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Targeted Therapy for Core Binding Factor Leukemia

University of Massachusetts Medical School
John Pulikkan, PhD
Grant Type: 
Young Investigator Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 


Leukemia is one of the most deadly and most common childhood cancers. Current leukemia treatment using non-selective cytotoxic chemotherapy results in a good initial response, but limited long-term survival, likely due to inadequate targeting of deregulated genes.

Project Goal

We recently developed an inhibitor which can selectively target deregulated gene in inv(16), a type of leukemia observed both in children and adults. We show that this inhibitor can kill cancer cells and prolong survival in leukemic mice. Our findings show the usefulness of targeting mutated genes using novel approaches that can help in the design of better therapeutic methods in the treatment of various cancers including leukemia.


“I am thrilled and honored to receive the Young Investigator Grant from Alex’s Lemonade Stand Foundation. Understanding how the molecular changes in hematopoietic progenitor cell direct leukemia transformation has allowed us to develop small molecules that inhibit the function of oncoproteins and eliminate leukemic cells. Funding from the Alex’s Lemonade Stand Foundation will enable me to continue my research in elucidating the mechanism of action of core binding factor oncoproteins, and to test the efficacy of candidate targeted therapies in humanized mouse models. This support will be critical on our efforts in the design of novel targeted therapy strategies that eliminate the leukemic cells with minimal impact on the normal cells of the patient. Finally, the grant is also important because it will strengthen  becoming an independent investigator in cancer research." ~ John Pulikkan, PhD