New perspective for the treatment of autoimmune diseases

Enlarge

When two rare autoimmune diseases—immune thrombocytopenia (ITP) and antiphospholipid syndrome (APS)—combine in one patient, this can lead to a life-threatening spiral of severe bleeding and blood clots. An interdisciplinary team at Medical Clinic and Polyclinic III at LMU Hospital in Munich has now demonstrated for the first time that a new form of immunotherapy using bispecific antibodies can specifically target and eliminate the underlying autoimmune reaction. The results of this groundbreaking treatment have now been published in the New England Journal of Medicine.

In immune thrombocytopenia, the immune system attacks the body's own blood platelets, resulting in spontaneous bleeding. Antiphospholipid syndrome, on the other hand, leads to pathological coagulation activation and life-threatening thrombosis. When both diseases occur together, a dangerous contradiction arises: the body is at risk of bleeding to death and forming blood clots at the same time. "Such patients present us with extreme medical challenges," explains Professor Karsten Spiekermann, a hemostaseologist at LMU Hospital.

Novel immunotherapy activates the body's own T cells

In recent years, a special form of immunotherapy—known as CAR-T cell therapy—has already shown impressive success in treating autoimmune diseases. However, this therapy is complex, must be customized for each individual patient, and requires chemotherapy, which can cause infertility and new cancers.

The Munich team led by Professor Marion Subklewe, Dr. Adrian Gottschlich, Professor Karsten Spiekermann, and Professor Michael von Bergwelt used its expertise in T-cell-recruiting immunotherapies to test an alternative, gentler method: the bispecific antibody blinatumomab. "Bispecific antibodies act as a molecular link between T cells and disease-causing B cells," explains Marion Subklewe, head of the Immunotherapy Research Group at LMU Hospital. "This allows us to specifically target and eliminate the cells that produce harmful autoantibodies – without the need for chemotherapy."

Disappearance of autoantibodies – stable blood platelets

As part of a compassionate use program, a young patient with therapy-resistant ITP and APS received two cycles of treatment with blinatumomab. Shortly after the start of therapy, her platelet count rose and the antibodies that had previously caused the disease disappeared completely. “After countless unsuccessful attempts at therapy, we were able to gradually reduce the hematopoietic drugs for the first time – and the platelet count remained stable,” reports Adrian Gottschlich, lead author of the study. “At the same time, the autoantibodies responsible for both the bleeding tendency and the thromboses disappeared.” Accompanying laboratory analyses, carried out in cooperation with PD Dr. Rainer Kaiser (Medical Clinic I), also showed that the formation of procoagulant platelets completely normalized after therapy.

Since treatment, the patient has normal platelet counts and is free of pain, bleeding, and thrombosis—and was able to start oral blood-thinning therapy for the first time in years and dispense with daily subcutaneous injections. This means an enormous gain in quality of life and a return to normal life. “The data show the enormous potential of targeted immunotherapies for autoimmune diseases,” emphasizes Michael von Bergwelt. “Bispecific antibodies in particular open up new, safe treatment options – especially for young patients.”

Adrian Gottschlich et al.: Blinatumomab in Combined Immune Thrombocytopenia and Antiphospholipid Syndrome. The New England Journal of Medicine 2026