Revolutionary cell therapy: Restarting immune system switches off autoimmune diseases

Sieben der Patientinnen und Patienten mit Autoimmunerkrankungen, die im Rahmen der Studie erfolgreich mit CAR-T-Zellen behandelt wurden. ( Bild: SIMOarts/Simone Kessler) / Seven of the patients with autoimmune diseases who were successfully treated with CAR-T cells as part of the study. (Image: SIMOarts/Simone Kessler)

One single infusion with CAR-T cells replaces immune-suppressive medicines

In March 2021, doctors from FAU treated a young woman with a severe autoimmune disease with chimeric antigen receptor (CAR) T-cells, the first time a treatment of this kind has been attempted anywhere in the world. Today, nearly 3 years later, they were able to publish a pilot study in the New England Journal of Medicine demonstrating that CAR T-cell therapy can make various autoimmune diseases disappear completely for a long period of time, waiving the need for a further drug-based therapy.

“We believe that we have succeeded in finding the reset button that works like with a computer to reboot the system and restart the immune system without errors,” explains Prof. Dr. Georg Schett (director of Department of Medicine 3 – Rheumatology and Immunology) who conducted the study together with Prof. Dr. Andreas Mackensen (director of Department of Medicine 5 – Hematology and Oncology). The team pressed this “reset button” with 15 patients with severe forms of autoimmune diseases, such as “red wolf disease” (systemic lupus erythematosus), systemic sclerosis (also known as scleroderma) and the autoimmune disease triggering inflammation of the muscles known as myositis. One single infusion of cells known as CAR T-cells from the patient’s own body manipulated outside the body led to a resolution of chronic inflammation in organs such as the heart, lungs and kidneys, in the joints and in the skin, allowing patients to stop using immune-suppressive drugs such as cortisone altogether for several years.

Treatment with CAR T-cells was made possible by using a special cleanroom in Department of Medicine 5 at Uniklinikum Erlangen. In this laboratory, the patients’ own immune cells are transformed into therapeutic weapons (CAR T-cells). PD Dr. Michael Aigner and his team from the GMP laboratory monitor the manufacturing process of these living medicines and the quality of the cells before they are returned to the patient. The CAR T-cells very effectively attack pathogenic B-lymphocytes in the bone marrow, in the lymph glands and in all other organs. Completely eliminating B-cells finally leads to the disease being cured, as has now been demonstrated very impressively with the publication in the New England Journal of Medicine.

“It is particularly interesting that the B-cells return at some point after treatment, but the disease does not,” explain Dr. Jule Taubmann and Dr. Fabian Müller, who are part of the interdisciplinary team caring for patients after treatment. The new B-cells in the patients are naive “innocent” cells similar to those in a child, and no longer trigger disease.

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These findings are a breakthrough in immunomedicine. It is already possible for the CAR T-cell therapy to be used on further patients with severe forms of systemic lupus erythematosus, systemic sclerosis and myositis as part of the CASTLE study conducted at Uniklinikum Erlangen. Anyone affected by one of these diseases can write to Car-T-Cell.UKER@uk-erlangen.de.