HCC Member

Yana Pikman, MD

Dana-Farber Cancer Institute

Academic Titles
Assistant Professor, Pediatrics, Harvard Medical School
Research Program Affiliation
Member, Leukemia
Research Abstract

I am a physician scientist, developing and implementing therapies for acute leukemia while taking care of pediatric oncology patients at Boston Children’s Hospital and Dana-Farber Cancer Institute. My translational research program focuses on the: 1) implementation of clinical genomics, 2) characterization and validation of novel genomic findings, 3) development of biomarkers and correlative biology studies for early phase clinical trials, and 4) development of combination drug approaches for pediatric patients with acute leukemia. My collaborative research program bridges the gap between basic and clinical research. I am the overall PI for the 15-institution LEAP Consortium Matched Targeted Therapy clinical trial (NCT02670525), determining the feasibility of identifying actionable alterations with a matched therapy for pediatric patients with relapsed/refractory or high risk leukemias and myelodysplastic syndrome (MDS). The LEAP Consortium trial has revealed novel genomic findings and treatment hypotheses which we are studying in pre-clinical models. Using a genomic approach, I have identified enzymes of the mitochondrial one carbon folate pathway as being differentially suppressed with therapy in acute myeloid leukemia (AML). I am thus studying the role of this pathway in AML and ALL, using both genetic and chemical approaches.