末梢神経疾患の治療の進歩

抄録

Peripheral neuropathies are very common neurological disorders that are caused by various etiologies. This review focuses on four neuropathies which substantial advances have been made recently. In Guillain–Barré syndrome, treatment for severe cases is still a challenge. Clinical trials of several anti–complement drugs are currently underway and results are awaited. In CIDP (Chronic inflammatory demyelinating polyneuropathy), subcutaneous immunoglobulin has been added as a standard maintainace therapy. The efficacy of rituximab or neonatal Fc receptor (FcRn) for CIDP patients has been investigated. It is also interesting from the point of view of elucidating the pathology, and the publication of the results is awaited. Currently, the treatment for the patients with MMN (Multifocal motor neuropathy) is only IVIg and novel treatments for advanced cases are desired. Based on the findings that IgM anti–GM1 antibodies, which are highly positive in MMN patients, bind to motorneurons and activate complement pathway, a trial of AGX722, an antibody against C2, is currently underway. Therapeutic advances in ATTR amyloidosis have been remarkable : in addition to stabilizers of transthyretine, interference therapeutic agents are becoming the main treatment. Phase I trials with gene–editing therapeutics have also been underway and further developments are expected. Compared to other autoimmune diseases such as Myathenia Gravis or Neuromyelitis Optica, autoimmune peripheral neuropathies currently have limited treatment options. However, the development of these novel therapies may lead to new treatment options. While keeping a close eye on the development of new treatments, efforts should be made to accurately diagnose these diseases at an early stage and provide appropriate treatment to patients.