CAAR T-cell therapy offers a more targeted route for neurological autoimmune diseases

by German Center for Neurodegenerative Diseases

edited by Sadie Harley, reviewed by Robert Egan

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Researchers at DZNE and Charité—Universitätsmedizin Berlin are working on a targeted therapy for neurological autoimmune diseases. So far, they have successfully tested their approach in mouse studies involving two such conditions. Their concept is based on deliberately modified immune cells, so-called CAAR T cells, designed to eliminate only the immune cells responsible for causing disease.

The team led by Prof. Harald Prüß aims to translate these findings into clinical trials and, ultimately, therapies for humans. Currently, this development is still at the laboratory stage.

The results on the neuromuscular autoimmune disorder "myasthenia gravis" were published in Nature Communications.

Previously, the researchers had already tested their approach in "anti-NMDA receptor encephalitis."

"Neurological autoimmune diseases" encompass a group of conditions with a wide range of symptoms in which the immune system attacks the body's own nervous system. In these diseases, white blood cells from the B-cell family produce so-called autoantibodies that bind to the body's own cells—specifically, certain proteins on the cell surface.

The consequences vary depending on the disease: In anti-NMDA receptor encephalitis, signal transmission among neurons in the brain is disrupted. In myasthenia gravis, communication from nerves to muscles is impaired. The autoantibodies involved also differ across the two diseases.

Targeted elimination of cells

Conventional treatments aim to remove harmful antibodies from the blood or broadly suppress the immune system. Although this can often relieve symptoms, the effect usually does not last long and may be associated with side effects.

The research group led by Prüß is therefore pursuing a different approach: The harmful B cells are to be selectively eliminated with the help of other immune cells—called "CAAR T cells"—so that no more autoantibodies can be produced.

This involves a complex process in which specialized immune cells are first collected from the blood and then genetically modified so they can specifically recognize and kill the B cells involved in the disease. These CAAR T cells are then administered by infusion.

Two connectors

"B cells can secrete the problematic autoantibodies, but they also carry them on their surface. That's exactly where CAAR T cells can bind, as we equip them with a matching docking mechanism for this purpose," says Dr. Niels von Wardenburg, first author of the current publication.

In myasthenia gravis, however, the population of disease-relevant B cells and autoantibodies is relatively diverse. A single docking mechanism is therefore not sufficient to capture as many disease-causing B cells as possible, the scientist explains.

"The situation is more complex than in NMDA receptor encephalitis, where we successfully demonstrated the feasibility of our approach some time ago. That's why we further developed our procedures and equipped the CAAR T cells with two different couplings for autoantibodies. This allows them to specifically identify and eliminate a broad spectrum of disease-relevant B cells."

Toward clinical trials

Genetically modified immune cells have already been successfully applied to cancer therapy for several years. This approach is now also being tested in clinical trials for neurological autoimmune diseases. Although the method used here, known as CAR T-cell therapy, is similar to the one pursued by the Berlin experts—as reflected in the nearly identical name—there is one key difference.

"Conventional CAR T-cell therapy targets not only disease-causing, so-called autoreactive cells. It also targets cells that serve useful functions. Our approach is much more specific. We selectively act only against disease-causing immune cells. That is why our method is called CAAR-T therapy and, unlike the traditional approach, is spelled with two A's. This name includes the term Chimeric Autoantibody Receptor," explains von Wardenburg.

Currently, the researchers at DZNE and Charité are working to establish the necessary conditions for clinical trials in humans. One key focus is manufacturing CAAR T cells under strictly controlled conditions and according to defined quality standards.

Publication details

Niels von Wardenburg et al, Bispecific chimeric autoantibody receptor T cells eliminate acetylcholine receptor-specific B cells in myasthenia gravis models, Nature Communications (2026). DOI: 10.1038/s41467-026-76750-7

Journal information: Nature Communications

Key medical concepts

Myasthenia GravisAutoantibodies

Clinical categories

NeurologyAllergy and immunology Provided by German Center for Neurodegenerative Diseases Who's behind this story?

Sadie Harley

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Robert Egan

Bachelor's in mathematical biology, Master's in creative writing. Well-traveled with unique perspectives on science and language. Full profile →

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