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Genetically modified adoptive immunotherapy- new therapeutic option to cure relapsed and/or refractory leukemia in children and adults?

Susanne Reuther

Refractory/relapsed leukemias in children, adolescents and adults are generally associated with a poor prognosis and usually require allogeneic stem cell transplantation. Despite the significant developments over the past few decades, a reliable therapy leading to long-term complete remission is still not available. Therefore, the development of adoptive immunotherapies has gained enormous importance. In recent years various leukemia-specific antibodies have been established for treatment of relapsed and/or refractory leukemias. Checkpoint inhibitors have provided significant progress in the therapy of various relapsed/refractory/metastatic tumor entities, but were not successful in highrisk leukemias. A recent milestone in the development of new specific treatment for relapsed/ refractory leukemias are genetically engineered T cells expressing a Chimeric Antigen Receptor that targets specific antigens (CAR-T cells). These CAR-T cells represent a combination of gene and immunotherapy with impressive results in the treatment of pediatric and young adult patients with aggressive acute lymphoblastic leukemia or diffuse large B-cell lymphoma and in the treatment of adults with primary mediastinal B-cell lymphoma. This review summarizes the current possibilities of adoptive immunotherapies, with a focus on leukemias and the situation in Germany.