Successful Treatment of Concomitant Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome with Autologous CD19-targeted CAR-T Cells (P4-14.006)

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. Disclosure: The institution of Dr. Motte has received research support from Ruhr-Univerity Bochum. Melissa Sgodzai has nothing to disclose. Dr. Borie has received personal compensation for serving as an employee of Kyverna Therapeutics Inc. Dr. Borie has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Kyverna Therapeutics Inc. Dr. Borie has stock in Kyverna Therapeutics. Dr. Hegelmaier has nothing to disclose. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Biogen. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Sanofi. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Novartis. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Merck Serono. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Celgene. Prof. Mougiakakos has nothing to disclose. Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen . Dr. Gold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genzyme. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer Vital. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai Pharamaceuticals. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for SAGE Publishers. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Novartis. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Biogen. The institution of Dr. Gold has received research support from Novartis. The institution of Dr. Gold has received research support from Biogen.