Who We Fund

Perelman School of Medicine at the University of Pennsylvania

Dr. Carroll is a physician scientist who has been studying leukemia biology for 3 decades.  Until recently Dr. Carroll saw patients with blood cancers at the Philadelphia Veterans Administration Hospital but is now focused solely on research to improve therapy for AML. He has performed that research at the Univeristy of Pennsylvania since 1998.  Dr. Carroll has had a long term commitment to building tools to enhance the understanding of human AML.  These tools have included development of a large tissue bank of patient samples. He has also lead the field in development and application of the study of AML in immunocompromised mice.  His work in xenograft AML models lead to the characterization of chemotherapy resistance as not always being dependent on leukemic stem cells but on metabolic adaptations of the cells to chemotherapy.  Dr. Carroll has been involved in development of previous novel therapies for AML and continues to focus on developing safer and more effective treatments.

Program Name(s)

Specialized Center of Research Program

Project Title

Precision Targeting of Metabolism and Mitochondria for Chemotherapy Resistance in Acute Myeloid Leukemia

Stanford University

Dr. Kathleen Sakamoto is Professor of Pediatrics at Stanford University School of Medicine. She has been studying the causes of AML and developing new therapies for the past 30 years. Her research funded by Blood Cancer United currently focuses on repurposing a drug used to treat tapeworms, niclosamide, for children with relapsed/refractory AML. Niclosamide is an FDA approved drug and is well tolerated in children. Dr. Sakamoto’s research has resulted in a Phase I clinical trial that will study toxicity, response in AML cells, and drug levels. She is also studying mechanisms of resistance of AML cells to niclosamide to look for drugs that will act synergistically for future clinical trials. Her goal is to take discoveries in the laboratory and translate them to the clinics to improve the overall survival and quality of life in children with AML.

Program Name(s)

Translational Research Program

Project Title

Niclosamide for the Treatment of Relapsed/Refractory Pediatric Acute Myeloid Leukemia

Memorial Sloan Kettering Cancer Center

I am an MD/PhD physician scientist in the oncology fellowship at MSKCC where I treat leukemia and perform research to understand molecular underpinnings of the disease. My prior work at Brandeis University, Cold Spring Harbor Lab, Harvard Medical School, and Albert Einstein College of Medicine was focused on basic mechanisms of gene expression regulation and I am now studying RNA dysregulation in leukemia. During my PhD, I developed novel methods to study RNA binding proteins in collaboration with Nobel Laureate Dr. Michael Rosbash and Rosenstiel Prize winner Dr. Robert H Singer (my PhD mentor). This integrative approach to studying RNA is ideal for understanding understudied proteins that regulate splicing factor mutations in the Abdel-Wahab lab. For my career, I aim to use novel approaches to understand how the most prevalent splicing factor mutations in blood cancers drive progression and transformation.

Program Name(s)

Career Development Program

Project Title

Targeting SF3B1 splicing factor mutant myeloid malignancies through dependency on GPATCH8

Princess Margaret Cancer Centre, University Health Network

Dr. Courtney Jones received her PhD from New York University in 2014 and trained as a postdoctoral fellow at the University of Colorado. Throughout her training, Courtney’s research focused on understanding and targeting acute leukemia cells. She is currently a Scientist at the Princess Margaret Cancer Centre and an Assistant Professor at the University of Toronto. The goal of Dr. Jones’s research is to discovery new ways to target leukemia cells with the overall goal of improving outcomes for patients with leukemia while minimizing side effects. To date, her largest contribution to blood cancer research was the discovery of metabolic vulnerabilities of leukemia cells that have been translated into clinical trials in collaboration with her clinical colleagues. The objective of her current research studies is to discover and develop a new strategy to eradicate leukemia cells in patients for whom standard chemotherapy has failed.

Program Name(s)

Discovery

Project Title

Interrogation of glutathione biology in relapsed acute myeloid leukemia stem cells

University of California, San Francisco

Dr. Arun Wiita is a physician-scientist and Associate Professor in the Dept. of Laboratory Medicine and Dept. of Bioengineering & Therapeutic Sciences at the University of California, San Francisco. Dr. Wiita’s research focuses on mass spectrometry-based proteomics, target discovery, protein engineering, and cellular engineering toward the development of novel therapies for blood cancers. Dr. Wiita also directs the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative (MMTI) Laboratory. Dr. Wiita is a prior recipient of the NIH Director’s New Innovator Award, a Clinical Scientist Development Award from the Doris Duke Charitable Foundation, and a Chan Zuckerberg Biohub Investigator Award, among others, and is an elected member of the American Society for Clinical Investigation. He completed his residency in Clinical Pathology at UCSF, his MD and PhD at Columbia, with graduate training in single molecule biophysics, and undergraduate studies in Chemistry at Princeton.

Program Name(s)

Translational Research Program

Project Title

Optimized, computationally engineered CD70-targeting CAR-T cells for high-risk multiple myeloma

University of Virginia

I am a trained clinical hematopathologist and physician-scientist who is well versed in both basic and translational studies in hematologic tumors, with a special interest and emphasis in B-cell malignancies: the genetic and epigenetic mechanisms of tumor microenvironment (TME)-induced survival and drug resistance. My long-term goal is to characterize the pathobiology of B-cell lymphomas, especially aggressive B-cell malignancies, and the evolution of resistance to drugs and, more recently, immunotherapy.

Over the last decade, I have developed an active and unique research program for drug screening, chemical proteomics profiling, bulk and scRNAseq, ChIP-seq, ATAC, scATAC, functional pharmacogenomic computational biology, and multiplex immune profiling, and applied it to cell line and primary lymphoma samples to determine the major intrinsic and TME extrinsic molecular determinants governing lymphoma cell response and resistance. By capitalizing a “” opportunity and approach, my long-term goal is to provide major advances in our understanding of the lymphoma biology, develop innovative therapies and exert an immediate favorable impact on treatment for lymphoma patients.

My extensive background in cancer biology and clinical hematology/oncology, with my expertise in novel lymphoma therapies and therapy resistance, make me well-suited to serve as a Principal Investigator on many projects.

Program Name(s)

Mantle Cell Lymphoma Research Initiative

Project Title

Understanding Resistance Mechanism to Enhance CAR-T Immunotherapy for MCL

University of California San Francisco

Dr. Smith is a physician-scientist whose laboratory focuses on therapeutic resistance mechanisms and novel treatment strategies for acute myeloid leukemia (AML). She has a particular interest in AML associated with mutations in Fms-like Tyrosine Kinase-3 (FLT3), which is the most frequently mutated gene in AML and associated with resistance to conventional therapy. She has been involved in the development of multiple active FLT3 inhibitors as a clinical-translational investigator. Dr. Smith was born and raised in San Francisco, California. She attended Yale University where she majored in Chemistry, graduated cum laude, and was awarded the Howard Douglas Moore Prize for excellence in chemistry. She attended medical school at Duke University School of Medicine. Dr. Smith has been the recipient of numerous career development awards, including a prior Blood Cancer United Special Fellow in Clinical Research Award.

Program Name(s)

Career Development Program

Project Title

SHP2 and BCL2 Inhibition in Acute Myeloid Leukemia

University of Virginia

I am currently the Director of the Comprehensive Cancer Center at University of Virginia. I was also founding Director of Penn State Cancer Institute. I had excellent training at the Fred Hutchinson Cancer Research Center under Nobel Laureate, E.D. Thomas, and served on its faculty for many years. I was instrumental in discovering a new hematologic malignancy which I termed large granular lymphocyte (LGL) leukemia. My career path has been a testament to translational research as a vehicle to directly improve disease management and patient outcomes. I also have extensive leadership experience in team science, exemplified by my current NCI P01 funding. Since my discovery of LGL leukemia, I have consistently been at the forefront of translational research into this disease and have built an entire Program at the University of Virginia dedicated to helping improve outcomes for patients with this disease, which includes the LGL Leukemia Registry with >2,100 consented participants.

Program Name(s)

Translational Research Program

Project Title

Epigenetic Therapy and Nano-Drug Development for Large Granular Lymphocyte Leukemia

Houston Methodist Research Institute

My research focuses on the translation of bench works into new therapeutic approaches/strategies for multiple myeloma and other blood cancers. One of my research goals is to study the mechanism underlying the pathogenesis of myeloma and its associated tumor microenvironment. I have been investigating the interaction of myeloma cells with bone marrow adipocytes, bone cells, and the signaling pathways associated with obesity, inflammation, chemo-, immune-therapeutic resistance, and bone disease, which are the major hurdles in treating myeloma. As a PI, I have managed many projects funded by extramural grants, private funds, and industry alliances. Some highlights include the NIH/NCI R01s and funding received from ASH and ACS. I have an excellent publication track record with peer-review articles that are in top-tier journals with high impact factors (e.g. Cell Metabolism, Science Translational Medicine, Cancer Cell). My research experience shows my capacity to be a PI for this Project.

Program Name(s)

Translational Research Program

Project Title

Targeting acetyl-CoA synthetase 2 to remodel obesity-evoked inflammatory microenvironment in myeloma

Moffitt Cancer Center

Gary Reuther, PhD (Professor in the Departments of Molecular Oncology and Malignant Hematology at the Moffitt Cancer Center) earned his PhD from Duke University and did his post-doctoral training with Channing Der (University of North Carolina at Chapel Hill), where he studied signaling by the RAS oncoprotein and identified novel leukemia oncogenes. His current research centers on JAK2 signaling and novel therapeutic strategies for MPNs. Over the past 20 years, he has obtained research funding from multiple NIH R01 grants, the MPN Research Foundation, the Leukemia and Lymphoma Society, the Department of Defense, and the V Foundation for Cancer Research, and was the recipient of an American Cancer Society Research Scholar Award and an NIH/NCI Howard Temin Award. His experience leading research that led to this funding and successful completion and publication of these projects makes him highly qualified to serve as the PI on the proposed studies.

Program Name(s)

Translational Research Program

Project Title

Novel therapeutic strategies to improve the outcomes of patients with myeloproliferative neoplasms

The University of Texas MD Anderson Cancer Center

Dr. Luis Malpica is a physician-scientist at MD Anderson Cancer Center focused on creating better treatments for T-cell lymphomas. Originally from Peru, his early work on virus-associated blood cancers provided a strong foundation for his current research. Throughout his career, Dr. Malpica has earned prestigious awards from the American Society of Hematology, American Society of Clinical Oncology, Lymphoma Research Foundation and Leukemia Lymphoma Society. Dr. Malpica leads pioneering clinical trials that use the unique biology of a patient's cancer to guide smarter, targeted therapies, moving beyond traditional chemotherapy. He is deeply committed to collaboration, evidenced by his role in founding a Latin American lymphoma consortium and co-directing MD Anderson's multidisciplinary skin lymphoma clinic to speed patient access to new therapies. His ultimate goal is to establish a new, more effective standard of care for patients everywhere.

Program Name(s)

Career Development Program

Project Title

Defining Optimal Therapeutic Strategies in T-Cell Lymphomas Through Pathway Inhibition, Immune Modulation, and Chemotherapy Refinement

University of Chicago

Dr. Stock is the Anjuli Seth Nayak Professor of Leukemia Research at the University of Chicago. She is the co-chair of the Leukemia Committee for the Alliance, the NCI National Clinical Trials Cooperative Group that leads practice-changing clinical trials in cancer. Dr. Stock's career has focused on clinical/translational research in acute leukemias, with a particular focus on acute lymphoblastic leukemia (ALL). Her work has led to a paradigm shift in treatment of younger adults with ALL, translating preclinical insights from her laboratory studies on mechanisms of treatment resistance. She is the co-chair of the Clinical and Experimental Therapeutics Program at the University of Chicago Comprehensive Cancer Center. Dr. Stock serves on the editorial board of Blood Advances and has published more than 250 peer-reviewed manuscripts. She was recently elected to a 4-year term as the Councilor for the American Society of Hematology.

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

A phase 1b/2 study targeting apoptotic and signaling pathways in T-acute lymphoblastic leukemia