Successful Treatment of Concomitant Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome with Autologous CD19-targeted CAR-T Cells
Jeremias Motte1, Melissa Sgodzai1, Christiane Schneider-Gold1, Dominic Borie2, Tobias Hegelmaier3, Aiden Haghikia5, Dimitrios Mougiakakos4, Roland Schroers6, Ralf Gold7
1Neurology, UK RUB St. Josef-Hospital, 2Kyverna Therapeutics, 3Neurology, 4Hematology and Oncology, Otto-von-Guericke University, 5Neurologische Klinik der Ruhr-Universitat Boc, 6Hematology and Oncology, UK RUB Knappschaftskrankenhaus, 7Neurologische Universitaetsklinik
Objective:
To describe the first successful treatment of concomitant Myasthenia gravis and Lambert-Eaton myasthenic syndrome with autologous CD19-targeted CAR-T cells.
Background:
Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Despite advancements, some patients remain challenging to treat.
Design/Methods:
Retrospective description of a case with clinical case presentation including video documentation and deep immunophenotyping
Results:
A 33-year-old woman diagnosed with concomitant MG and LEMS in 2019 faced multiple ineffective immunotherapies and a severe course of diseases. Recently, chimeric antigen receptor (CAR)-T lymphocytes targeting CD19 showed promise in addressing autoimmune diseases. This report details the safe and effective use of anti-CD19 CAR-T cells for the treatment of this patient with combined MG and LEMS. After CAR-T cell therapy, she experienced rapid clinical recovery with regain of full mobility. Deep B cell depletion and normalizing AChR and VGCC (N type) autoantibody levels paralleled the major neurological response. Within two months she moved from wheelchair dependence to bicycling and now at four months has started mountain touring.
Conclusions:
In conclusion, this report highlights the potential for an anti-CD19 CAR-T cell approach in achieving autoimmune resetting for the treatment of neuroimmunological diseases.