Who We Fund

Cincinnati Children’s Hospital Medical Center

I am an Assistant Professor at Cincinnati Children's Hospital Medical Center. My areas of research interest include drug development and signaling with focus on small GTPases. My research is both basic and translational.

My research efforts encompass several methodologies, including structural biology, biophysical and biochemical studies, cellular functional assays, and ultimately, identifying small molecule compounds that bind to and modulate GTPase signaling in in vivo pre-clinical models of cancer.

RAC GTPases are key regulators of cell growth. By reorganizing the actin cytoskeleton, RAC plays a key role in cancer cell metastasis. It is also involved in mechanisms of resistance to therapies. My lab's goal is to inhibit RAC in leukemia by understanding the molecular mechanisms driving its hyperactivity.

One of my lab’s groundbreaking discoveries is the identification of a small molecule inhibitor of VAV3, a RAC activator. Current research studies the efficacy of VAV3 inhibition in models of relapsed/recurrent leukemia.

Program Name(s)

Translational Research Program

Project Title

Overcoming RAS-driven Mechanisms of Resistance in Leukemia

Columbia University Irving Medical Center

My research interests are the identification of genetic alterations responsible for leukemia and lymphoma development and the elucidation of the mechanisms leading to transformation in lymphoid diseases. My academic training and research experience give me an excellent background in multiple biological disciplines including cellular and molecular biology and genetics. I completed my PhD at the INSERM in France where I demonstrated the cooperation between an activating mutation of Jak3 and trisomy 21 in the development of cutaneous T cell lymphoma. I also identified a major cooperative role for the RAS/MAPK signaling pathway in the development of Down syndrome-associated B-cell acute lymphoblastic leukemia. Currently, I am a postdoctoral researcher at Columbia University, under the mentorship of Dr. Teresa Palomero, and I study the mechanisms of transformation in Peripheral T-cell lymphoma using multidisciplinary approaches from animal models to transcriptomic and epigenomic analysis.

Program Name(s)

Career Development Program

Project Title

Role of ID2 in mature T-cell lymphoma

Children's Research Institute

Dr. Bollard received her medical degree at the University of Otago. She is board certified both in pediatrics and hematology. She is currently the Bosworth Chair for Cancer Biology, the Director of the Center for Cancer and Immunology Research, and the Director of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National Health System. She is a Professor of Pediatrics and of Microbiology, Immunology, and Tropical Medicine at The George Washington University and the Associate Center Director for Translational Research and Innovation at the GW Cancer Center. Dr. Bollard is a member of the American Society for Clinical Investigation (ASCI) and is the current President of the Foundation for the Accreditation of Cellular Therapy (FACT). She is currently Editor in Chief of Blood Advances. She has over 200 peer reviewed publications. Her bench and translational research focuses on improving outcomes for patients after hematopoietic stem cell transplantation and on the development of novel cell therapies for cancer and virus-associated diseases.

Program Name(s)

Translational Research Program

T cells with native and chimeric receptors against multiple tumor targets for acute myeloid leukemia

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

TAP Partner

Vittoria Biotherapeutics is developing novel CAR-T cell therapies that transcend the limitations of current cell therapies. Based on technology exclusively licensed from the University of Pennsylvania, Vittoria's proprietary Senza5 platform unlocks the antitumor potential of engineered T cells and utilizes a five-day manufacturing process to maximize stemness, durability, and target cell cytotoxicity. By acting on the fundamental biology of T cells, Senza5 can be used to improve the efficacy of engineered T cell therapies with pipeline applications in oncology and autoimmune diseases.

Program Name(s)

Therapy Acceleration Program

A phase 1 study of VIPER-101, a CD5-edited dual population chimeric antigen receptor cell therapy, to enhance immunotherapy against T-cell non-Hodgkin lymphoma

TAP Partner

Caribou is a clinical-stage biotechnology company, co-founded by CRISPR pioneer and Nobel Prize winner Jennifer Doudna, Ph.D., using next-generation CRISPR genome-editing technology to develop “off-the-shelf” (allogeneic) CAR therapies for hard-to-treat blood cancers.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 1 study of CB-010, a CRISPR-edited allogeneic CAR-T targeting CD19, in patients with B-cell NHL

A phase 1 study of CB-011, a CRISPR-edited allogeneic CAR-T targeting BCMA, in patients with multiple myeloma

Van Andel Research Institute

I am an Associate Professor in the Department of Epigenetics at Van Andel Institute. My research is focused on epigenetic regulation of gene expression during blood cell formation and in the pathogenesis of blood cancer. I obtained PhD in Biochemistry from the Chinese Academy of Sciences in 2001 and joined Dr. Joseph Lipsick’s laboratory at Stanford University for my postdoctoral training, where I became interested in blood cancer research. In 2008, I joined MD Anderson Cancer Center as a research track Assistant Professor, where I made seminal discoveries of the ENL protein as a novel histone acetylation reader and an attractive new therapeutic target for acute leukemias. In 2018, I started my independent lab at Van Andel Institute studying epigenetic regulation of gene expression in blood cancers. The overarching goal of my research is to understand and target ENL in acute leukemias. My long-term research goal is to translate our discoveries at bench to help leukemia patients in the clinic.

Program Name(s)

Career Development Program

Project Title

Investigating and targeting the histone acetylation reader protein ENL in acute leukemias

University of California, San Francisco

Dr. Elliot Stieglitz is a physician-scientist at the University of California, San Francisco whose research focuses on children diagnosed with juvenile myelomonocytic leukemia (JMML). He recently chaired a study, ADVL1512, a phase II clinical trial that tested the safety of trametinib in children with relapsed JMML. This trial met its primary objective and closed to accrual at the end of 2022. Dr. Stieglitz’s main laboratory focus is on developing novel therapies for JMML including CAR-T cells. He has generated patient-derived xenograft (PDX) models of JMML that will serve as the pre-clinical model in which to test CAR-T cells on this grant. These PDXs were generated in collaboration with Dr. Eric Padron, a key opinion leader in CMML and a collaborator on this grant. Dr. Stieglitz has also collaborated extensively with Dr. Tasian, an international leader in CAR-T therapy who is a Co-PI on this grant. This multi-disciplinary team will work together to advance CLL-1 CAR-T cells and trametinib into the clinic for CMML and JMML patients.

Program Name(s)

CMML Initiative

Project Title

CLL-1 CAR-T cells and trametinib for the treatment of Ras-mutated CMML and JMML

The University of Melbourne

Professor Dawson is the Associate Director for Research Translation, a Program Head in Laboratory Research and a Consultant Haematologist at the Peter MacCallum Cancer Centre. His research interest is studying the role of epigenetic regulators in the initiation, maintenance and progression of cancer. His current research spans cell and molecular biology, functional genomics, cancer immunology, chemical biology and clinical translation. He is the Sir Edward Dunlop Fellow for the Cancer Council of Victoria and a HHMI International Research Scholar. In recognition of his research achievements, he has been elected to the Australian Academy of Science, the Australian Academy of Health and Medical Sciences and an EMBO member. He has received several prestigious awards including the McCulloch & Till Award from the International Society of Experimental Haematology, the Jacques Miller Medal from the Australian Academy of Science and the Prime Minister’s Prize as Life Scientist in 2020.

Program Name(s)

Translational Research Program

Project Title

Understanding molecular determinants of immune evasion to CAR-T cells at single clone resolution

Institute of Cell Biology (Tumor Research) at the Medical school Essen

Marc Seifert graduated in Biology at the University of Cologne and did his PhD at the Institute of Cell Biology (IFZ) at the University Hospital Essen. Dr. Seiferts research group at the IFZ (Cancer Reseach) exists since 2014 and combines B cell immunology with lymphoma pathogenesis. Dr. Seiferts research aims at decoding the B cell system in healthy people and to determine the critical alterations in risk groups, such as infants and elderly or patients suffering from infections or tumors. This research unravels the B cell immune dynamics throughout life and clarifies which B cell subsets or antibody specificities are beneficial in health and limited in patients. Dr. Seiferts cancer research focusses on Chronic Lymphocytic Leukemia, Burkitt-Lymphoma and rare entities, such as Hairy Cell Leukemia and Splenic Marginal Zone Lymphoma. Dr. Seifert habilitated in 2017 at the Biological Faculty of the University of Duisburg-Essen in the subjects immunology and cancer research.

Program Name(s)

Special Grants

Project Title

Exploiting metabolic dependencies, tumor plasticity and their consequences for drug response of HCL

The Trustees of the University of Pennsylvania, Medical Center

Dr. June is the Richard W. Vague Professor in Immunotherapy in the Department of Pathology and Laboratory Medicine and is currently Director of the Center for Cellular Immunotherapies at the Perelman School of Medicine, and Director of the Parker Institute for Cancer Immunotherapy at the University of Pennsylvania. He is a graduate of the Naval Academy in Annapolis, MD and Baylor College of Medicine in Houston, TX. In 2011, his research team published findings detailing a new therapy in which patients with refractory and relapsed chronic lymphocytic leukemia were treated with genetically engineered versions of their own T cells, CAR-Ts. CTL019, the CAR T cell developed in the June laboratory was the first cell and gene therapy to be approved by the US FDA. He has published more than 500 manuscripts and is the recipient of numerous honors, including a lifetime achievement award from the Leukemia and Lymphoma Society.

Program Name(s)

Specialized Center of Research Program

Pan-heme CAR: Anti-CD38 CAR T cells for myeloid, lymphoid and plasma cell malignancies

MD Anderson Cancer Center

Dr. Carter, a Professor in the Section of Molecular Hematology and Therapy, Department of Leukemia has 20+ years of experience in molecular biology, biochemistry, and leukemia research. Her research focuses on understanding the mechanisms of drug resistance and targeting anti-apoptotic proteins and cell survival signaling pathways in myeloid leukemia. She is developing mechanism-based combinational strategies in therapy-resistant AML to overcome drug resistance and eradicate myeloid leukemia cells and leukemia stem/progenitor cells. She has published extensively in the field and several clinical trials have been developed based on her pre-clinical studies. Her recent works demonstrated the effectiveness and mechanisms of action of combined inhibition of antiapoptotic proteins Bcl-2 and Mcl-1 using BH3 mimetics (Blood Cancer Journal, 2023) and targeting HSP90 epichaperomes (Blood, 2023) in TP53 mutant AML.

Program Name(s)

Translational Research Program

Project Title

Targeting TP53-Y220C mutant AML