Who We Fund

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

Dana-Farber Cancer Institute

Robert J. Soiffer, MD, is Chief of the Division of Hematologic Malignancies and Chair of the Executive Committee for Clinical Programs at Dana Farber Cancer Institute as well as the Worthington and Margaret Collette Professor of Medicine in the Field of Hematologic Oncology at Harvard Medical School.

Dr. Soiffer served as President of the American Society for Transplant and Cellular Therapies and Chair of the Advisory Board for the Center for International Blood and Marrow Transplant Research. He is a member of the Executive Steering Committees for Blood and Marrow Transplant Clinical Trials Network. Dr. Soiffer is Immediate Past Chair of the Board of Directors for the National Marrow Donor Program.

Dr. Soiffer conducts research focused on modulation of immune reconstitution in the setting of allogeneic hematopoietic stem cell transplantation (allo-HCT). His goal is to develop technologies to improve patient outcomes by optimizing graft versus leukemia (GVL) activity without inducing graft versus host disease (GVHD). Dr. Soiffer has co-authored more than 400 peer-reviewed manuscripts and numerous book chapters, review articles, editorials, and monographs.

Program Name(s)

Specialized Center of Research Program

Project Title

Understanding and Overcoming Mechanisms of Immune Evasion after Allogeneic Transplant

Memorial Sloan Kettering Cancer Center

I am a clinical-translational researcher focusing on new treatments for lymphomas with a particular focus on T-cell/cutaneous lymphomas. Since entering the field of hematology/oncology, I have been drawn to the study of lymphoma and the care of patients facing lymphoma given how varied these conditions are and the major unmet needs that exist. In my clinical practice, I see patients and caregivers navigating these diseases, treatments, and side effects, and this stimulates me to pursue advancements in my research endeavors. My overarching goals are to bridge laboratory investigations with work done in clinical trials towards ultimately improving and refining our therapeutic approaches for these challenging diseases.

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

Targeting mutant IDH2 in angioimmunoblastic T-cell lymphoma

Dana-Farber Cancer Institute

Constantine Mitsiades MD, PhD, is an Assistant Professor at Dana-Farber Cancer Institute (DFCI), Harvard Medical School, an Associate member of the Broad Institute, Cambridge, MA, and holds the "Shawna Ashlee Corman" Investigatorship in Multiple Myeloma at DFCI. His research focuses on understanding the mechanisms through which myeloma and other blood cancers interact with the bone marrow microenvironment and develop resistance to existing or investigational drugs or immune therapies, and how to target therapeutically those resistance mechanisms. His studies established that inhibition of BET bromodomain proteins blocks the critical oncoprotein c-Myc. His research also informed the design of several regimens which are now FDA-approved, represent a standard-of-care for MM treatment, and have become a "backbone" for combination with other novel agents, e.g., monoclonal antibodies. Several of these regimens contributed to the increased overall survival of MM patients in the last decade.

Program Name(s)

Translational Research Program

Project Title

Pharmacological strategies to enhance T- and NK-cell-based therapies in blood cancers

St. Jude Children's Research Hospital

Dr. Trent Hall is currently a Postdoctoral Research Associate in the laboratory of Dr. John Crispino at St. Jude Children’s Research Hospital in Memphis, TN. Dr. Hall received his doctorate from the University of Tennessee Health Science Center in 2020 studying hematopoietic stem cell development in Dr. Shannon McKinney-Freeman’s laboratory. Dr. Hall’s current research interests include predisposition to myeloid malignancies and hematopoietic development.

Program Name(s)

Career Development Program

Project Title

Identifying novel regulators of leukemic progression in GATA2-deficiency syndrome

University of Colorado Denver, Anschutz Medical Campus

Terry Fry, MD, is a professor of pediatrics, hematology and immunology at the University of Colorado School of Medicine. He is the chair of the Gates Institute Advisory Board and holds the Charles C. Gates Endowed Chair in Regenerative Medicine. He arrived at Colorado in 2018 after serving as head of the Hematologic Malignancies Section in the Pediatric Oncology Branch at the National Institutes of Health (NIH), where he led efforts in cellular immunotherapy for pediatric leukemia. Prior to the NIH, Dr. Fry was chief of Blood and Marrow Transplantation at Children’s National Medical Center in Washington, D.C. His research focuses on the preclinical and clinical development of chimeric antigen receptor T cells for pediatric cancers. He serves on the Committee for Scientific Affairs for the American Society of Hematology, is vice chair for biology in the Cellular Therapy Committee of the Children’s Oncology Group, and was elected into the American Society for Clinical Investigation.

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

A phase 1 study of anti-CD64 CAR T cells in patients with venetoclax-refractory myeloid neoplasms

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

TAP Partner

Ryvu Therapeutics is a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology using a proprietary discovery engine platform.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 2 study of RVU120, a novel CDK8 inhibitor, in patients with myelofibrosis

A phase 2 study of RVU120, a novel CDK8 inhibitor, in combination with venetoclax in patients with AML

Weill Cornell Medicine

Dan Landau, MD, PhD is Associate Professor of Medicine at Weill Cornell Medicine and a Core Member of the New York Genome Center. He is an hemato-oncologist whose long-term goal is to develop novel approaches to address cancer evolution as a central obstacle to cure. His research group is funded by the NCI, NHLBI and NHGRI, and his work has led to recognition and awards including Stand Up to Cancer, Burroughs Wellcome Fund, Vallee Scholar, and the NIH Director’s New Innovator Award.

Program Name(s)

Career Development Program

Project Title

Defining the role of DNA methylation modifier mutations in reshaping blood differentiation topology

Washington University in St. Louis

Dr. Fehniger is a physician-scientist that leads a research program focused on translational NK cell biology and therapy. His group pioneered studies characterizing memory-like (ML) NK cell biology and activity against AML and has led clinical trials advancing ML NK cell adoptive therapy for both adult and pediatric patients. Dr. Fehniger is director of the Biologic Therapies Core Facility and Laboratory Director of the Center for Gene and Cellular Immunotherapy. His team developed the platform and protocols for production of GMP grade ML NK cells for use in academic clinical trials. His lab performs correlative immunology to understand ML NK cell biology and identify mechanisms of resistance to NK cells in patients. Dr. Fehniger has extensive experience in human NK cell biology, flow and mass cytometry, single cell analysis, and immunotherapy. For the proposed clinical trial, Dr. Fehniger will work with clinical co-investigators in pediatric and adult stem cell transplant programs.

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

NK cell immunotherapy to reduce relapse after haploidentical transplant for high-risk pediatric AML

Washington University in St. Louis

Stephen Oh, MD, PhD is Associate Professor of Medicine in the Division of Hematology at Washington University School of Medicine. Dr. Oh’s clinical and laboratory research efforts are focused on myeloproliferative neoplasms (MPNs). His group employs patient samples and animal models to investigate fundamental mechanisms driving the initiation, development, and progression of MPNs. A major focus of the laboratory is to utilize mass cytometry and multiplex imaging approaches to interrogate dysregulated cytokine signaling networks in MPNs. Dr. Oh has extensive clinical experience in the diagnosis and management of MPN patients and has contributed to numerous clinical trials investigating novel targeted therapies for MPN patients. The long-term objective of his work is to translate the findings from his laboratory research into improved therapies for MPN patients.

Program Name(s)

CMML Initiative

Project Title

Exploiting Novel Therapeutic Vulnerabilities in Chronic Myelomonocytic Leukemia

Case Western Reserve University School of Medicine

I (Reshmi Parameswaran) am a scientist working in the field of cancer immunotherapy for past 22 years. I received my Ph.D degree from Weizmann Institute of Israel in cell biology and then did postdoctoral training at Children's Hospital Los Angeles working on understanding the drug resistance mechanism of Acute Lymphoblastic Leukemia. Later I moved to Case Western University and my laboratory is focusing on developing new immunotherapies for liquid and solid cancers. We generated a new BAFF ligand based CAR-T therapy to treat B cell cancers and this entered into three clinical trials for treating lymphoma, multiple myeloma and autoimmune lupus. All are currently in phase-1 clinical trials. I have published about 25 articles on cancer immunotherapy and received multiple research grants to develop new immunotherapy strategies for cancer. I am committed to find the reason why HCLv patients fail chemotherapy and to develop a new therapy to sensitize these patients to standard chemotherapy.

Program Name(s)

Hairy Cell Leukemia Research Initiative

Project Title

Understanding the drug resistance mechanism and developing new therapeutic strategies for Hairy Cell Leukemia variant