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Review
. 2022 Feb;21(2):189-202.
doi: 10.1016/S1474-4422(21)00463-4.

Advances and ongoing research in the treatment of autoimmune neuromuscular junction disorders

Jan Jgm Verschuuren  1 , Jacqueline Palace  2 , Hiroyuki Murai  3 , Martijn R Tannemaat  4 , Henry J Kaminski  5 , Vera Bril  6
Affiliations
Review

Advances and ongoing research in the treatment of autoimmune neuromuscular junction disorders

Jan Jgm Verschuuren et al. Lancet Neurol. 2022 Feb.

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Abstract

Myasthenia gravis and Lambert-Eaton myasthenic syndrome are antibody-mediated autoimmune diseases of the neuromuscular junction that usually present with weakness in ocular muscles and in proximal muscles of the limb and trunk. Prognosis regarding muscle strength, functional abilities, quality of life, and survival is generally good. However, some patients do not respond to treatment. Symptomatic drugs, corticosteroids, and steroid-sparing immunosuppressive drugs remain the cornerstone of treatment. In the past few years, new biological agents against complement, the FcRn receptor, or B-cell antigens have been tested in clinical trials. These new therapies extend the possibilities for targeted immunotherapies and promise exciting new options with a relatively rapid mode of action. Challenges in their use might occur, with barriers due to an increase in cost of care and additional considerations in the choice of drugs, and potential consequences of infection and vaccination due to the COVID-19 pandemic.

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Conflict of interest statement

Declaration of interests JJGMV has been involved in myasthenia gravis research sponsored by the Prinses Beatrix Fonds and Health Holland; reports consultancies for Argenx, Alexion Pharmaceuticals, Ra Pharmaceuticals, and NMD Pharma, with reimbursements received by Leiden University Medical Center; is the coinventor of patent applications for MuSK-related research, with Leiden University Medical Center receiving royalties for MuSK antibody assays; and is a member of the European Reference Network for Rare Neuromuscular Diseases. JP is partly funded by highly specialised services to run a national congenital myasthenia service; has received support for scientific meetings and honorariums for advisory work from Novartis, Chugai Pharmaceutical, Bayer Schering, Alexion Pharmaceuticals, Roche, Genzyme, Abide, Argenx, Union Chimique Belge (UCB), and Viela Bio; reports grants from Merck Serono, Novartis, Abide, MedImmune, Genzyme, Chugai Pharmaceutical, and Alexion Pharmaceuticals; reports research grants from the Multiple Sclerosis Society UK, Guthy Jackson Foundation, National Institute of Health Research, John Fell, Myaware, and Amplo. HM has served as a consultant for Alexion Pharmaceuticals, Argenx, UCB, and Sanofi; has received speaker honoraria from Japan Blood Products Organization and Chugai Pharmaceutical; and reports research support from the Ministry of Health, Labour and Welfare, Japan. MRT has been involved in myasthenia gravis research sponsored by Argenx, Alexion Pharmaceuticals, and NMD Pharma, with all reimbursements received by Leiden University Medical Center. HJK is a consultant for Roche, Cabeletta Bio, Takeda Pharmaceutical, and UCB Pharmaceuticals; is the CEO and CMO of ARC Biotechnology based on US Patent 8 961 98; is the principal investigator of the Rare Disease Network for Myasthenia Gravis National Institute of Neurological Disorders & Stroke (U54 NS115054), and Targeted Therapy for Myasthenia Gravis (R41 NS110331-01) to ARC Biotechnology. VB is a consultant for Grifols, Commonwealth Serum Laboratories (CSL), UCB, Argenx, Takeda Pharmaceutical, Alnylam Pharmaceuticals, Octapharma, Pfizer, Powell Mansfield, Akcea Therapeutics, Ionis Immunovant, Sanofi, Momenta, Roche, Janssen Pharmaceuticals, Alexion, and NovoNordisk; reports research support from Alexion, Grifols, CSL, UCB, Argenx, Takeda Pharmaceutical, Octapharma Plasma, Akcea Therapeutics, Momenta, Immunovant, Ionis Pharmaceuticals.

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