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Targeting Metabolic Vulnerabilities in ETP-ALL

Columbia University
Adolfo A. Ferrando, MD/PhD
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 

Early T cell precursor leukemia (ETP ALL) is an aggressive hematologic malignancy that requires treatment with intensified chemotherapy. Thus, further advances in the treatment of this disease require the development of effective and highly specific molecularly targeted anti-leukemic drugs. There is growing awareness that targeting the metabolic differences between tumor and normal cells holds promise as a novel anticancer strategy. 

Project Goal
This project seeks to unveil key metabolic factors required for proliferation and survival in ETP ALL. These studies will be instrumental for the development of novel targeted therapies for the treatment of this high-risk leukemia group.

Project Update - June 1, 2020
This project seeks to identify new therapeutic strategies against high-risk leukemias targeting metabolism. Leukemia cells depend on specific nutrients and energy sources for growth and survival and inhibition of these metabolic circuitries may offer a path for the development of new therapies. Our studies have profiled the metabolic pathways active in pediatric leukemias and identified two major groups with different metabolic wiring. Analysis of drugs targeting metabolism confirms that these leukemias show differential drug response and identifies cholesterol metabolism, a readily druggable pathway, as a potential selective vulnerability in high-risk tumors.