Which agents are being investigated as possible treatments for myasthenia gravis (MG)?

Updated: Aug 27, 2018
  • Author: Abbas Jowkar, MD; Chief Editor: Nicholas Lorenzo, MD, MHA, CPE  more...
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Answer

Investigational agents

  • Ocrelizumab, a second-generation, humanized monoclonal antibody against B-cell (anti-CD20), which appears to be better tolerated (over first-generation rituximab), is a potential consideration.
  • Abatacept is a cytotoxic T-lymphocyte protein 4 (CTLA-4) monoclonal antibody, which binds to CD80/CD86 and blocks both activating (CD28) and inhibitory signals (CTLA-4).
  • Daclizumab is a monoclonal antibody that binds to CD25 thereby antagonizing the activating effects of IL-2 on T cells.
  • Belimumab binds to soluble B-cell activating factor (BAFF) and reduces B-cell activation and differentiation into antibody-producing plasma cells.
  • Bortezomib is a protease inhibitor that has shown experimental benefit in MG. It reduced anti-AChR antibody titers, inhibited damage to the postsynaptic muscle membrane, and resulted in clinical improvement. [9]
  • Fc receptor modulators (FcRn–IgG) in the form of monoclonal antibody is being studied in passive and active models of rat experimental MG and shown to ameliorate symptoms and lower autantibody levels. [10]
  • Several human monoclonal antibodies directed against IL-17 are in development, including brodalumab (AMG 827), ixekizumab (LY2439821), and secukinumab.
  • Toclizumab, a humanized monoclonal antibody targeting the IL-6 receptor, is being studied as a potential therapy.
  • Autologous stem cell transplantation has been proposed as a therapeutic approach for refractory autoimmune disease and is based on the idea that the transplanted immune cells will be “reset” and free of autoimmune reactivity. [11]
  • 3,4-Diaminopyridine is another symptomatic therapy with potential application in MuSK MG. Exploring use of this drug, which enhances AChR release at the motor nerve terminal, in MuSK MG is supported by preclinical models, experience with congenital MG with MuSK mutations, and case reports. [12, 13]
  • Tirasemtiv is a selective fast skeletal muscle troponin activator. This small molecule binds to skeletal muscle troponin, thereby sensitizing the muscle to calcium and ultimately improving muscle strength under submaximal stimulation. A study of tirasemtiv in experimental MG improved grip strength and muscle force after a single dose. [14]

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