Forscherdatenbank
Prof. Dr. Uwe Platzbecker
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Fetscherstr. 74
01307 Dresden
Programm
Molecularly Targeted Therapy (MTT)
Übersicht
Translational Clinical Studies in MDS and AML
To date, treatment options for patients with hematological malignancies, especially myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), are still limited. Also the pathomechanisms that drive the disease just get deciphered and are assumed to unveil potential therapeutic interventions. Therefore, novel strategies beyond the few approved drugs and therapies represent an important medical need for these patients.
Within the professional clinical trial networks of the European MDS Study coordination office (EMSCO) and the Study Alliance Leukemia (SAL) numerous multinational investigator initiated trials have been initiated that evaluate cutting-edge treatment concepts and innovative compounds at all stages of MDS and AML. Furthermore, these study concepts are aligned by targeted translational research projects that aim to further dissect the complex pathophysiology and to identify reliable prognostic and predictive biomarkers of response.
In my lab, we characterize and functionally examine the cellular components of the disrupted osteo-hematopoietic stem cell niche in MDS by various preclinical models. Potential changes of this microenvironment are investigated upon specific therapeutic modulation by approved drugs and those that currently undergo clinical testing. Especially the contribution of the stromal compartment to the development of the disease has been thoroughly analyzed in close collaboration with groups at our centre and other DKTK sites.
Moreover, we are establishing a biobank of MDS patient material, i.e. bone marrow and peripheral blood samples, at initial diagnosis and during follow up with and without treatment in order to enable subsequent research projects studying MDS pathology.
DKTK Junior Group Leader for Cancer Systems Biology
Single-cell approaches have not only revealed a wide variety of cell states, characterized by cells exhibiting striking differences in their transcriptional profile, but have also illuminated the mechanisms underlying state transitions in health and disease. Cellular plasticity and adaptive state changes have recently emerged as a basis for therapeutic resistance in cancer, and a better understanding of how cell state transitions are regulated is critical to develop therapeutic approaches that can overcome therapy resistance.
Our research focuses on understanding the mechanisms driving non-genetic cellular heterogeneity and therapy resistance in malignancy. Using novel single-cell sequencing approaches, we seek to develop new experimental and computational strategies to define altered cell states in both, cancer and immune cells. Our aim is to leverage a data driven strategy combined with single cell genomics and systems biology to address the challenges posed by heterogeneity in cancer, and to develop new strategies to overcome it, with the aim of translating laboratory-based findings into the clinic.