Pediatric Hematology and Oncology
Childhood cancers remain a significant global health challenge and are among the leading causes of disease-related death in children. Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy, accounting for approximately 25% of all cases. It arises from malignantly transformed lymphoid progenitor cells and is classified into B-cell precursor ALL (B-ALL) and T-cell ALL (T-ALL), which differ in biology, clinical course, and response to therapy.
Survival rates have improved significantly due to risk-adapted combination chemotherapies, resulting in cure rates exceeding 80% in many patient groups. However, treatment is intensive and associated with significant toxicity. High-risk or relapsed patients often require hematopoietic stem cell transplantation, which carries additional risks.
In recent years, immunotherapy has established itself as a promising approach, including antibody-based therapies and CAR-T cell therapies. These strategies target leukemia-specific antigens and activate the immune system, demonstrating high efficacy particularly in relapsed or refractory diseases. Nevertheless, treatment resistance and relapses remain key challenges and underscore the need for further research.
Research Profile Dresden
Translational research in pediatric hematology and oncology is conducted by the “Pediatric Hematology and Experimental Oncology” (PHEO) research group at the University Hospital Carl Gustav Carus Dresden and the NCT/UCC Dresden. The group develops new therapeutic strategies for acute leukemias, with a particular focus on ALL. The research centers on modern immunotherapeutic approaches that specifically harness the immune system to eliminate malignant cells.
A key goal is to optimize these therapies in terms of efficacy and tolerability. We investigate the causes of treatment failure and relapse and develop strategies for the early detection and overcoming of resistance. A particular focus is on combination therapies as well as the identification of new target structures for personalized treatment approaches to improve cure rates and reduce side effects.
The research group is part of the CATCH-ALL research network (KFO5010) and works closely with the AIEOP-BFM ALL study group to efficiently translate scientific findings into clinical practice.
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Combination of therapeutic antibodies
Our work focuses on the development of novel immunotherapeutic approaches and the refinement of existing treatment strategies for acute lymphoblastic leukemia. A central aspect is the investigation of mechanisms underlying therapy resistance and the development of approaches to overcome them, particularly in relapsed or refractory disease. A key finding from our studies is that combining antibodies targeting leukemia-associated antigens (such as CD19 in BCP-ALL or CD38 in T-ALL) with immune checkpoint blockade (CD47 inhibition) is consistently more effective than single-agent therapy. This combinatorial approach significantly enhances macrophage-mediated clearance of leukemic cells and improves treatment efficacy across diverse preclinical models, including patient-derived xenografts and refractory disease settings. In both B- and T-ALL, combination therapy not only reduces leukemia burden more efficiently but also prolongs survival compared with monotherapies, highlighting its potential to overcome resistance mechanisms and antigen escape. These results provide a strong preclinical rationale for developing combinatorial immunotherapies to improve outcomes for patients with high-risk or relapsed ALL.
Lenk et. Blood 2024; Schewe et al. Hemasphere 2024; Müller et al. Blood 2022
Explore new targets for individualized therapies
In addition to immunotherapy, our research explores new targets for individualized therapies in acute leukemia and other cancers. Using advanced functional genomics and multi-omics approaches, we identify genes and pathways that drive therapy resistance or represent vulnerabilities in malignant cells. For example, in acute myeloid leukemia (AML), we uncovered targets that regulate dormant, drug-resistant subpopulations, providing potential avenues to improve responses to standard chemotherapy. More broadly, by integrating large-scale molecular and genomic data from patient-derived models, we can predict cancer-specific vulnerabilities and prioritize novel, druggable targets. These strategies support the development of personalized treatment approaches, tailored to the unique molecular features of each patient’s disease, with the ultimate goal of improving outcomes and reducing therapy-related toxicity.
Rahimian et al. Drug Resist Updat. 2025; Wurm et al. Cell Rep Med. 2023