one third. Since then, many new drugs including monoclonal antibodies have become available which target specific immune mechanisms to be even more effective and convenient. "MS treatment today is focused on avoiding relapses to the greatest possible extent and to halt the progression of disability. However, some of these drugs may also cause dangerous side effects which have to be considered when counseling our patients," says Prof Franz Fazekas, EAN President elect who was also involved in the development of the European MS guidelines. With competent treatment, disease control has been achieved for 80 to 85 percent of the patients by suppressing the chronic inflammatory disease of the central nervous system as effectively as possible.
Cladribine and ocrelizumab: new options expanding MS treatment
Two new drugs and forms of treatment were recently approved in the EU and should help to bring about further optimization. Cladribine was originally used in anti-cancer treatment. Its mode of action on the immune system allows administering it in a pulse like manner with long lasting effects on the lymphocytes and beyond. Patients undergoing this treatment take cladribine tablets for 14 days, followed by a one-year break in treatment. In the second year, a 14-day treatment is prescribed as therapy, finally giving way to a two-year post-observation period. While this is a convenient and exciting concept it is not yet clear in how many patients and for how long such treatment will eventually succeed to silence MS activity and what to exactly do if new relapses occur. Long-term experience will be necessary to fully appreciate the safety profile of this drug.
The second new MS therapy is the monoclonal antibody ocrelizumab, approved since the beginning of the year in the EU for treatment of active relapsing-remitting MS, but also for patients with early primary chronic progressive MS. Prof Fazekas: "There had not been any therapy for these patients before." Ocrelizumab has a targeted effect on the B cells of the immune system, which play a part in MS.
Following an initial application, further treatment with ocrelizumab entails infusions every six months. Long-term safety data are badly awaited also in this case. Prof Fazekas: "The possibility to choose among many drugs allows neurologists to adapt MS treatment to the individual needs of their patients and along the course of their disease. MS diagnosis means that patients have to live with the condition for 60 or 70 years, so we have to help them to control MS as efficiently as possible considering both benefits and risks."
New treatment guideline assists physicians with their choice of therapy
At the initiative of the EAN and ECTRIMS, neurologists from 13 countries drew up 21 recommendations for MS treatment. The new European treatment guideline is based on the analysis of clinical studies and supports physicians in weighing the opportunities and risks of the available treatment options when making therapy decisions. Prof Deuschl: "This new guideline is part of the EAN guideline program under which all important neurological diseases are to be reappraised. About 20 of the guidelines are currently being worked on," Prof Deuschl added. "These efforts underscore the major significance of the EAN, which is working through overarching topics of this kind and thus providing important principles not only to medical practitioners but also for health care policy."