Who We Fund

The University of Texas MD Anderson Cancer Center

Nitin Jain, MD, is an Associate Professor in the Department of Leukemia at MD Anderson Cancer Center in Houston. He earned his medical degree from the All India Institute of Medical Sciences, New Delhi. He completed Internal Medicine residency at the Medical College of Wisconsin and fellowship in Hematology/Oncology at the University of Chicago. Dr. Jain research interests include new drug development for patients with chronic lymphocytic leukemia (CLL) and acute lymphoblastic leukemia (ALL). Dr. Jain is Principal Investigator of several investigator-initiated phase I-II clinical trials, including combination targeted therapies (ibrutinib and venetoclax) in CLL, checkpoint inhibitors in Richter transformation, novel CD22 antibody drug conjugate in B-ALL, venetoclax + chemotherapy in B- and T-ALL, and off-the-self allogeneic CAR-T in B-ALL. The trial combining ibrutinib and venetoclax was published in New England Journal of Medicine in 2019. He has won many awards including ASCO merit award, Sabin Family Foundation Award in 2018 and MD Anderson Faculty Scholar award in 2020.

Program Name(s)

Career Development Program

Project Title

Combination Targeted Therapy in Chronic Lymphocytic Leukemia

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

The University of New South Wales

Dr. Lock is investigating mechanisms of glucocorticoid resistance in pediatric acute lymphoblastic leukemia (ALL) and tests potential new treatments for fast-tracking into early phase clinical trials.

Dr. Lock is Professor of Medicine at UNSW Sydney, Head of the Leukaemia Biology Group and the Blood Cancers Theme at Children’s Cancer Institute and an international leader in preclinical drug testing in acute leukemia. His work has led to the identification of novel mechanisms of glucocorticoid resistance in pediatric ALL and to the prioritization of several new agents into clinical trials aimed at improving the treatment of acute leukemia. Dr. Lock has been a Principal Investigator in the NCI-funded pediatric preclinical testing program since 2005 and carries out all of the leukemia testing for the program. He also provides a leadership role in the Australian national precision medicine clinical trial (PRISM) to utilize preclinical drug testing to assist in patient treatment decisions.

Program Name(s)

Translational Research Program

Project Title

Therapeutic targeting of T-cell acute lymphoblastic leukemia using an AKR1C3-activated prodrug

TAP Partner

BioInvent International AB is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently four drug candidates in five ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors, respectively. The Company's validated, proprietary F.I.R.S.T™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new drug candidates to fuel the Company's own clinical development pipeline and providing licensing and partnering opportunities.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 1/2 study of BI-1206, a monoclonal antibody to CD32b (FcyRIIB), in combination with rituximab in patients with indolent NHL that has relapsed or is refractory to rituximab

A phase 2 study of BI-1808, a monoclonal antibody to TNFR2, as a single agent and in combination with pembrolizumab in patients with solid tumors and CTCL

Stanford University

Dr. Jaiswal is an assistant professor at Stanford University in the Department of Pathology and Institute for Stem Cell Biology and Regenerative Medicine, where his lab focuses on understanding the biology of the aging blood system. Several years ago, he identified a common, pre-malignant state for blood cancers, termed "clonal hematopoiesis”, by analyzing large human genome sequencing studies. He discovered that clonal hematopoiesis is prevalent in the aging population and increases the risk of not only blood cancers, but also cardiovascular disease and overall mortality. Understanding the biology of these mutations and how they contribute to the development of cancer and other age-related diseases is the current focus of work in the lab. Ultimately, Dr. Jaiswal and his team aim to develop novel therapies that can prevent adverse outcomes due to clonal hematopoiesis.

Program Name(s)

Discovery

Project Title

Uncovering the role of TCL1A as a driver of clonal hematopoiesis and hematological malignancies

Memorial Sloan Kettering Cancer Center

I am an MD/PhD physician scientist in the oncology fellowship at MSKCC where I treat leukemia and perform research to understand molecular underpinnings of the disease. My prior work at Brandeis University, Cold Spring Harbor Lab, Harvard Medical School, and Albert Einstein College of Medicine was focused on basic mechanisms of gene expression regulation and I am now studying RNA dysregulation in leukemia. During my PhD, I developed novel methods to study RNA binding proteins in collaboration with Nobel Laureate Dr. Michael Rosbash and Rosenstiel Prize winner Dr. Robert H Singer (my PhD mentor). This integrative approach to studying RNA is ideal for understanding understudied proteins that regulate splicing factor mutations in the Abdel-Wahab lab. For my career, I aim to use novel approaches to understand how the most prevalent splicing factor mutations in blood cancers drive progression and transformation.

Program Name(s)

Career Development Program

Project Title

Targeting SF3B1 splicing factor mutant myeloid malignancies through dependency on GPATCH8

Johns Hopkins University

As a physician-scientist in the Division of Hematologic Malignancies at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Dr. Dalton has a clinical specialty in myeloid leukemias and a research laboratory that focuses on the study of DNA mutations that drive those leukemias. In particular, he is working to better understand DNA mutations in the spliceosome, which occur in many patients with MDS and AML and are currently difficult to treat. He uses bone marrow and blood samples generously donated by patients, together with cell ‘models’ that he genetically engineers in the lab, to understand what these DNA mutations do and how they might be targeted with new treatments. His work has led to identification of vulnerabilities in cells containing these mutations that he aims to translate into novel therapeutic approaches in MDS and AML.

Program Name(s)

Translational Research Program

Project Title

Therapeutic modulation of serine availability for SF3B1-mutant myeloid malignancies

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

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

Columbia University Medical Center

I’m a Research Scientist at Columbia University Multiple Myeloma Program. I have over 20 years of research experience, especially with profound expertise in pharmaceutical biochemistry and drug development in multiple myeloma. I received my Ph.D. degree in Pharmacology and Chemistry of Peptides in 2005. After that, I did my postdoctoral training at the University of Pittsburgh Cancer Institute and have focused on multiple myeloma drug development since then. My current research aims to identify the druggable targets and develop therapeutic approaches for relapsed/refractory multiple myeloma-carrying RAS mutation. My work has been awarded by the International Myeloma Foundation Brian D. Novis Research Grant, the International Myeloma Society, and the Paula and Rodger Riney Foundation Translational Research Award. My research has also resulted in over 18 peer-reviewed articles in top journals such as JCI, Blood, and Leukemia.

Program Name(s)

Translational Research Program

Project Title

Targeting GCK as a novel and selective therapeutic strategy against RAS mutated Multiple Myeloma

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

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