Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR T cells for treating concomitant MG/LEMS. After CAR T cell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage-gated calcium channel N-type autoantibody levels paralleled major neurological responses. Within 2 months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4 months post-CAR T cell infusion, respectively. This report highlights the potential for anti-CD19 CAR T cells to achieve profound clinical effects in the treatment of neuroimmunological diseases.
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http://dx.doi.org/10.1016/j.neuron.2024.04.014 | DOI Listing |
Wiley Interdiscip Rev Nanomed Nanobiotechnol
November 2024
Research Institute for Medicines (iMed.ULisboa), Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal.
Messenger ribonucleic acid (mRNA) therapeutics are attracting attention as promising tools in cancer immunotherapy due to their ability to leverage the in vivo expression of all known protein sequences. Even small amounts of mRNA can have a powerful effect on cancer vaccines by promoting the synthesis of tumor-specific antigens (TSA) or tumor-associated antigens (TAA) by antigen-presenting cells (APC). These antigens are then presented to T cells, eliciting strong antitumor immune stimulation.
View Article and Find Full Text PDFExpert Opin Biol Ther
December 2024
Research Center for Immunodeficiencies, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.
Introduction: Immune checkpoint inhibitors (ICIs) have revolutionized the field of cancer immunotherapy and have enhanced the survival of patients with malignant tumors. However, the overall efficacy of ICIs remains unsatisfactory and is faced with two major concerns of resistance development and occurrence of immune-related adverse events (irAEs). Bispecific antibodies (bsAbs) have emerged as promising strategies with unique mechanisms of action to achieve a better efficacy and safety than monoclonal antibodies (mAbs) or even their combination.
View Article and Find Full Text PDFCommun Biol
October 2024
RocRock Biotechnology Co. Ltd, Suzhou, China.
Mol Ther Oncol
December 2024
Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA.
Mol Ther
November 2024
Cellectis Inc, New York, NY 10016, USA. Electronic address:
Adoptive cell therapy using chimeric antigen receptor (CAR) T cells has proven to be lifesaving for many cancer patients. However, its therapeutic efficacy has been limited in solid tumors. One key factor for this is cancer-associated fibroblasts (CAFs) that modulate the tumor microenvironment (TME) to inhibit T cell infiltration and induce "T cell dysfunction.
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