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Targeting Secondary Mutations in Juvenile Myelomonocytic Leukemia

University of California San Francisco
Elliot Stieglitz, MD
Grant Type: 
Young Investigator Grants
Year Awarded: 
Type of Childhood Cancer: 
Leukemia, Juvenile Myelomonocytic Leukemia (JMML)
Project Description: 


While most types of childhood leukemia are routinely cured with therapy, juvenile myelomonocytic leukemia (JMML), a type of blood cancer that affects young children remains exceedingly difficult to treat. Even when cured, the infants and toddlers affected by this disorder have difficulty tolerating the required treatment, which can be very intense. Equally frustrating, it is challenging to predict who will respond to the treatments we currently have versus those that will require experimental therapy in order to cure them of their cancer.

Project Goal

Over the past two years, I have looked into the DNA of over 100 children with this disease and found changes, called mutations that likely explain why doctors are unable to rid some children of their disease. By understanding how these mutations in the DNA cause the leukemia to become so aggressive I hope to offer smarter and more easily tolerated medications to treat this disease. It is my goal with this project to make the treatments for JMML easier on children and at the same time improve cure rates for this disease.

Co-funded by: 
Northwestern Mutual Foundation