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Rational Combination Therapy of AML

Institution: 
Memorial Sloan-Kettering Cancer Center
Researcher(s): 
Alex Kentsis, MD, PhD
Grant Type: 
'A' Award Grants
Year Awarded: 
2013
Type of Childhood Cancer: 
Acute Myelogenous Leukemia (AML)
Project Description: 

Background
Acute myeloid leukemia (AML) is a cancer of the blood that affects about 500 children every year in the United States. Current treatment of AML with chemotherapy is toxic, and new therapies are direly needed for children whose disease is resistant to intensive chemotherapy.

Project Goal
Our research into molecular signaling pathways that drive leukemia cell survival has revealed a new set of therapeutic targets. The support of Alex's Lemonade Stand Foundation will enable us to determine the precise molecular mechanisms responsible for signaling that sustains AML cell growth. We will then use this knowledge to rationally devise combination treatments to block AML signaling and effect cure.

2016 Project Update

Acute myeloid leukemia (AML) is a cancer of the blood that affects about 500 children every year in the United States. Current treatment of AML with chemotherapy is toxic, and new therapies are direly needed for children whose disease is resistant to intensive chemotherapy. Our research into molecular signaling pathways that drive leukemia cell survival has revealed a new set of therapeutic targets. In particular, we have discovered an unexpected signaling pathway that controls leukemia cell fate. Blockage of this pathway with new targeted inhibitors leads to leukemia cell death, pointing to a new treatment direction for patients with therapy resistant AML. Ongoing work will enable us to determine the precise molecular mechanisms responsible for signaling that sustains AML cell growth and resistance to therapy. We will then use this knowledge to rationally devise combination treatments to block AML signaling to effect cure. We expect that the successful completion of this project will lead to immediate translation of these approaches into improved treatment of patients with AML, and should accelerate progress towards our long-term goal of rational curative therapy of AML.