CAR T-cell therapy successfully treats rare autoimmune disease of the peripheral nervous system

Physicians from Bochum have used a cancer treatment method to treat severe autoimmune diseases.

A team of physicians from Bochum are the first to successfully use CAR T-cell therapy to treat two patients with a rare autoimmune disease of the peripheral nervous system. This treatment modifies endogenous immune cells such that they can specifically target and eliminate B lymphocytes, which are here the origin of autoimmunity. This is the world's first clinical report in the use of CAR T-cell therapy to treat a serious autoimmune neuropathy. The team at the Department of Neurology, St. Josef-Hospital Bochum, and the Department of Hematology and Oncology at Knappschaftskrankenhaus Bochum, both university hospitals of Ruhr-University Bochum in North-Rhine Westphalia, Germany, describe their results in the journal The Lancet Neurology, published online on June 17, 2025.

For this study, the team treated two patients with chronic inflammatory demyelinating polyneuropathy (CIDP). This rare disease leads to paralysis and loss of sensation, often resulting in severe disability with loss of mobility and in some instances fatal conditions. On average, two to five out of 100,000 people suffer from CIDP on average. Treatment options have been seriously limited up to now, especially against severe and treatment-resistant forms of the disease. CAR T-cell therapy is used in hematology since nearly a decade, but has not been systematically tested for autoimmune diseases of the nervous system.

Individual immune cells genetically altered

CIDP is an autoimmune disease in which the immune system's B-cells attack the peripheral nervous system. In the current study, the CAR CD19 T-cell therapy was used to combat these rogue B-cells. The physicians removed blood from the patients via leukapheresis and isolated millions of T-immune cells. By viral gene manipulation, the American Biotech company Kyverna therapeutics (California, USA) integrated fully humanized chimeric antigen receptors into these immune cells that can identify the pathologically altered B-cells. Each patient was treated with autologous CAR T-cells.

"After reinfusion, we monitored the spread of the CAR T-cells every day at the cellular level, the decrease in the circulating B-cells, and other immune cell parameters to be on the safe side," explains the team. Due to the cell death of mature B-cells and the release of inflammatory cytokines and metabolites, the patients experienced moderate side effects between days 4 and 10 that were immediately resolved by established immune medications. All treatments were carried out in a permanent monitoring setting in an intermediate care unit. "Surprisingly, we saw early signs of improvement in the symptoms caused by the neuritis," the physicians said.

The treatment showed rapid and long-lasting effect. Within a few days, the pathogenic B-cells disappeared from the blood entirely. Functional improvements also occurred: The patients regained the ability to move around safely, some for the first time in years. Objective parameters, including clinical scores and neurophysiological tests, improved by over 200 percent. No further immune therapy was required after the single-time CAR-T treatment in both patients.

CAR T-cell therapy as option for diseases of the peripheral nervous system

Our work shows that the targeted use of CAR T-cells is possible and of significant clinical benefit, even for severe autoimmune diseases of the peripheral nervous system. This breakthrough was only possible because of the close, interdisciplinary teamwork between the clinic and the lab."

 Professor Jeremias Motte, lead author of the study and managing senior physician at the Department of Neurology

The cell therapy itself was guided by Professor Roland Schroers, director of the Department of Hematology and Oncology. "The safe production and use of this innovative therapy for patients with autoimmune diseases is a new area in the field of hematolog. Our work is a good example of translational healthcare in university medicine," says Schroers.

The senior author of the study is Professor Ralf Gold, director of the Department of Neurology. He highlights the significance of the results: "Bochum belongs to the leading neuroimmunological centers in Europe. This publication shows that our years of expertise in fundamental research, diagnostics, and patient care are leading to innovative forms of treatment, to the benefit of our patients with severe illnesses. We especially appreciate the University hospitals in Bochum for supporting us in the individual treatment tests without hesitation."

Eleven patients with various neuroimmunological diseases already treated

Eleven patients with various severe neuroimmunological diseases - including CIDP, myasthenia gravis, and stiff person syndrome - have already been treated at the University Hospital in Bochum in individual treatment attempts and clinical studies with CAR T-cells. This work involved cooperation with partners within and outside of Germany. This success was also made possible by the significant involvement of Dr. Melissa Sgodzai, Dr. Rafael Klimas, and associate professor Dr. Kalliopi Pitarokoili.

"We are starting a new chapter in the treatment of severe autoimmune diseases, emphasizes Jeremias Motte. "Our results show that precision medicine can become reality in neuroimmunology, bringing new hope to patients whom all conventional treatments have failed."