Researcher - Dr. Maxim Pimkin 
Facility - Dana-Faber Cancer Institute
Location - Boston, MA
Amount - $50,000.00

Biography: While a medical student in Russia, my research was focused on various aspects of clinical and molecular microbiology. After graduating from medical school, I pursued a Ph.D. in biochemistry and my research under the mentorship of Dr. G. Douglas Markham at the Fox Chase Cancer Center in Philadelphia was focused on the role of the metabolic enzyme IMP dehydrogenase in the regulation of purine and energy metabolism. Subsequently, my interest in pediatric hematology/oncology and blood development prompted me to join the laboratory of Dr. Mitchell Weiss at the Children’s Hospital of Philadelphia, where I studied the global transcriptional networks that govern hematopoietic lineage priming and specification. In 2011 I joined a Pediatric Residency program at the Children’s Hospital of Pittsburgh, which I finished in 2014, followed by fellowship training in Pediatric Hematology Oncology at the Boston Children’s/Dana-Farber Cancer and Blood Disorders Center from 2014-2017. I am looking forward to a productive academic career where I hope to combine clinical work with basic research in hematopoiesis, stem cell biology and cancer, with the ultimate goal of establishing an independent, NIH-funded laboratory.

Lay Description: MLL-rearranged acute myeloid leukemia (AML) is a subtype of pediatric AML with extremely poor prognosis. Our laboratory studies transcription factors - proteins that regulate the work of genes. In our prior study, we identified several transcription factors as strong selective dependencies in MLL-rearranged AML, i.e. depletion of these two proteins is selectively toxic to a specific type of leukemia cells. Importantly, our preliminary data indicates that inhibition of these proteins does not impair normal blood development. This suggests a potential “Achilles heel” for leukemia-specific therapy with little or no toxic effects on normal tissues. We propose to study why MLL-rearranged AML is so "addicted" to these proteins. We will use a combination of genetic, biochemical and systems biology approaches. In addition, we propose to use a cutting-edge engineered chemical degradation system to evaluate the therapeutic potential of inhibiting these transcription factors in a mouse model of human AML. Our study will validate and mechanistically characterize transcriptional addiction as a potential therapeutic strategy in pediatric AML.

Researcher - Dr. Hamza Celik
Facility - Washington University in St. Louis
Location - St. Louis, MO
Amount - $50,000.00

Biography: Dr. Celik completed his BS (Honors) in Molecular Biology and Genetics at the University of Westminster (London, UK) and his MS in Functional Genomics at the University of York (York, UK). He received his PhD from the French Center for Scientific Research (C.N.R.S) prior to joining Dr. Grant Challen’s Lab as a post-doctoral research fellow in December of 2013. His research focus is to understand the fundamental biology behind hematological malignancies and developing therapeutics for treatments. This partnership between basic science and its clinical application is central to his career ambitions. His current research focus includes functional characterization of tumor suppressors (exp. DNMT3A, JARID2) in leukemic transformation of chronic myeloid malignancies using novel mouse models that have many features of human blood diseases.
Lay Description: Myeloproliferative neoplasms (MPNs) are a group of diseases in which the bone marrow generates a detrimental excess of red blood cells, white blood cells, or platelets. Current MPN therapies are ineffective at treating this deadly disease. Moreover, a substantial proportion of MPN patients transform into an aggressive and therapeutically-refractive acute myeloid leukemia (AML). Patients who develop AML have a poor prognosis with an average survival time after transformation of less than five months. Given the poor survival of individuals with MPN or AML, it is important to delineate the genetic mechanisms of these diseases to develop effective therapies. In this study, we developed the first humanized animal model of MPN closely recapitulating the disease characteristics as it is observed in patients. This animal model will act as a reliable platform to study mechanisms of MPN disease biology and for pre-clinical drug screening to accelerate development of more efficacious drugs.