In a groundbreaking development, a patient with primary immune thrombocytopenia (ITP) has been successfully treated with CAR-T cells, a therapy previously used primarily for cancer treatment. This innovative approach was developed through collaboration between the University Hospital Magdeburg and the University Hospital Carl Gustav Carus Dresden. The results were published in the esteemed medical journal The Lancet on January 4, 2025.
ITP is an autoimmune disorder where the immune system produces misguided antibodies that attack and destroy the body’s platelets, essential for blood clotting. While most patients can manage the condition with medication, a small group, including a 35-year-old man who had been unsuccessfully treated with over ten different therapies for eight years, remain resistant to conventional treatments.
Dr. Karolin Trautmann, a senior physician at the Medical Clinic I for Hematology, Cell Therapy, and Medical Oncology at TU Dresden, and Prof. Dimitrios Mougiakakos, director of the Hematology, Oncology, and Cell Therapy Clinic in Magdeburg, sought new therapeutic options. “We were convinced that CAR-T cell therapy could be an option for patients who do not respond to conventional measures,” Prof. Mougiakakos stated. “In this case, we were backed against the wall, which led us to use the therapy as part of an individual healing attempt,” added Dr. Trautmann.
The CAR-T cells, provided free of charge by the U.S. biotech company Kyverna Therapeutics, are patient-derived immune cells reprogrammed to specifically target and eliminate the disease-causing B-lymphocytes. Following the infusion, the patient’s platelet count rose steadily and has since normalized. Five months post-therapy, the patient remains symptom-free and no longer requires medication. “His quality of life is better than it has been in years,” Dr. Trautmann noted.
After treatment, the previously destroyed B-cells regenerated as “naive” young cells that do not produce harmful antibodies. “We are optimistic that this state will remain stable long-term following the immunological reset,” said Prof. Mougiakakos.
This success establishes a foundation for further scientific and clinical advancements. The University Hospitals of Magdeburg and Dresden plan to conduct a clinical study to systematically investigate CAR-T cell therapy in other patients with severe ITP.
“This case demonstrates the significant potential of CAR-T cell therapy in serious hematological autoimmune diseases. We have successfully applied this in cancer treatment for the benefit of patients. With the Saxon Cell and Gene Therapy Cluster Saxocell, we have ideal conditions to leverage this knowledge and expertise in other indications,” said Prof. Uwe Platzbecker, Medical Director at the University Hospital Dresden.
Prof. Hans-Jochen Heinze, Medical Director of the University Hospital Magdeburg, emphasized, “We are very pleased with the outcome of this individual healing attempt made possible by collaboration. The University Medicine Magdeburg has outstanding expertise in cell therapy and will strengthen this further by expanding a specialized cell therapy center.”