Myelodiplastic syndromes and Acute Myeloid Leukemia
Myelodysplastic syndrome (MDS) is a clonal disorder of hematopoietic stem cells (HSCs) characterized by ineffective hematopoiesis with a tendency to progress to Acute myeloid leukemia (AML). While most MDS and AML cases are associated with oncogenic somatic mutations and thought to be random, a few arise from underlying conditions classified as hereditary syndromes predisposing to MDS/AML. For these patients allogeneic stem cell transplantation is the only curative therapy; hence there is a clear unmet medical need.
The identification of targetable molecular pathways will allow a more specific/effective and less toxic molecules to treat MDS/AML patients before proceeding to HSCT allowing better outcomes.
FPS Grant “GATA2-related Myelodysplastic Syndromes and Acute Myeloid Leukemia in Adult patients”
PI: Alessandra Giorgetti
FPS people involved: Alessandra Giorgetti, Chiara Mazzanti and Paolo Aretini
External collaborators: Julio Castaño Cardoso (Centro di Medicina Rigenerativa in Barcelona, CMRB), Anna Bigas (Institut Hospital del Mar d’Investigacions Mediques, IMIM, Barcelona), Grupo Español de Síndromes Mielodisplásicos (GESMD, Spain)