IMPATHY-AML: Integrating Molecular and digital PATHologY to track new response biomarkers in high-risk Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a rare and aggressive cancer of the hematopoietic tissue. Genetic features define different risk categories. High-risk AML (HR-AML) is characterized by a high relapse rate and mostly poor clinical outcomes. For this reason, it represents an urgent unmet medical need and a current area of active research.

Beyond genetics, recent studies have highlighted the role of the tumor microenvironment (TME)—and particularly of the immune system—as another key factor influencing disease dynamics and prognosis.

This project aims to study the disease in its various aspects, as well as its microenvironment, taking on the challenge of understanding their complex interactions.

The project involves a prospective, non-interventional clinical study enrolling patients diagnosed with HR-AML, approved by the relevant regulatory bodies. Through a multidisciplinary approach, we aim to:

1. analyze, at the single-cell level, the dynamics of the disease and the immune system in patients undergoing drug treatment and bone marrow transplantation;
2. apply spatial multi-omics and digital pathology techniques to diseased tissue to discover new parameters characterizing these dynamics;
3. understand the mechanisms of disease resistance, identify predictive biomarkers, and validate them in dedicated experimental models.

All this will be made possible thanks to the valuable collaboration of patients who choose to take part in the study, combined with the long-standing clinical and translational research expertise in hematologic malignancies of a network of onco-hematologists, immunologists, and pathologists, and the use of cutting-edge technologies. The results of our project have the potential to innovate diagnostic pathways and therapeutic strategies in AML, improve patient outcomes, and lead to a revision of the current standards for the diagnosis and treatment of high-risk acute myeloid leukemia.