press releases | 24/07/2025

CAR-T cell therapy in neuroimmunology

World's first patient with myasthenia gravis included in phase I study at LMU Hospital
CAR-T cell therapy (chimeric antigen receptor T cells), which has now become firmly established as a promising innovative treatment option for certain forms of blood cancer (leukemias; lymphomas), has also increasingly become the focus of treatment for therapy-refractory autoimmune diseases in recent years. In addition to treatment successes in systemic lupus erythematosus (SLE), promising individual cases of treatment with CD19 CAR T cells in neuroimmunological diseases such as multiple sclerosis and myasthenia gravis have also been reported in the last two years.
From left to right: Dr. Veit Bücklein, Senior Physician in Haematology; PD Dr. Lisa Ann Gerdes, Senior Physician in Neuroimmunology; Prof. Dr. Tania Kümpfel, Head of the Neuroimmunology Outpatient Clinic; Franca Fuchslocher, Study Nurse in Neuroimmunology; Prof. Dr. Marion Subklewe; Senior Physician and Head of the CAR-T Program in Haematology

It is of great importance that the treatment of these patients with CAR-T cells is now being systematically investigated in clinical trials in order to be able to examine the tolerability and efficacy of this new therapeutic option in these diseases in the best possible way. The Institute of Clinical Neuroimmunology (Principal Investigator Prof. Dr. Tania Kümpfel together with PD Dr. Lisa Ann Gerdes and team) is participating together with the Hematology Department (Prof. Dr. Marion Subklewe, Dr. Veit Bücklein and team) at the LMU Klinikum in an international phase 1 trial with CD19-targeted CAR-T cells, which is being conducted worldwide by Bristol Myers Squibb.

Patients with active multiple sclerosis, progressive multiple sclerosis and active, severe myasthenia gravis who have not responded adequately to previous therapies can be included in this trial.


The LMU Klinikum has included the world's first patient with antibody-positive myasthenia gravis in this study

The first patient worldwide with myasthenia gravis has been included in the study at the LMU Clinic. Myasthenia gravis (also known as myasthenia) is a rare autoimmune disease. In those affected, misdirected antibodies disrupt the communication between nerve and muscle. This causes muscle fatigue in various parts of the body, especially under stress and when tired.

The eyes are frequently affected: the eyelids close and those affected often see double vision. If the disease progresses severely, patients suffer from significant muscle weakness in their arms and legs, and vital functions such as speaking, swallowing and, above all, breathing can be severely impaired.

"We have already been able to treat one patient with progressive multiple sclerosis with CAR-T cells as part of the study, and another patient with multiple sclerosis is planned," says Prof. Dr. Tania Kümpfel from the Institute of Clinical Neuroimmunology.

The CAR-T cell infusion has already been carried out without complications on the hematology ward at the hospital. The patients will continue to be closely monitored and cared for by the neuroimmunology department in close cooperation with the hematology department.

The prerequisite for conducting this highly complex phase 1 study is the excellent cooperation in an interdisciplinary team at a university center, above all between neuroimmunology and hematology at the LMU Clinic. Other departments relevant to the study at the LMU Clinic include the Department of Transfusion Medicine, Cell Therapeutics and Hemostaseology (ATMZH) - where the leukapheresis required to produce the CAR-T cells takes place - as well as Neuroradiology, Neurology, Laboratory Medicine and the Max von Pettenkofer Institute.

The Großhadern site is therefore ideal for conducting this study at the LMU Clinic. The Institute of Clinical Neuroimmunology has great expertise in the treatment of neuroimmunological diseases, has been participating in international studies for over 20 years and cares for many patients with neuroimmunological diseases. On the other hand, there is already a great deal of expertise in the use of CAR-T cells and management of this therapy in haematology: over 150 patients have already been treated with CAR-T cells there.

Study:

A Phase 1, Multicenter, Single-arm, Dose-escalation Study of CC-97540 (BMS-986353), CD19-Targeted NEX-T Chimeric Antigen Receptor (CAR) T Cells, Evaluating Safety and Tolerability in Participants with Autoimmune Neurological Diseases: Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS), or Refractory Myasthenia Gravis (MG) (Breakfree-2); Protocol Number CA061-1001

All LMU Hospital Press Releases

contact

Prof. Dr. med. Tania Kümpfel

Institute for Clinical Neuroimmunology, LMU Hospital

Originally translated with DeepL