From life-threatening bleeding risk to disease control
Joint therapeutic success from Magdeburg and Freiburg: New immunotherapy approach enables patient with rare blood disorder to live without ongoing treatment
A joint team from Magdeburg University Medical Center and Freiburg University Hospital has successfully treated a patient with a particularly severe form of the rare blood disorder immune thrombocytopenia (ITP). A so-called T-cell engager was used – an active ingredient known from modern cancer therapy, which was used here for the first time to specifically intervene in the misdirected immune response in ITP.
All previously used therapies had not shown any lasting effect in the patient. The new therapeutic approach enabled sustained disease control for the first time – without the patient having to take medication on a permanent basis. The team is now reporting on this extraordinary case in the renowned journal The Lancet.
Immune thrombocytopenia is a rare autoimmune disease in which the immune system attacks and destroys the body's own blood platelets. Blood platelets are essential for blood clotting and wound closure. If their number drops significantly, even minor injuries can lead to severe, potentially life-threatening bleeding. While many patients respond to standard therapies, the disease remains uncontrolled in some patients despite intensive treatment – as was the case with the patient described here over several years.
In this situation, the treatment team decided to use a T-cell engager. These active substances belong to a new class of immunotherapeutic drugs. They establish a targeted connection between the body's own defense cells – the T cells – and disease-causing immune cells, especially plasma cells. In this way, T cells can selectively eliminate the degenerated plasma cells. After treatment, the patient achieved stable blood counts and currently no longer requires ongoing therapy.
Cross-location collaboration as the key to success
“Such sustained disease control in a case of ITP that has been treated so intensively is exceptional,” says Prof. Dr. Jesus Duque-Afonso from the University Medical Center Freiburg, who has been treating the patient for years. “The success of the therapy is the result of close and trusting collaboration between the teams at both locations. The combination of clinical experience, immunological expertise, and translational research was crucial,” emphasizes Prof. Dimitrios Mougiakakos, MD, Director of the University Clinic for Hematology, Oncology, and Cell Therapy in Magdeburg. “This case demonstrates the potential of targeted T-cell-based immunotherapies beyond cancer medicine.”
“Our next joint goal is to understand what immunological changes take place after treatment and why the therapy is so successful in the long term,” adds Prof. Robert Zeiser, MD, Deputy Medical Director of the Department of Internal Medicine I at the University Medical Center Freiburg.
oto: Prof. Dimitrios Mougiakakos, MD. Photographer: Jana Dünnhaupt / OVGU Magdeburg
To date, there have been very few reports worldwide on the use of T-cell engagers in autoimmune diseases. Such agents have primarily been used in cancer medicine. However, the researchers emphasize that further large, controlled clinical trials are necessary to systematically investigate the efficacy, safety, and long-term effects of this approach.
Magdeburg University Hospital is one of the few centers internationally with substantial clinical experience in the use of T-cell-based immunotherapies, including for severe autoimmune diseases, and is systematically advancing their translational development. The successful course of therapy also underscores the importance of the newly founded Magdeburg Center for Cell and Immunotherapies (MAZI) as a platform for innovative, interdisciplinary immunotherapy concepts from Saxony-Anhalt.
Original publication: BCMA-CD3 T-cell engager as salvage therapy for multirefractory primary immune thrombocytopenia, in: Lancet, January 23, 2026, DOI: 10.1016/S0140-6736(25)02515-2
Scientific contact
Prof. Dimitrios Mougiakakos, MD, Director of the University Clinic for Hematology, Oncology, and Cell Therapy Magdeburg, dimitrios.mougiakakos@med.ovgu.de, Tel: +49 391 67 13266