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Dana-Farber Cancer Institute

44 Binney Street
Boston, MA 2115
United States

Background

Acute myeloid leukemia (AML) is one of the deadliest pediatric cancers, responsible for 10% of total cancer mortality in children. AML is caused by mutations in normal bone marrow stem cells that make them grow out of control (i.e. become malignant). There are many AML-causing mutations and they result in many different subtypes of AML with very different responsiveness to treatment and outcomes. We need a better mechanistic understanding of both common and divergent (i.e. subtype-specific) pathways leading to AML.

Background

Diffuse intrinsic pontine gliomas (DIPG) are devastating childhood brain tumors. There are no known curative treatments for DIPG. DIPGs arise in a delicate part of the brain known as the brainstem, rendering them un-resectable. Even biopsy of the tumors has been historically avoided due to the critical function of the brainstem. The lack of biopsy samples has greatly hampered understanding of the biology of the tumors.

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