Pediatric Oncology, Hematology and Immunology
We rely on the concept of personalized medicine to improve our understanding of our patients' conditions and develop new treatment strategies. This term "personalized medicine" is based on the fact that the conventionally defined malignant diseases are extremely heterogeneous when it comes to the underlying molecular processes. These differences can have a great deal of influence on the required therapy and the patient's prognosis. We know today that we not only have to cater to our patients as individuals, but that we must also recognize the uniqueness of their conditions and use this information to develop an individualized therapy. To this end we use subtle molecular and immunological analyses that permit us to obtain a unique "fingerprint" of the patient's condition. In our efforts to translate this in-depth diagnosis into new treatment strategies, we have developed pre-clinical models and initiated clinical trials for our patients.
Leukemias are the most common malignant diseases in children and adolescents; consequently, our leukemia patients are an important focus of both our patient care and our research. Our scientific efforts center on one subgroup of leukemias in particular, which is known as T-cell acute lymphoblastic leukemias (T-ALL). In this subgroup, we were able to identify molecular fingerprints that signal a particularly good response or a poor response to treatment. One objective of our current projects is to refine these fingerprints to the point where we can better personalize the treatment of our patients. Another is to understand how a T-ALL relapse differs from the initial occurrence of the disease, that is why some patients develop a resistance to therapy and how we can disrupt these resistance mechanisms in order to finally arrive at a successful treatment.
In spite of numerous advances in the treatment of childhood brain tumors through more intensive chemotherapy and improved radiotherapy protocols, many patients still suffer relapses that are particularly difficult to treat or suffer from the long-term side effects of therapy. The Pediatric Brain Tumors Unit offers top-level care according to the best medical standards for children and adolescents with brain and spinal tumors. Due to our scientific groundwork in this area, our particular concern is to translate the latest findings on the molecular causes of childhood brain tumors, on the risk assessment and treatment prognoses into new, individualized and specific treatment concepts. The unit offers special consultation hours for personalized therapy where brain tumor patients who suffer a relapse after standard therapy or whose condition progresses in spite of therapy are advised by a team of experienced doctors and scientists.
Neuroblastoma is characterized by a large number of very diverse clinical manifestations that range from spontaneous regression without treatment to very aggressive and therapy-resistant cases. Our research focuses on epigenetic therapeutic approaches that aim at reprogramming dysregulated differentiation, survival and self-renewal mechanisms in neuroblastoma cells with the aid of drugs. For this we characterize the function of the family of histone deacetylases and develop new selective inhibitors from the lab to the clinical application stage. Another focus of our work is the investigation of oncolytic parvoviruses for neuroblastoma treatment.
Osteosarcoma, the most common malignant bone tumor in children and adolescents, is characterized by aggressive growth, a high tendency to metastasize and recidivate, and a varied response to conventional therapies. In spite of intensive efforts, researchers around the world have not yet been able to significantly improve treatment results, in contrast to many other malignant childhood diseases. In pre-clinical models and in a clinical trial, we develop new forms treatment based on radiation therapy such as heavy ion or proton radiotherapy and on the combination of radiotherapy with epigenetically effective agents. We hope that this will contribute to new and improved treatment strategies that can help patients who have proven resistant to therapy so far.
Benign blood disorders include both congenital and acquired disorders of the red blood cells (anemias), white blood cells (immunodeficiencies) and platelets (coagulation disorders). In addition to rare diseases in the strict sense, we also count congenital blood conditions like sickle-cell disease or thalassemia among the rare anemias. In terms of the world population, these are actually the most common hereditary diseases, but since they occur almost exclusively among immigrants here in Central Europe, they are regarded as rare anemias.
These disorders may be caused by iron deficiency or by iron overload. In the past few years, scientists have increasingly focused on hereditary disorders of the iron metabolism. At one end of the spectrum, we frequently find a functional iron deficiency, usually in combination with anemia, that reduces the patients' quality of life and impairs their cognitive abilities. The genetic causes of this disease are often difficult to diagnose and treat, due to the lack of diagnostic strategies. At the other end of the spectrum there are hereditary hemochromatoses caused primarily by mutations in genes that are expressed in the liver. Our research focuses on the discovery of new genes with a key role in the iron metabolism, the identification of molecular mechanisms that maintain the iron balance and the development of animal models that will help us understand the pathogenetic processes involved.
Understanding what causes disease on the molecular level is an important step in the development of new treatment concepts. The interdisciplinary character of the research groups leads to a deeper understanding of disease and therapy.