Emanuele Azzoni, PhD

University of Milano-Bicocca

Titles + affiliations

Researcher, School of Medicine and Surgery
University of Milano-Bicocca

Research

Generating a New Model of Juvenile Myelomonocytic Leukemia to Study its Cellular Origins and to Identify Therapeutic Vulnerabilities

Summary

Juvenile myelomonocytic leukemia (JMML) is a serious chronic blood cancer affecting young children of age 4 or less. It occurs in about 1 in one million children, and as such it represents the most common non-acute pediatric blood cancer. Without treatment, this disease is rapidly fatal. Currently, the only cure is stem cell transplantation, also known as bone marrow transplant. However, compatible donors are not always available, and there is still a high risk of relapse (approximately 50%). Research has studied the genetics of this disease, but this has not yet translated in new cures for the patients. Unfortunately, there is still a lot that we don’t know about how this disease arises and progresses. JMML has a pre-natal origin, meaning it can already begin during gestation, and this makes it very difficult to study. This is especially true because blood cells in the foetus are fundamentally different from those in the adult. For these reasons, it is particularly important to create disease models that can allow researchers to study how leukemic cells arise, and learn how to kill them. In this project, we will generate a new model of JMML which will enable us to identify its cellular origins; in other words, we will identify the cells that will eventually become leukemic. We will search for the factors that can permit this malignant transformation, and we will use this information to search for the “Achilles heel” that could make JMML cells more vulnerable to therapies.

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Leukemia Research Foundation grant
$100K awarded in 2021

Disease focus
Juvenile myelomonocytic leukemia (JMML)

Research focus
Cancer cell biology (leukemia stem cells)