Emerging drugs for the treatment of myasthenia gravis

Deepak Menon1, Alejandra Urra Pincheira1, Vera Bril1

  • 1Department of Medicine, Ellen & Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, Canada.

Introduction: Advances in understanding the immune pathomechanisms in myasthenia gravis (MG) allow for the development of novel targeted immune therapies. By working at specific points in the immunopathogenesis, these agents have the potential to provide rapid and efficacious responses compared to conventional immunosuppressive therapy (IST), addressing unmet needs and consequently are a research priority.Areas covered: This paper reviews the advances in MG treatment modalities with their scientific rationale. A search of clinicaltrials.gov and a literature search of PubMed from January 2015 to the end of June 2021 was done using the search terms: MG, treatment, immune targets to obtain information on recent developments of complement inhibitors, FcRn receptor inhibitors, direct and indirect B cell inhibitors, CAR and CAAR- T cell therapy, and hematopoietic stem cell transplantation. Specific agents in various phases of clinical development, evidence from ongoing trials and potential roadblocks are examined.Expert opinion: Despite several promising novel agents, existing data as to the timing of initiation and duration of treatment, long-term safety profile and utility in certain patient subsets are limited and require further research. Despite these considerations, the future of MG treatment is transitioning from broad-spectrum IST toward precise, target-driven and personalized immunotherapy.

Related Concept Videos

Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

Myasthenia gravis is a neuromuscular transmission disorder characterized by weakness and increased fatigability of skeletal muscles. It is an autoimmune disease affecting approximately one in 2000 people, where antibodies against the α1 subunit of nicotinic acetylcholine receptors are produced.
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which...
2.2K
Myasthenia Gravis: Diagnostic Tests01:15

Myasthenia Gravis: Diagnostic Tests

Myasthenia gravis is an autoimmune condition affecting neuromuscular transmission, causing generalized weakness in skeletal muscles. Initial diagnoses rely on patients' signs, symptoms, and medical history. The challenge lies in distinguishing myasthenia from other muscular dystrophies. An important diagnostic feature is the significant improvement of symptoms after administering anticholinesterase inhibitors.
The edrophonium test is a diagnostic tool for myasthenia gravis. It involves...
1.6K
Classification of Skeletal Muscle Relaxants01:28

Classification of Skeletal Muscle Relaxants

Skeletal muscle relaxants are a group of drugs that can reduce muscle stiffness and induce temporary paralysis to relieve pain. These agents can act centrally to reduce muscle tone or spasms in painful conditions such as multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), or spinal injuries; they are called antispasmodics or spasmolytics.
Peripherally acting skeletal muscle relaxants interfere with the neurotransmission at the neuromuscular end plate to induce paralysis during...
2.8K
Skeletal Muscle Relaxants: Therapeutic Uses01:31

Skeletal Muscle Relaxants: Therapeutic Uses

Skeletal muscle relaxants are used to relax muscle tone and alleviate painful muscle contractions. However, the choice of skeletal muscle relaxants depends on the duration of the surgical procedure in order to minimize potential side effects. Skeletal muscle relaxants like neuromuscular blocking agents [NMBAs] are commonly employed as adjuvants alongside general anesthetics in clinical settings. NMBAs are also used to maintain controlled ventilation during surgery of the larynx or pharynx...
705
Indirect-Acting Cholinergic Agonists: Pharmacological Actions01:30

Indirect-Acting Cholinergic Agonists: Pharmacological Actions

Indirect-acting cholinergic agonists, also known as anticholinesterases, exert their pharmacological effects by enhancing cholinergic transmission in various body parts, including the neuromuscular junction, autonomic cholinergic synapses, and the brain.
At the neuromuscular junction, these agents work by inhibiting the breakdown of acetylcholine, allowing it to remain bound to the receptor and bind to nearby receptors. This process leads to repetitive firing of the endplate, causing muscle...
1.0K
Nondepolarizing (Competitive) Neuromuscular Blockers: Pharmacological Actions01:27

Nondepolarizing (Competitive) Neuromuscular Blockers: Pharmacological Actions

Nondepolarizing neuromuscular blockers prevent the membrane depolarization of muscle cells and inhibit muscle contraction. These are usually administered with anesthetics to achieve complete muscle relaxation. Upon administration, these drugs first block the small, rapidly contracting muscles of the face and hands, followed by the larger muscles of the trunk and the intercostal muscles. The diaphragm is the last muscle to be affected.
Although all competitive neuromuscular blockers are designed...
657