Chimeric Antigen Receptor (CAR) T Cell Therapy for Neuromuscular Disorders: A Systematic Review

Case reports and case studies increasingly demonstrate that chimeric antigen receptor (CAR) T cell therapy (CTCT) is beneficial not only in hematologic malignancies but also in immunologic diseases, including neuromuscular disorders. The aim of this review is to provide an overview of the current status of CTCT in immune-mediated neuromuscular disorders. This is a systematic review of relevant literature recruited using PubMed, Embase, Scopus, and Google Scholar search terms. Neuromuscular disorders for which CTCT has been used to date include myasthenia gravis (n = 4), Lambert-Eaton syndrome (n = 1), myasthenia/Lambert-Eaton overlap (n = 2), dermatomyositis (n = 2), immune-mediated necrotizing myositis (n = 2), idiopathic inflammatory myopathy (n = 1), anti-synthetase syndrome (n = 4), and chronic inflammatory demyelinating polyneuropathy (n = 2). In most cases, CTCT was directed against CD19-positive lymphocytes and in some cases against B cell maturation antigen. In all reported patients, there was a significant improvement in motor function and quality of life, with some even making a full recovery several months after the application of CTCT. In conclusion, CTCT appears to be a promising therapeutic option for patients with severe immune neuromuscular disorders in whom previous treatment with multiple immunomodulatory therapies has been ineffective. CTCT should be considered in patients with immune neuromyopathy who do not respond to immunomodulatory therapies.