Physicians in Germany and the Netherlands have achieved breakthrough results using CAR-T cell therapy to treat severe autoimmune neurological diseases, marking the first successful clinical applications of this cancer treatment approach for conditions affecting the nervous system.
German Team Achieves First Success in Autoimmune Neuropathy
A team from Ruhr-University Bochum successfully treated two patients with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare autoimmune disease that affects two to five out of 100,000 people and leads to paralysis, loss of sensation, and severe disability. The results, published in The Lancet Neurology on June 17, 2025, represent the world's first clinical report of CAR-T cell therapy for serious autoimmune neuropathy.
"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," said Professor Jeremias Motte, lead author of the study and managing senior physician at the Department of Neurology.
The treatment process involved removing blood from patients via leukapheresis and isolating millions of T-immune cells. American biotech company Kyverna Therapeutics genetically modified these cells using viral gene manipulation, integrating fully humanized chimeric antigen receptors that specifically target pathological B-cells responsible for attacking the peripheral nervous system.
Rapid and Sustained Clinical Improvements
The results exceeded expectations. Within days of treatment, pathogenic B-cells disappeared entirely from patients' blood. Objective parameters, including clinical scores and neurophysiological tests, improved by over 200 percent. Patients regained the ability to move around safely, some for the first time in years, and no further immune therapy was required after the single CAR-T treatment.
"Surprisingly, we saw early signs of improvement in the symptoms caused by the neuritis," the physicians reported. The treatment showed both rapid onset and long-lasting effects, with patients experiencing moderate side effects between days 4 and 10 that were immediately resolved with established immune medications.
Dutch Breakthrough in Neurological Lupus
Meanwhile, researchers at Leiden University Medical Center (LUMC) became the first in the Netherlands to treat an autoimmune disease patient with CAR-T cell therapy. The patient, who received treatment in February for severe neurological lupus, has shown remarkable improvement.
Professor Hans Ulrich Scherer, a professor of rheumatology, explained the treatment rationale: "Current therapies face challenges in penetrating deeper tissues such as muscles, tendons, connective tissue, bone marrow, and organs. As a result, some autoreactive B cells may persist, making the treatment less consistently effective. CAR T-cells offer a promising solution, as they can effectively penetrate tissues and organs."
The Dutch patient has shown no detectable autoantibodies following treatment and was able to discontinue immunosuppressive therapy. Physical improvements included markedly decreased spinal cord swelling and restored function in previously non-functional torso muscles.
Expanding Treatment Applications
The success has encouraged broader applications. 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 through individual treatment attempts and clinical studies.
Professor Hendrik Veelken, head of the hematology department at LUMC, emphasized the institutional preparation required: "Over the years, doctors, nurses, scientists, and analysts have accumulated extensive specialized expertise. Our team knows exactly how to administer this treatment safely and effectively, and they are well-versed in legal and regulatory requirements."
Technical Innovation and Infrastructure
LUMC's success was enabled by substantial investment in a dedicated production facility—the Center for Cell and Gene Therapy—where researchers develop CAR-T cells in-house. This approach significantly reduces the time between collecting a patient's blood and reinfusing the modified cells.
Professor Roland Schroers, director of the Department of Hematology and Oncology at Bochum, noted: "The safe production and use of this innovative therapy for patients with autoimmune diseases is a new area in the field of hematology. Our work is a good example of translational healthcare in university medicine."
Future Clinical Development
Both research teams acknowledge that widespread implementation will require extensive clinical validation. "The close collaboration between hematologists and rheumatologists at the LUMC has been instrumental in achieving this crucial first step in applying CAR T-cell therapy to autoimmune diseases," Scherer said. "However, widespread implementation of this treatment will take a long time."
Professor Ralf Gold, director of the Department of Neurology at Bochum, highlighted the broader significance: "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."
The breakthrough represents a paradigm shift in treating severe autoimmune neurological diseases, offering new hope for patients who have exhausted conventional treatment options and face progressive disability or life-threatening complications.
