Summary

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.