Titles + affiliations
Researcher, School of Medicine and Surgery
University of Milano-Bicocca
Generating a New Model of Juvenile Myelomonocytic Leukemia to Study its Cellular Origins and to Identify Therapeutic Vulnerabilities
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.
The Leukemia Research Foundation grant has been instrumental to open up a new line of research in my recently established laboratory, which we will continue to pursue in the coming years. Our results in the last year have already provided important information highly relevant for JMML biology, and the data collected over the course of this project will constitute the basis of applications to additional sources of funding. The results obtained in the last year have already set the foundation for future investigations aimed at better characterizing the onset of JMML, and potentially other pediatric hematological malignancies. Our results are ultimately expected to contribute to the identification of therapeutic vulnerabilities in JMML that will eventually lead to the development of targeted therapies, which will benefit patient health. Moreover, our findings might also reveal cellular or molecular mechanisms more generally relevant for other types of hematological disorders. All of this would not have been possible without the generous support of the Leukemia Research Foundation.
Leukemia Research Foundation grant
$100K awarded in 2021
Juvenile myelomonocytic leukemia (JMML)
Cancer cell biology (leukemia stem cells)