2025 American Academy of Neurology Abstract Website

Jeremias Motte¹, Rafael Klimas², Melissa Sgodzai³, Franziska Wunsch⁴, Charmaine Schücke⁴, Min-Suk Yoon⁵, Anna Lena Fisse⁴, Anke Salmen⁶, Dominic Borie⁷, Aiden Haghikia⁸, Kalliopi Pitarokoili⁹, Roland Schroers¹⁰, Ralf Gold¹¹
¹UK RUB- St. Josef-Hospital, ²St. Josef Hospital - Ruhr-University Bochum, ³Neurology, UK RUB - St. Josef- Hospital, ⁴UK RUB - St. Josef- Hospital, ⁵Neurology, Evangelisches Krankenhaus Hattingen, ⁶St. Josef-Hospital, Ruhr -University Bochum, ⁷Kyverna Therapeutics Inc., ⁸Medizinische Hochschule Hannover, ⁹St Josefs Hospital, ¹⁰Department of Hematology and Oncology, Knappschaftskrankenhaus, Ruhr-University Bochum, ¹¹Neurologische Universitaetsklinik

Objective:

To describe the first use of autologous CD19-targeted CAR-T-cells for the treatment of three cases of autoimmune neuropathies.

Background:

Autoimmune neuropathies are heterogeneous disorders of the peripheral nervous system. Antibody-associated neuropathies, such as paranodopathies or antiganglioside-related neuropathies, are particularly challenging to treat. Despite advances in immunotherapies, some patients remain refractory to treatment.

Design/Methods:

Analysis of three Anti-CD19-CAR-T-cell-treated patients, including clinical presentations with video documentation and deep immunophenotyping at various stages post-treatment.

Results:

The first case is a 73-year-old male with GM1-IgM-positive multifocal motor neuropathy (MMN), presenting with tetraparesis and bedridden status. Stabilization was initially achieved with weekly IVIG and plasmapheresis. Following CAR-T-cell therapy, he remains fully ambulatory without further immunotherapy for over 6 months.

The second case is a 54-year-old male with NF155 paranodopathy (antibody titer 1:1000). Despite plasmapheresis and rituximab, he experienced rapid progression to tetraparesis, wheelchair dependency, and severe hand tremor. CAR-T-cell therapy was well-tolerated; by the first follow-up month, neurological status stabilized with improved walking and reduced tremor.

The third case is a 46-year-old female with severe CIDP. Despite rituximab and frequent plasmapheresis, her walking ability deteriorated. Anti-CD19-CAR-T-cell therapy demonstrated a favorable safety profile. Updates on patients status will be presented.

CAR T-cell expansion was sufficient around day 10 post-infusion in all patients, and the therapy was well-tolerated with no significant safety signals observed. Only mild, short-term CRS occurred, and no ICANS was observed.

Conclusions:

Here we report on the therapeutic potential of an anti-CD19 CAR-T cell approach to achieve stabilization of severe autoimmune diseases of the peripheral nervous system. This case series highlights the potential of CD19-targeted CAR-T-cell therapy as a promising approach for rare and severe autoimmune neuropathies. We will present up to 12 months follow up of these 3 patients after this novel treatment strategy.