HIND MEDYOUF (FRANKFURT)
MESENCHYMAL STEM CELLS IN MDS: THE BETTER TARGETS?
Myelodysplastic syndromes (MDS) are a heterogenous group of stem cell driven disorders primarily
affecting the elderly and characterized by inefficient production of mature blood cells and a high risk
(30%) of evolution to secondary acute myeloid leukemia. Despite tremendous progress in the past
decade, treatment options for MDS patients remain limited, and primarily address disease symptoms,
rather than altering disease course. This points to the urgent need to better understand the
pathogenesis of this heterogenous group of syndromes to develop new therapies that address disease
vulnerabilities. However, this effort has been largely hampered by the limited availability of model
systems that allow the exploration of MDS biology in a fully humanized setting. In recent years, studies
from our laboratory and others, have highlighted the crucial role niche cells play in human MDS, hence
reinforcing the notion that MDS is a disease of a tissue rather than hematopoietic cells alone.
Therefore, exploration of MDS biology requires the further development of fully human MDS models
in which both constituents of the disease, namely hematopoietic and niche cells, are present.
The presentation will review the platforms that are currently available to study MDS biology in a
humanized setting as well as share current efforts from our laboratory that contributes towards
developing modular systems that could be exploited for evaluating novel therapeutic strategies in a
personalized setting.