Myasthenia Gravis Links and News Community Group
This group is for keeping track of news, links, studies, and other information that is helpful for people with Myasthenia, whether you are newly diagnosed or you have had MG for awhile.
This group is for keeping track of news, links, studies, and other information that is helpful for people with Myasthenia, whether you are newly diagnosed or you have had MG for awhile.
"Eculizumab has been shown to produce a clinically meaningful benefit in patients with severe and refractory generalized myasthenia gravis, an ultra-rare neurological disorder caused by uncontrolled complement activation resulting from auto-antibodies that recognize a specific target in the nerve-muscle junction. In a Phase II study involving 14 patients, eculizumab was superior to placebo in improving disease severity scores, and the improvement was achieved more rapidly with eculizumab than with placebo"
http://www.alxn.com/News/article.aspx?relid=605575.
Eculizumab safe and effective in severe MG
Neurologist James F. Howard Jr., from the University of North Carolina at Chapel Hill, presented positive results from a phase 2 pilot trial of eculizumab (Soliris) in people with severe, generalized (affecting muscles throughout the body) MG whose disease could not be adequately treated with at least two standard immunosuppressant medications for more than one year.
Eculizumab is a type of immunosuppressant known as a complement inhibitor. The drug is on the market to treat two conditions unrelated to MG (paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome).
This multicenter, double-blind, placebo-controlled study included 14 people who had moderate to severe muscle weakness despite treatment with immunosuppressants for more than a year.
Among the findings:
Six of the seven people treated with eculizumab for 16 weeks (86 percent) saw a three-point reduction in their quantitative myasthenia gravis (QMG) score. On this assessment, lower scores reflect better muscle strength.
During the first 16 weeks, the average change in QMG score was an improvement of 7.43 points in the eculizumab group and an improvement of 2.71 points in the placebo group.
Four of the seven people treated with eculizumab had an eight-point improvement in the total QMG score compared with one of seven in the placebo group.
When data from all patient visits were assessed, the overall change in average QMG total score was significantly better in the eculizumab group compared to the placebo group. The average improvement in the treated group was 6.43 points; in the placebo group, it was 3.18 points.
Eculizumab was well-tolerated. No one discontinued treatment.
The conclusions:
The positive data from this trial highlight the important role of "uncontrolled complement activation" in severe, generalized MG that is resistant to treatment. The complement system is a group of proteins that enhances an immune response. In MG, there is an undesirable, destructive immune response that attacks the place where nerve and muscle fibers interact.
The data show that eculizumab is apparently safe and beneficial in severe, generalized MG that has not responded to other treatments."