Who We Fund

Dana-Farber Cancer Institute

Eric Smith, MD PhD is Director of Translational Research for Immune Effector Cell Therapies at Dana-Farber Cancer Institute (DFCI) where his primary responsibility is as PI of a gene and cell engineering pre-clinical laboratory. He is an Assistant Professor at Harvard Medical School (Immunology PhD Faculty); Associate Member at the Broad Institute of Harvard and MIT; a Member at the Parker Institute for Cancer Immunotherapy; and the founding faculty director of the IMmunotherapy Platform for Antibody and CAR Therapeutics Development and Translation (IMPACT2) Center at DFCI. He received his MD/PhD and internal medicine training at the Mount Sinai School of Medicine and medical oncology and post-doctoral training at Memorial Sloan Kettering Cancer Center and stayed on as faculty in the Cellular Engineering Center and Myeloma/Cellular Therapy services. He was recruited to DFCI in 2020 to advance the home-grown adoptive cellular therapy pipeline there. The Smith Lab for Genetic and Cellular Engineering focuses on pre-clinical efforts to advance the field and developing therapies for both hematologic and solid tumors for the benefit of patients. More than a dozen clinical trials have been initiated stemming from his team’s lab work. The most developed of which is a first-in-class CAR targeting GPRC5D, a target he described as important for the immunotherapy of myeloma; he and his colleagues reported the phase I study in NEJM, and a phase II multi-center registration study is now ongoing.

Program Name(s)

Discovery

Project Title

Interrogating T-cell apoptotic priming to improve CAR-T persistence in treatment of lymphoid malignancies

Boston Children's Hospital

Vijay G. Sankaran, MD, PhD is the Jan Ellen Paradise, MD Professor of Pediatrics at Harvard Medical School, an Investigator of the Howard Hughes Medical Institute, an Attending Physician in the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and an Associate Member of the Broad Institute. Dr. Sankaran's lab seeks to understand the influence of human genetic variation on blood and immune cell production in health and disease. Their work has resulted in a number of therapies for blood diseases, including work that led to the development of Casgevy for sickle cell disease and beta-thalassemia. Dr. Sankaran has received a number of awards for his work including the 2019 Seldin-Smith Award for Pioneering Research from the American Society of Clinical Investigation, the 2022 E. Mead Johnson Award from the Society for Pediatric Research, and 2024 Trailblazer Prize from the Foundation for the National Institutes of Health.

Program Name(s)

Discovery

Project Title

Inherited resilience to clonal hematopoiesis and myeloid malignancy by modifying stem cell RNA regulation

Ospedale Pediatrico Bambino Gesù

Dr. Del Bufalo is a physician-scientist with interest in childhood malignancies, tumor immunology, immunotherapy and hematopoietic stem cell transplantation. She participated in the preclinical and clinical development of the academic clinical trials with CAR-T cells at OPBG. In particular, she coordinated 2 studies on different CD9-CAR T cell platforms for the treatment of children with relapsed/refractory (r/r) BCP-ALL and the phase I/II study to test GD2-CART01 in children with r/r neuroblastoma. She is the PI of a research project to test donor-derived CAR T cells for the treatment of children/young adults with BCP-ALL. She managed the activation and conduction of the phase I clinical trial on CD7-CART.PEBL for the treatment of children/young adults with r/r T-ALL (PI Prof. Franco Locatelli). She is the new chair of the European iBFM Resistant Disease Committee and President of the Young Investigators Section of the scientific technical committee of the Italian Ministry of Health.

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

CD7-CAR T cells with PEBL for treatment of children/young adults with relapsed/refractory T-ALL

Dana-Farber Cancer Institute

My passion for hematology started during my studies in Italy and France, where I investigated signaling pathways and mutated transcription factors in B-cell malignancies. After graduation, I switched focus to myeloid malignancy, by pursuing a PhD in the Vyas lab at the University of Oxford, known for having characterized leukemic stem cells in acute myeloid leukemia. There, by developing and deploying novel single-cell approaches, I studied how pre-leukemic clones gain advantage in human clonal hematopoiesis. Moreover, I assessed how gene mutations were associated with altered transcription and epigenetic de-regulation, therefore likely promoting leukemic cell maintenance and fueling relapse. Now, by joining the Armstrong Laboratory, I look forward to studying the principles of epigenetic regulation underlying oncogenic programs in this deadly disease, by performing more mechanistic studies characterizing the functional roles of chromatin complexes in leukemogenic gene expression.

Program Name(s)

Career Development Program

Project Title

Mechanisms of oncogenic gene regulation by MYST-family acetyltransferases KAT6A and KAT7 in AML

The Ohio State University

I am a tenured Professor in the Division of Hematology at The Ohio State University, with a focus on translational research in Chronic Lymphocytic Leukemia, the Associate Director for Clinical Research within the Division of Hematology, the Section Head for the CLL/Hairy cell leukemia group, and the co-leader of the Leukemia Research Program within the OSUCCC. I am the PI for multiple early-stage clinical trials investigating novel targeted therapies for CLL and other hematologic malignancies, chaired the intergroup Phase III study A041202 which investigated chemoimmunotherapy versus targeted therapy as initial therapy for older adults with CLL, and now chair A041702 which is investigating continuous versus intermittent targeted therapy in the same patient population. My laboratory research focuses on resistance to targeted therapies in CLL. Our group identified C481S and PLCG2 mutations as the primary resistance mechanisms for covalent BTK inhibitors and were the first to investigate reversible BTK inhibitors preclinically as a strategy to circumvent BTK inhibitor resistance.

Program Name(s)

Translational Research Program

Project Title

Inhibition of PKCβ as a strategy for BTK inhibitor refractory CLL

City of Hope National Medical Center

Dr. Siddiqi is an associate professor in the Department of Hematology/Hematopoietic Cell Transplantation and Director of the chronic lymphocytic leukemia (CLL) program at COH. As an active member of the Toni Stephenson Lymphoma Center and the Immunotherapy Center at COH, she has been the institutional and, for some studies, national principal investigator of many phase 1, 2 and 3 clinical trials involving novel targeted therapies and cellular therapeutics such as chimeric antigen receptor (CAR) T cells in CLL and non-Hodgkin lymphomas. She works closely with Cancer Therapy Evaluation Program (CTEP), cooperative groups, and pharmaceutical companies on important clinical trials in order to bring novel, potentially lifesaving, therapeutics to our patients. As of June 1, 2021, she will be the Lymphoma Medical Director at the Irvine campus, set up open August 2022, which puts her in an ideal position to open impactful hematology clinical trials at CAN sites, starting with Orange county.

Program Name(s)

IMPACT

Project Title

Establishing Hematology Clinical Trial Hubs within the City of Hope Community and Affiliate Network

University of Miami

Dr. Nimer has cared for patients with MDS, AML, multiple myeloma, and lymphoma for over three decades. This melding of clinical studies and care, with both basic laboratory and translational studies, reflects the fundamental focus of his career. Since coming to the University of Miami-Miller School of Medicine in 2012 and assuming the Directorship of the Sylvester Comprehensive Cancer Center, the center received the prestigious National Cancer Institute designation in July 2019. In November 2019, Dr. Nimer was named the inaugural Oscar de la Renta Endowed Chair in Cancer Research. He has been elected to the American Society of Clinical Investigators and the Association of American Physicians. He is a Fellow of the American College of Physicians and serves on the editorial board of several medical journals. In April 2021, Dr. Nimer was inducted into the Academy of Science, Engineering, and Medicine of Florida. Dr. Nimer is also the Chairman of the Myelodysplastic Syndrome Foundation, and the Chairman of the Medical Advisory Board of Gabrielle's Angel Foundation for Cancer Research.

Program Name(s)

Specialized Center of Research Program

Interventional Epigenetics in Myeloid Malignancies

Stanford University

Gerlinde Wernig, M.D. is an Associate Professor of Pathology at Stanford University and a world expert on fibrosis and blood cancers. She co-discovered the JAK2 V617F mutation that drives myeloproliferative neoplasms and identified c-JUN as a key regulator of fibrosis across organs. Her laboratory develops advanced models of human fibrotic disease, using patient-derived organoids, xenografts, and mouse systems to uncover how inflammation and cancer signaling reshape tissues. By combining next-generation single-cell and multi-omics approaches, Dr. Wernig’s research identifies new targets to treat fibrosis and bone-marrow failure. As a clinical hematopathologist, she brings deep diagnostic expertise to bridge scientific discovery and patient care.

Program Name(s)

Hairy Cell Leukemia Research Initiative

Project Title

Identifying Prognostic Markers of Relapse in Hairy Cell Leukemia (HCL) and HCL-Variant by Profiling Mutated Blood Stem Cells and Fibrosis Pathways

Washington University in St. Louis

Dr. Tyler Parsons completed his PhD research at Oakland University and the Beaumont Research Institute in the lab of Dr. Gerard Madlambayan where he published on the role of blood stem cells in tumor response to radiation therapy. During the final year of his PhD, he was diagnosed with a myeloproliferative neoplasm (MPN) which directed his career focus and passion to furthering the understanding of MPNs and their transformation potential to leukemia. To this end, he joined the lab of Dr. Grant Challen at Washington University School of Medicine (St. Louis) where he is investigating clonal evolution in MPNs and the mutational trajectory leading to secondary leukemia. The aim of his post-doctoral fellowship is to describe the clonal architecture of MPN disease progression to leukemia which could lead to early detection and improved disease surveillance. He is passionate about improving outcomes for patients by advancing our understanding of both the biology and disease evolution of MPNs.

Program Name(s)

Career Development Program

Project Title

Mechanisms of Clonal Evolution in the Transformation of MPN to sAML

Stanford University

I am a postdoctoral researcher at Stanford University studying how changes in blood-forming stem cells contribute to aging and blood cancers. I completed my PhD in Molecular Genetics at the University of Nebraska Medical Center, where I discovered new gene regulatory mechanisms that support red blood cell recovery after stress, including blood loss, inflammation, and aging. My graduate work resulted in multiple first-author publications and several research awards. At Stanford, my research focuses on how inherited genetic factors and hormone signaling influence the earliest stages of blood cancer development. I am passionate about translating basic scientific discoveries into better ways to detect and treat leukemia. My long-term goal is to lead my own research team dedicated to improving outcomes for people with blood disorders and preventing their progression to blood cancer.

Program Name(s)

Career Development Program

Project Title

Dissecting the Role of Thyroid Hormone Receptor Beta in Driving Clonal Hematopoiesis and Hematologic Malignancy

Virginia Commonwealth University

Venkata Battula, Ph.D., serves as Assistant Director of Massey Comprehensive Cancer Center’s Cancer Research Training and Education Coordination (CRTEC) program and is a Professor in the Department of Internal Medicine at the VCU School of Medicine. He joined VCU Massey from The University of Texas MD Anderson Cancer Center, where he was an Associate Professor in the Departments of Leukemia and Breast Medical Oncology within the Division of Cancer Medicine. He earned his Ph.D. in Human Cell Biology from Justus Liebig University in Giessen, Germany, and brings extensive expertise in translational cancer research.

Dr. Battula’s research focuses on understanding how cancer cells develop resistance to chemotherapy, radiation, and targeted therapies; identifying novel drug resistance mechanisms; and developing new combination therapies. His work has been instrumental in identifying GD2 as a novel marker in patients with triple-negative breast cancer (TNBC), an aggressive subtype with limited treatment options. GD2, expressed on cancer stem cells, is associated with tumor growth and poor clinical outcomes. Building on these findings, Dr. Battula recently secured funding from the U.S. Department of Defense to initiate a clinical trial targeting GD2 in TNBC.

In his role within CRTEC, Dr. Battula is committed to expanding graduate-level training opportunities and advancing programs that prepare the next generation of cancer researchers.

Program Name(s)

Translational Research Program

Project Title

Arming NK Cells to Target B7-H3+ AML Cells

University of Manchester

Sam Butterworth joined the University of Manchester as a Senior Lecturer in Medicinal Chemistry in November 2016. Prior to this he worked at the University of Birmingham from 2013 and at AstraZeneca from 2005-2013. During this time he has been accountable for chemistry strategy and delivery for all phases of drug discovery projects from target review and hit generation, through to lead optimisation and pre-clinical development. His work at AstraZeneca led to the development of a targeted anti-cancer agent osimertinib that was approved by the FDA in November 2015, and along with his colleagues Sam has been recognised for this work through the 2017 RSC Malcolm Campbell Award and the 2018 ACS Heroes of Chemistry award. Since returning to academia he has established national and international collaborations focussing on translation research, predominantly in Oncology, and has been awarded >£8M translational funding as PI.

Program Name(s)

CMML Initiative

Project Title

Development of peptide-drug conjugates for the treatment of Chronic Myelomonocytic Leukaemia (CMML)