Neuromuscular Disease | Harnessing CAR T-Cells for Neuromuscular Disease: Emerging Science and Therapeutic Potential*
Date: October 19, 2026
Time: 3:45 pm to 5:15 pm
Room: Seaglass
Track: Traditional Special Interest Group (SIG)
Session Description
This session will highlight the fundamental principles of CAR-T therapy, differentiate safety and tolerability profile of CAR-T therapies in cancer versus autoimmune disorders, and evaluate the emerging therapeutic applications of CAR-T in neuromuscular disorders, particularly in myasthenia gravis and myositis. Attendees will gain practical clinical insights, understand evolving safety frameworks, and be prepared to assess how these engineered immunotherapies may shape future care strategies for neuromuscular patients in general. The session will focus specifically on emerging data from experimental clinical use as well as recent and ongoing clinical trials in the lead indications of myasthenia gravis and immune mediated myopathies.
Learning Objectives
At the conclusion of this session, attendees will be able to:
- Describe the mechanism of action and therapeutic approach of CAR-T therapy in neuromuscular disease.
- Evaluate safety considerations, monitoring, and logistical challenges associated with CAR-T therapy.
- Review current and emerging treatment approaches for myasthenia gravis and myositis.
Speakers
- (Chair) Jinsy Andrews, MD, MSc, FANA
- (Co-Chair, Speaker) Tahseen Mozaffar, MD, FANA
- (Speaker) Ali A. Habib, MD
- (Speaker) Christina Ulane, MD, PhD
- (Speaker) Charles Carpenter, MS3 (2026 SIG Oral Presenter)
Car-T Therapy in Inflammatory Muscle Disease
Description
Advances in cellular immunotherapy are transforming the treatment of autoimmune diseases, with chimeric antigen receptor T-cell (CAR-T) therapy emerging as a promising approach for patients with refractory autoimmune neuromuscular disorders. As clinical experience expands, understanding the potential role of cell-based therapies is essential for advancing precision medicine in autoimmune neuromuscular disorders.
This presentation will provide an update on the current state of the art in cell therapy for autoimmune neuromuscular disorders, with a focus on the emerging role of CAR-T therapy in autoimmune neuromuscular disorders. Participants will review the scientific rationale for CAR-T–based approaches, examine current clinical and translational research, and discuss the potential benefits, risks, and challenges associated with implementing these novel therapies. Topics will also include future directions in cellular immunotherapy and the implications of these advances for the treatment of patients with refractory autoimmune neuromuscular disorders.
Attendees will leave with a greater understanding of the rapidly evolving landscape of cell therapy for autoimmune myopathies and practical insights into the potential of CAR-T therapy to reshape the management of Autoimmune Neuromuscular Disorders.
Car-T Therapy in Autoimmune Neuromsucular Disorders - Clinical Trial Logistics and Safety
Description
Chimeric antigen receptor T-cell (CAR-T) therapy is emerging as a promising treatment for autoimmune neuromuscular disorders, offering the potential for durable disease control in patients with refractory disease. As these therapies move from oncology into neurology, successful implementation requires careful multidisciplinary coordination, specialized infrastructure, and rigorous safety monitoring.
This presentation will examine the multidisciplinary requirements for the development, administration, and monitoring of CAR-T therapies in autoimmune neuromuscular disorders. Participants will review the clinical trial infrastructure needed to safely deliver these treatments, including patient selection, treatment workflows, adverse event monitoring, and coordinated care among neurology, immunology, hematology, and supportive care teams. Topics will also include current safety data from clinical trials and practical considerations for integrating CAR-T therapy into future clinical practice.
Attendees will leave with a greater understanding of the logistical and safety considerations associated with CAR-T therapy in autoimmune neuromuscular disorders and practical insights into the multidisciplinary collaboration required to successfully implement these innovative treatments.
Car-T Therapy in Myasthenia Gravis
Description
In the past ten years there have been significant advances in the therapeutic landscape for myasthenia gravis. Innovative biologics are advancing immunotherapy options for targeting the underlying mechanisms of autoimmunity in myasthenia gravis. Among these advances, an exciting new technique is chimeric antigen receptor (CAR) T-cell therapy targeting CD19 and BCMA, which transformed the field of hematologic B-cell malignancies and is now expanding to the treatment of autoimmune disease, including myasthenia gravis. Both RNA and DNA platform CAR-T cell therapies targeting B-cells are being investigated in the treatment of myasthenia gravis, and thus far have shown promise as potential disease-modifying treatment capable of inducing durable, medication-free remission in patients with myasthenia gravis.
This presentation will explore the emerging role of B-cell targeted CAR-T cell therapy in the treatment of myasthenia gravis. Participants will review the background and scientific rationale for applying CAR-T cell technology to autoimmune disease, the distinctions between RNA and DNA-based CAR-T cell platforms, examine early clinical evidence supporting its efficacy and safety, and discuss its potential to achieve sustained disease remission without ongoing immunosuppressive therapy in autoimmune disease with a specific focus on myasthenia gravis. This presentation will also review the potential future role for CAR-T cell therapy within the evolving therapeutic landscape for myasthenia gravis.
Attendees will leave with an understanding of the rationale and mechanisms of CAR-T cell therapy for patients with myasthenia, understand the current early clinical data and ongoing studies for CAR-T cell therapy in myasthenia gravis, and gain an appreciation of the potential for CAR-T cell therapy to revolutionize the myasthenia gravis treatment paradigm, as well as the practical opportunities and challenges to integrate CAR-T cell therapy into practice.
Towards a Digital Twin for Myasthenia Gravis
Description
Prediction of clinical outcomes is central to the goal of truly personalized medicine. This presentation will examine a trajectory-based framework for forecasting individual outcomes in myasthenia gravis (MG), integrating longitudinal clinical data from four studies comprising nearly 600 patients. The session will review approaches for harmonizing disparate clinical outcome measures—including the MG-ADL, QMG, and MGC—into continuous patient-level trajectories; describe the development of a stochastic “score portrait” and a baseline “natural” or “standard” trajectory anchored in placebo-arm data; and explore how an “Index of Predictability” can quantify the extent to which early alignment with a given trajectory predicts subsequent treatment response.
The presentation will also address the use of unsupervised clustering to identify clinically and potentially biologically meaningful patient subgroups, as well as associations between trajectory-defined response patterns and covariates such as age and smoking status.
Attendees will gain an understanding of the methodological challenges inherent in predicting outcomes in rare autoimmune diseases and of how trajectory-based approaches may improve clinical-trial efficiency, enable earlier treatment-response assessment, and provide a foundation for digital-twin strategies and precision medicine in myasthenia gravis.
