A pilot study published in the New England Journal of Medicine reveals that CAR T-cell therapy can induce long-term remission in severe autoimmune diseases, potentially eliminating the need for continuous drug-based therapy. Researchers at Universitätsklinikum Erlangen (UKER) in Germany conducted the study, marking a significant advancement in the treatment of autoimmune conditions.
The study, led by Prof. Dr. Georg Schett and Prof. Dr. Andreas Mackensen, involved 15 patients with severe forms of autoimmune diseases, including systemic lupus erythematosus, systemic sclerosis, and myositis. These patients received a single infusion of CAR T-cells, which were engineered from their own immune cells to target and eliminate pathogenic B-lymphocytes. The results showed a resolution of chronic inflammation in organs such as the heart, lungs, and kidneys, as well as in the joints and skin.
CAR T-Cell Therapy Details
The CAR T-cell therapy was administered using a specialized cleanroom at UKER. Patient's own immune cells were transformed into therapeutic CAR T-cells. PD Dr. Michael Aigner and his team monitored the manufacturing process and the quality of the cells before they were returned to the patient. These CAR T-cells effectively target and eliminate pathogenic B-lymphocytes in the bone marrow, lymph glands, and other organs. The complete elimination of B-cells leads to disease remission.
Clinical Outcomes and B-Cell Reconstitution
Notably, researchers observed that while B-cells eventually returned post-treatment, the autoimmune diseases did not relapse. According to Dr. Jule Taubmann and Dr. Fabian Müller, the new B-cells were 'naive' and did not trigger the original autoimmune response, suggesting a 'reset' of the immune system. This finding is particularly interesting and warrants further investigation to understand the mechanisms behind this phenomenon.
Implications and Future Directions
"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. The success of this pilot study has paved the way for further clinical trials, such as the CASTLE study, to evaluate the efficacy of CAR T-cell therapy in a larger cohort of patients with severe autoimmune diseases. Individuals affected by systemic lupus erythematosus, systemic sclerosis, and myositis are encouraged to inquire about potential participation in these trials.
The study represents a significant breakthrough in immunomedicine, offering a potential curative approach for patients with severe autoimmune diseases who have not responded to conventional therapies. The ability to induce long-term remission with a single infusion of CAR T-cells could transform the treatment landscape for these debilitating conditions.
