Who We Fund

The University of Texas at Austin

William Matsui is a Professor of Oncology, Director of the Hematologic Malignancy Program, Associate Chair of Research, and the Deputy Director of the LiveSTRONG Cancer Institutes at the Dell Medical School and the University of Texas at Austin. He also serves as the interim Vice Dean of Research for Dell Med. Dr. Matsui's research has focused on understanding the intersection between cancer, stem cell, and developmental biology. His laboratory first identified unique populations of cancer cells with stem cell properties in multiple myeloma and found that several pathways regulating normal stem cells, including those involved in embryonic development, are abnormally activated in cancer stem cells. Importantly, these basic research studies have simultaneously been translationally relevant and served as the basis for over a dozen novel clinical trials.

Program Name(s)

Translational Research Program

Project Title

Stem cell features and Notch signaling in p53 deleted multiple myeloma

Indiana Universty

My clinical and research interest is to develop therapies that enhance the immune system to fight blood cancer. I serve as the IND holder responsible for the clinical development for the CD4CAR , and the CAR T cells are manufactured under this IND and the GMP conditions at Indiana University. This work is done in collaboration with IcellGene therapeutics. It is designed to treat T cell leukemia and lymphoma. We have treated three patients so far and results were very promising. CD4, the target for this CAR is expressed on CMML and we are not only planning to try this CAR in CMML, but we will be studying mechanisms of response and resistance to this treatment as we go, and also improving on the structure of this CAR to be more effective with the goal of curing CMML.

Program Name(s)

CMML Initiative

Project Title

Development of cellular therapy for CMML and the Immune landscape of response and resistance

Rutgers University

A PharmD by training, I then obtained my PhD guided by Dr. Manuel Serrano at the CNIO studying Sirt1 role in metabolism and cancer, which led to 4 first-author and 1 corresponding papers, plus 11 co authorships. Next, I joined the Ferrando Lab at Columbia University where I published two seminal papers identifying a NOTCH1-driven Myc enhancer critical for T-cell development and leukemia, and dissecting the role of cancer metabolism in the resistance to anti-NOTCH1 therapy. I started my independent career at Rutgers in July 2017. In these 4 years, I have established a highly successful and productive laboratory, as reflected by the multiple funding sources obtained (including R01, ACS or AACR grants, among others), as well as by the publication of 3 corresponding author studies: a Blood Cancer Discovery paper identifying an enhancer of PTEN in leukemia; a Leukemia paper describing the antileukemic effects of SHMT inhibition; and a Blood paper describing the therapeutic effects of mitochondrial uncoupling in T-ALL.

Program Name(s)

Career Development Program

Project Title

Therapeutic exploitation of novel mouse models and metabolic interventions in leukemia

Dana-Farber Cancer Institute

Program Name(s)

Career Development Program

Project Title

Mechanisms of oncogenic transcription in NPM1-mutant myeloid leukemia

Weill Cornell Medicine

I completed my Ph.D. in pharmacology at Zhejiang University, China, in June 2020, specializing in cancer immunology. My doctoral research provided in-depth training in mouse models, particularly the construction of humanized immune recombinants such as PBMC and CD34-engrafted xenografts for pharmacodynamic studies. I developed skills in in vitro drug screening, immune cell sorting, and animal models, solidifying my expertise in experimental design. Currently, I am conducting research in Dr. Wendy Béguelin's lab, focusing on the role of the tumor microenvironment in B lymphoma progression. I pioneered a multiphoton microscopy approach for real-time imaging of cellular interactions within lymphoid tissues, enabling a detailed view of cellular interactions in lymphoma. Supported by experts from the Béguelin, Chris Xu and Melnick groups, our team leverages advanced murine lymphoma models and deep-tissue imaging techniques.

Program Name(s)

Career Development Program

Perelman School of Medicine at the University of Pennsylvania

Dr. Liling Wan is an Assistant Professor at the University of Pennsylvania. She received a B.S. in Biological Sciences and Biotechnology from Tsinghua University and a Ph.D. in Molecular Biology from Princeton University. She conducted postdoctoral research at Rockefeller University where she studied chromatin regulators in cancer. The Wan lab studies basic gene regulatory mechanisms and how these mechanisms are dysregulated in cancer, with the goal of harnessing these insights for therapeutics. Her research has revealed how chromatin “reader” proteins impact gene regulation in cancer such as acute myeloid leukemia and led to early drug development efforts targeting these mechanisms. Dr. Wan has been recognized for her innovative and impactful research through numerous awards including AACR NextGen Star, NIH Pathway to Independence Award, the NIH Director’s New Innovator Award, and was recently named a Pew-Stewart Scholar, V Foundation Scholar, and ASH Scholar.

Program Name(s)

Career Development Program

Project Title

Epigenetic Mechanisms in Acute Myeloid Leukemia

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

Perelman School of Medicine at the University of Pennsylvania

Dr. Monika Mittal is a dedicated researcher specializing in cancer biology, with a focus on acute myeloid leukemia (AML). With a strong background in molecular biology and biochemistry, Dr. Mittal aims to uncover the underlying mechanisms of blood cancers and develop innovative therapeutic strategies. Currently, Dr. Mittal is investigating the role of DCAF15, a protein that regulates cellular processes, to understand its impact on AML progression and treatment. By employing advanced techniques such as gene editing and targeted protein degradation, Dr. Mittal aims to create new therapeutic strategies that can improve patient outcomes. Passionate about translating scientific discoveries into clinical applications, Dr. Mittal is committed to improving outcomes for those affected by blood cancers, driving research forward to find more effective treatments for AML. Through collaboration and innovation, Dr. Mittal hopes to make a meaningful difference in the fight against leukemia.

Program Name(s)

Career Development Program

Project Title

Leveraging the ubiquitin proteasome system for targeted therapy in Acute Myeloid Leukemia

University of Perugia. Department of Medicine and Surgery

After graduation in Medicine and residency in Hematology at the University of Perugia (Italy) under the supervision of Prof. M. F. Martelli and Prof. B. Falini, Enrico Tiacci trained in the molecular pathogenesis of lymphomas at the University of Essen (Germany) in the laboratory of Prof. R. Küppers. He then established and independent translational and clinical research program in leukemias and lymphomas at the University and Hospital of Perugia, where he is Associate Professor of Hematology. He published as author and acted as reviewer in journals like the New England Journal of Medicine, Blood, Journal of Clinical Oncology, Lancet Oncology, Journal of Clinical Investigations and Blood Cancer Discovery.

Program Name(s)

Special Grants

Project Title

BRAF inhibition as an alternative to chemotherapy in the treatment strategy of hairy cell leukemia

Dana-Farber Cancer Institute

Dr. Julia Paczkowska completed her master’s degree in pharmacy at Poznan University of Medical Science, Poland. Thereafter, she obtained her PhD in medical sciences from the Institute of Human Genetics, Polish Academy of Sciences, Poznan, with a thesis focusing on deregulated transcription factors and microRNAs in the pathogenesis of classic Hodgkin lymphoma. After obtaining her degree, she began her post-doctoral studies in the laboratory of Dr. Margaret Shipp at Dana-Farber Cancer Institute in Boston. Dr. Paczkowska’s postdoctoral research focuses on the immunobiology of classic Hodgkin lymphoma and related B-cell malignancies.

Program Name(s)

Career Development Program

Project Title

Characterization and Targeting of Tumor-Associated Monocytes/ Macrophages that Limit the Efficacy of PD-1 Blockade in Lymphoma

Washington University School of Medicine in St. Louis

Dr. Magee directs the pediatric leukemia and lymphoma program at Washington University School of Medicine and St. Louis Children’s Hospital. He received his M.D. and Ph.D. from Washington University and then completed a pediatrics residency and hematology/oncology fellowship at the University of Michigan. He conducted postdoctoral research with Dr. Sean Morrison (Howard Hughes Medical Institute) at the University of Michigan and UT-Southwestern. Dr. Magee’s work focuses on causes and treatments for childhood acute myeloid leukemia. He has published several papers in high impact journals investigating interactions between genes that regulate normal childhood blood development and mutations that cause leukemia, with the goal of understanding why childhood leukemias respond differently to treatment than adult leukemias. He is also investigating changes in blood forming stem cells that lead to leukemia when children receive chemotherapy for other tumors, such as lymphomas or solid tumors.

Program Name(s)

Career Development Program

Project Title

Neonatal origins of pediatric AML

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