Who We Fund

The Brigham and Women’s Hospital

My scientific background involves the functional characterization of rare immune cells called dendritic cells in advanced melanoma patients. These cells are master regulators of immunity and are responsible for orchestrating anti-cancer responses driven by effector cells called T cells. My PhD focused on patients who received immunotherapy via antibodies that reinvigorate the immune system, also known as immune checkpoint inhibitors. I collected patient blood samples before and during treatment, and found that a critical subtype of dendritic cell is numerically and functionally impaired in patients who did not respond to immunotherapy compared to those who responded. In my current lab, I leveraged my experience in immune cell research and now study how a novel drug combination can be used to target and kill acute myeloid leukemia (AML) cells. This innovative approach targets two biologically important processes within a cell – the protein-making machinery and the control of cell death.

Program Name(s)

Career Development Program

Project Title

Cotargeting oncogenic protein translation and apoptosis in acute myeloid leukemia

TAP Partner

Allotera (formerly Wugen) is a clinical-stage biotechnology company focused on developing next-generation, allogeneic CAR-T cell therapies for cancer. Allotera's proprietary gene-editing platform is designed to overcome key limitations of first-generation cell therapies, enabling scalable, off-the-shelf treatments with biologics-like cost of goods margins.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 2 registration-directed clinical study of Sofi-cel, an allogeneic CAR-T targeting CD7, in patients with T-ALL/T-LBL

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 Wisconsin at Madison

I have been interested in basic and translational work related to cancer since my master’s resulting in a thesis on bone metastasis of breast cancer. Part of my PhD work explored the molecular mechanisms of chronic myelogenous leukemia (CML). I have studied the use of metformin, as an anti-cancer agent and demonstrated that metformin through activation of AMPK/ RUNX1/ SOCS3 axis and inhibition of glycolytic fluxes overcomes the imatinib resistance in CML patients. I joined Dr. Jing Zhang’s lab at the University of Wisconsin. Under her guidance, I had a chance to explore the molecular players involved in a subset of acute myeloid leukemia (AML) with concurrent RAS and ASXL1 mutations, which define one of the worst AML prognosis groups. I have been actively collaborating with numerous investigators on and off campus to work on patient derived xenograft model for pre-clinical development of therapeutic approaches targeting both leukemia cells and suppressed T cells in NRAS; ASXL1 AML.

Project Title

Enhancing the cytotoxicity of T cells in NRAS; ASXL1-AML through combined inhibition of MEK and HDACs

MD Anderson Cancer Center

Dr. DiNardo is an academic clinical researcher with a primary focus on individualized therapy and precision oncology for myeloid malignancies, including the optimal incorporation of genomics into standard risk assessments and treatment algorithms, and the clinical evaluation of targeted therapeutics for molecularly-defined patient subgroups.

Program Name(s)

Career Development Program

Project Title

A precision-based all-oral combination of venetoclax, oral decitabine, and IDH1/2 targeted inhibition for patients with IDH1 or IDH2 mutated AML

Washington University in St. Louis

John F. DiPersio MD, PhD is the Golman Professor of Medicine and Director of The Center for Gene and Cellular Immunotherapy at the Washington University School of Medicine. His research has focused on targeting key elements of the hematopoietic niche for optimal stem cell mobilization and chemosensitization, mitigating GvHD in T cell replete transplants, understanding the genomic alterations in AML, and developing and testing in the clinic novel therapeutics and immuno-therapeutics, including cellular therapies, for the treatment of AML, ALL, T/B-NHL and multiple myeloma. Dr. DiPersio was instrumental in the development and FDA approval of Plerixafor, Motixafortide, and Ruxolitinib. He is the recipient of multiple awards and was past president of the ASTCT and member of the NCI Board of Scientific Counselors. He has authored or co-authored more than 490 publications, is a co-founder of two companies (WUGEN and Magenta) and holds multiple patents.

Program Name(s)

Translational Research Program

Project Title

KT1, a novel NK trispecific antibody for the treatment of AML and MDS

Yale

Dr. Manyi Wei gained his B.S. degree at China Pharmaceutical University in Nanjing. Subsequently, he completed his Ph.D. training in Cell Biology at the Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences. During his Ph.D. research, Dr. Wei focused on the regulatory role of long non-coding RNA in gene expression and its impact on neuronal function. Dr. Wei has always nurtured a particular interest in understanding and developing novel treatment for leukemia. After his Ph.D. training, Dr. Wei joined the laboratory of Dr. Stephanie Halene at Yale Cancer Center and Yale University School of Medicine. He immediately began exciting work on RNA modifications and their functions in the initiation and progression of acute myeloid leukemia and myelodysplastic syndromes and importantly on novel therapeutic approaches exploiting cell intrinsic and extrinsic effects of targeted inhibitors of RNA methylation. His goal is to contribute to the cure of leukemia.

Program Name(s)

Career Development Program

Project Title

Harnessing METTL3 inhibition in acute megakaryoblastic leukemia driven by the t(1;22) fusion involving a member of the m6A writer complex

University of Michigan

Dr. Jolanta Grembecka is an Associate Professor in the Department of Pathology, University of Michigan. Dr. Grembecka’s research is focused on development of small molecule inhibitors of proteins involved in leukemogenesis. Her laboratory has developed the first small molecule inhibitors of the menin-MLL1 interaction as a treatment for acute leukemia, which were advanced to clinical studies in acute myeloid leukemia patients. Her laboratory is also developing new targeted therapies for hematologic cancers by blocking novel epigenetic targets, including ASH1L histone methyltransferase.

Dr. Grembecka has received PhD in Chemistry at Wroclaw University of Technology, Poland. She completed postdoctoral training in drug discovery at the University of Virginia and in 2009 started her independent position at the University of Michigan. Dr. Grembecka is a co-author on over 80 scientific publications and an inventor on 15 patents. She is LLS Scholar and ACS Research Scholar recipient.

Program Name(s)

Translational Research Program

Project Title

ASH1L degradation as a new treatment for acute leukemia

Targeted combination therapies for leukemia with NUP98 translocations

The Feinstein Institutes for Medical Research

My lab focuses on the tumor microenvironment as a risk factor for chronic lymphocytic leukemia (CLL) and Richter's transformation (RT). We develop therapeutic strategies targeting the interaction between CLL/RT and the TME. This research has been my focus since my postdoctoral studies, where I investigated auto-antigens in the TCL-1 mouse model and expanded this work in TCL-1/IgK-AID double transgenic mice that develop RT spontaneously. I've also created preclinical CLL patient-derived xenograft (PDX) models, including SRG-BA6 mice, which allow for the study of human lymph node fibroblastic stromal cells. Recently, I’ve gained expertise in single nucleus RNA sequencing, multiplex imaging, and using PDX models to develop novel therapies like R110-CART for CLL and RT. My lab is committed to interdisciplinary studies with therapeutic implications, and I have extensive experience in project management and collaboration, positioning me well to advance this research.

Program Name(s)

Translational Research Program

Project Title

Targeting TLR9 Signaling to restore immunomodulating function of FRCs in Richter's Transformation

Duke University

I am a board-certified oncologist and palliative care physician, Associate Professor of Medicine in the Duke University School of Medicine, and member of the Duke Cancer Institute. My practice focuses on the care of patients with blood cancers, while my research examines “patient experience” issues in hematology. As founding director of the Cancer Patient Experience Research Program (CPEP), I conduct randomized trials of integrated palliative care interventions in cancer care, and other patient experience studies, and have published over 175 manuscripts to date. I co-led the first-ever multisite, randomized trial of integrated palliative care in hematology, showing that palliative care integration dramatically improves quality of life, anxiety, depression, and post-traumatic stress among patients with acute myeloid leukemia hospitalized for high-dose chemotherapy. I am widely recognized as a leading expert in palliative care and patient experience research in hematology.

Program Name(s)

Career Development Program

Project Title

Patient Experience Research and Palliative Care Integration in Malignant Hematology

Dana-Farber Cancer Institute

I am a hematologic oncologist and cancer care delivery researcher. The focus of my research is to improve quality of life (QOL) and quality of care for patients with hematologic malignancies. I have designed, successfully applied for funding, and published many studies relating to health services and outcomes for patients with blood cancers. For example, I led the international validation of a QOL scale developed for patients with myelodysplastic syndromes (MDS), the QUALMS, which has since been incorporated into several clinical trials and registries for patients with that disease. Additionally, I led a study using QOL assessment to help with transfusion decisions for patients with MDS, which is highly relevant to the final aim of my HCL 2030 proposal. I would be delighted to lead the team to complete the aims of the proposed research; moreover, the project will satisfy my goal to bring my research expertise to bear on the care that I provide for patients with hairy cell leukemia.

Program Name(s)

Hairy Cell Leukemia Research Initiative

Project Title

Quality of Life, Outcomes, and Decision Making for Patients with Hairy Cell Leukemia

Princess Margaret Cancer Centre, University Health Network

Tak W. Mak is an international leader in cancer and immunology research. He is best known for his cloning of the human T cell receptor in 1984, which led to the CAR-T therapies now approved for leukemias/lymphomas. His lab also generated numerous genetically modified mouse strains to identify key factors in immune disorders and cancers. His group’s demonstration that CTLA4 negatively regulates T cell activation paved the way for checkpoint inhibitor immunotherapy. Most recently, his team showed that T and B cells produce acetylcholine in a manner influencing tumorigenesis and autoimmunity. On the biotech front, Dr. Mak co-founded Agios Pharmaceuticals, which produced two IDH inhibitors that are now FDA-approved for treatment of AML. The Mak team has also developed two novel agents targeting aneuploid cancer cells. These agents have shown promise in phase 2 clinical trials. Dr. Mak has published >950 papers, holds 20 patents, and has received over 35 national and international awards.

Program Name(s)

Specialized Center of Research Program

Project Title

The Immune Niche in the Development of Hematological Malignancies and Implications for Novel Therapy