Innovative Use of CAR T-Cell Therapy Restores Movement in Patients with Rare Autoimmune Neuropathy

Researchers at Ruhr University Bochum have achieved a groundbreaking milestone in the treatment of rare autoimmune neurological diseases. In their recent publication in The Lancet Neurology, the team reports the first successful application of CAR T-cell therapy to treat two patients suffering from chronic inflammatory demyelinating polyneuropathy (CIDP), a severe autoimmune disorder that causes paralysis, sensory loss, and significant disability. This pioneering approach modifies the patients' own immune cells to specifically target and eliminate B lymphocytes responsible for attacking the peripheral nervous system.

The process involved harvesting blood through leukapheresis, isolating millions of immune cells, and genetically engineering them with chimeric antigen receptors (CARs) to recognize diseased B-cells. The therapy, provided by the American biotechnology company Kyverna Therapeutics, employed autologous CAR T-cells, meaning they were derived from the patients' own cells to enhance safety and compatibility.

Following treatment, both patients experienced rapid declines in the pathogenic B-cell population. Clinically, they showed remarkable improvements: previous impairments in mobility improved significantly within days, with some patients regaining full walking ability in just a few months. Objective measurements, including neurophysiological tests, demonstrated an increase of over 200% in function, with no need for additional immunotherapy after a single dose.

Side effects, mainly moderate inflammatory reactions caused by B-cell death and cytokine release, were quickly managed with standard immune medications. Monitoring revealed that CAR T-cells dispersed and increased their activity at the cellular level daily, ensuring effective control over the autoimmune process.

This study highlights the potential of CAR T-cell therapy as a safe and effective treatment modality for autoimmune diseases affecting the peripheral nervous system, extending the horizon beyond hematological cancers where the therapy has been used for nearly a decade. The interdisciplinary collaboration between clinical and laboratory teams was crucial to this success, illustrating the benefits of translational medicine.

With eleven patients already treated across various neuroimmunological disorders, the researchers see this as a new chapter in personalized neuroimmunology. The promising results open the door to broader application, offering hope to patients with severe, treatment-resistant autoimmune conditions previously considered incurable.

For more details, see the full study: Jeremias Motte et al, 'CD19-targeted CAR T-cell therapy for treatment-refractory autoimmune neuropathies,' The Lancet Neurology, 2025. Source: https://medicalxpress.com/news/2025-06-mobility-rare-autoimmune-disease-successfully.html