Specialists investigate innovative muscle disease therapy
Specialists from the Medical University of Lublin have started clinical trials of an innovative therapy method for patients with myasthenia gravis, a potentially deadly form of muscle disease.
The disease is an autoimmune neuromuscular disease that disrupts neuromuscular transmission. When the immune system is over-activated, it produces abnormal antibodies, which leads to muscle weakness.
The disease most often begins with paresis of the facial muscles, ptosis, double vision, speaking and swallowing difficulties. Later, the disease may become more severe and other muscle groups can be attacked.
The patient is relatively well in the morning, but during the day, along with daily activities, the muscles start to weaken. The most dangerous thing is when respiratory muscles are attacked. This can lead to a myasthenic crisis, which can be fatal.
Symptomatic treatment is aimed at increasing muscle strength. The second parallel procedure involves weakening the immune system so that it does not attack its own tissues. Among the various therapeutic options, steroids are used first, but in the long run they cause numerous side effects.
Professor Konrad Rejdak, Head of Neurology at the Medical University of Lublin said: “In immune crises, we use the blood purification of antibodies (plasmapheresis), or we administer immunoglobulins. These are immediate rescue methods that can help during a myasthenic crisis, but they are not suitable for long-term, chronic therapy. Therefore, it was necessary to search for new drugs.”
In the project developed at the Medical University of Lublin, cladribine is used as an original therapeutic method in the treatment of patients with myasthenia gravis.
The drug cladribine has been known for about 30 years. It is intended for the treatment of selected forms of leukaemia, as it damages and destroys lymphocytes. Cladribine was also proposed in the treatment of multiple sclerosis, because in this case, too, the immune system is over-activated. Currently, this drug is registered in this indication.
Professor Rejdak said: “The pilot study showed a high effectiveness of the new method. In myasthenia gravis, the drug is given in a short course of therapy, but the clinical effect lasts a long time. We selectively damage abnormal lymphocytes, stop treatment, and allow normal lymphocytes to be restored. As a result, the clinical effect lasts for a long time and the patient does not need to take medications. This is a big advantage of this therapy.”
Over 10,000 people in Poland suffer from myasthenia gravis, and the greatest increase is seen in adults and elderly people with comorbidities. Steroid therapy in an elderly person exacerbates many other diseases, such as diabetes and osteoporosis. There is no such risk in the case of the new drug, and a short therapy has a long-term clinical effect.
The results of the pilot study have been published in the European Journal of Neurology. The prestigious journal Nature commented that the treatment was “a very interesting and promising therapy for patients with myasthenia gravis.” (PAP)
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