Who We Fund

Boston Children's Hospital

In the last 17 years, I developed gene editing tools to improve cancer immunotherapy or promote safer applications of human hematopoietic stem cell (HSC) gene therapy. I pioneered this field since when the first gene editing enzymes were shown to be potentially useful for therapeutic purposes. In 2012, I published a break-through work where we demonstrated for the first time the possibility to genetically inactivate the T cell receptor in primary T cells for improving safety/efficacy of cancer adoptive immunotherapies. This innovative approach is now widely used in immunotherapy field for generating allo-compatible T cells or to express CAR genes under the TCR promoter. In 2014, I developed the first protocol that allows targeted transgene integration in human HSC capable of long-term multilineage repopulation. My current efforts are aimed to move these advanced genetic engineering strategies towards an effective therapeutic treatment for inherited and acquired hematologic diseases.

 

Program Name(s)

Translational Research Program

Project Title

Towards clinical testing of epitope editing to enable novel adoptive immunotherapies

The Children's Hospital of Philadelphia

Sarah K Tasian, MD PhD is a pediatric oncologist and physician-scientist at the Children’s Hospital of Philadelphia (CHOP) and an Associate Professor of Pediatrics at the University of Pennsylvania School of Medicine who is interested in development of molecularly-targeted therapeutics and immunotherapies for children with high-risk leukemias. She specialises in the clinical care of children with leukemia and lymphoma, is an internationally-recognized expert in pediatric ALL and AML biology and novel therapeutics, and serves as Chief of the Hematologic Malignancies Program at CHOP, where she holds the Joshua Kahan Endowed Chair in Pediatric Leukemia Research. Her federally-funded translational laboratory research program focuses upon preclinical and early-phase clinical testing of small molecule inhibitors and chimeric antigen receptor (CAR) T cell immunotherapies in high-risk genetic subsets of childhood ALL, AML, and JMML. As an experienced clinical trialist, Dr Tasian also has several leadership roles in the Children’s Oncology Group ALL and Myeloid Diseases executive committees and is the clinical trials leader of the international Blood Cancer United PedAL consortium, working collaboratively with colleagues across the world to advance precision medicine therapeutics for children with relapsed/refractory leukemias.

Program Name(s)

Career Development Program

Project Title

Precision Medicine Inhibitor and Immunotherapy Approaches for High-Risk Childhood Leukemias

Medical College of Wisconsin

Siegfried Janz, MD, DSc, Professor and William G. Schuett, Jr., Multiple Myeloma Endowed Chair directs translational myeloma research at the Division of Hematology, Oncology & Bone Marrow Transplantation, Department of Medicine, Medical College of Wisconsin Milwaukee. After obtaining his medical degree and board certification in Clinical Immunology from Leipzig University Medical School, Germany, he received advanced training in genetic and biological pathways of myeloma development at the National Cancer Institute, NIH, Bethesda, Maryland. In 2018 he relocated his laboratory to Milwaukee, where he works in close association with his clinical colleagues to enhance our understanding of the natural history of myeloma and improve myeloma treatment and outcomes. His ongoing efforts concentrate on the design and testing of novel immunotherapies that rely on patient-derived T lymphocytes to seek out and kill myeloma.

Program Name(s)

Translational Research Program

Project Title

Improving outcomes of multiple myeloma using TGF-beta resistant BCMA-targeted CAR T cells

Northwestern University

I began my scientific career in the Marchesi laboratories (Yale, 1982-1985) and then as an undergraduate under Don Wiley (Harvard, 1986-1988). I entered the Medical Scientist Training Program (1988-1995) at the University of California, San Francisco (UCSF), performing doctoral research with Harold Varmus, at UCSF and the NIH. I defended my thesis at UCSF and completed medical training at Northwestern (1996-1997). I was an Internal Medicine house officer and Hematology/Oncology fellow at Univ of Chicago (UofC, 1997-2003)then stayed on as a UofC faculty member as a physician-scientist, with both research and clinical responsibilities (2003-2023).

Effective April 1, 2023, I have joined the Feinberg School of Medicine at Northwestern University as the inaugural director of the Jeff and Marianne Silver Family Blood Cancer Institute of the Robert H. Lurie Comprehensive Cancer Center. I will also serve as Clinical Director of Cancer Genetics.

I derive synergy between bench research and the bedside to understand disease on a molecular level. This proposal seeks to understand how inherited DNA changes influence the development of bone marrow cancers, with the goal of developing interventions that would prevent cancers in at-risk individuals to make my job as an oncologist obsolete.

Program Name(s)

Discovery

Project Title

CHEK2 as a predisposition gene for clonal hematopoiesis and hematopoietic malignancies

Dana-Farber Cancer Institute

Dr. Romee is a translational physician-scientist at Dana Farber Cancer Institute, Harvard Medical School. His long-term research goals are to translate novel aspects of immunology to improve treatments for patients with advanced cancer. He did his medical training at University of Minnesota and Washington University and was a faculty at Washington University before joining Dana Farber. He is the PI of Romee Lab for NK Cell Gene Manipulation and Therapy (https://romeelab.dana-farber.org) and the focus of his lab is gene editing of the immune cells particularly NK cells to enhance their cancer cell targeting and killing. His work helped describe memory-like NK cells which have enhanced activity against cancer cells and persist for months after their infusion into leukemia patients. He is leading efforts at Dana Farber to develop novel protocols using memory-like NK cells with other immunomodulating agents like checkpoint inhibitors in patients with advanced and otherwise incurable leukemia and solid tumors like Head and Neck Cancer, Ovarian Cancer and Kidney Cancer.

Program Name(s)

Career Development Program

Translational Research Program

Project Title

Cytokine induced memory-like NK cell immunotherapy to target post transplant relapse

Durham VA Health Care System

Dr. Friedman is a hematologist-oncologist at the Durham VA Health Care System (DVAHCS) and National TeleOncology (NTO) Program, a Professor at the Duke University School of Medicine, and is the Deputy Director of the VA National Oncology Program. She is the DVAHCS site PI for the NCI and VA Interagency Group to Accelerate Trials Enrollment (NAVIGATE) program, which facilitates enrollment of Veterans with cancer into NCI-funded clinical trials. She is the lead for the Cancer Clinical Research Service (CCRS) in NTO, which offers clinical trial navigation to Veterans with cancer and runs decentralized cancer clinical trials across the VA network.

Program Name(s)

Equity in Access

Project Title

REACH: Researching & Enhancing Access to Clinical trials in Veterans with Hematologic cancers

Washington University in St. Louis

Dr. Bern is a post-doctoral fellow in Dr. Timothy Ley’s lab at Washington University School of Medicine and a clinical fellow in the Hematology and Oncology program. He earned his bachelor’s degree in Physics from Duke University. Following undergraduate, he completed M.D./Ph.D. training at Washington University School of Medicine, where he earned his Ph.D. in Immunology, studying mechanisms regulating natural killer cell activation, in the laboratory of Dr. Wayne Yokoyama. He then joined the Physician Scientist Training Program at Washington University School of Medicine for Internal Medicine residency and Heme/Onc fellowship. His current research is focused on understanding mechanisms of chemotherapy resistance in Primary-Refractory Acute Myeloid Leukemia. His long-term goal is to identify novel therapeutic strategies for this population of patients with extremely poor outcomes and few treatment options.

Program Name(s)

Career Development Program

Project Title

Identifying Mechanisms of Chemoresistance Induced by MECOM in Primary-Refractory Acute Myeloid Leukemia

Columbia University Medical Center

I am a pediatric oncologist and health outcomes researcher at Columbia University Irving Medical Center. My research aims to identify how social determinants of health drive care and outcomes in children, adolescents, and young adults (AYA) with leukemia and lymphoma. At Columbia, I am the institutional Principal Investigator for the Dana-Farber Cancer Institute ALL Consortium and I serve on the Children’s Oncology Group Hodgkin Lymphoma (HL) Steering Committee. In these roles I participate in the design and implementation of new clinical trials, and in the development of embedded health services studies. My recent work includes a series of large-scale analyses (using clinical trials and population data) evaluating outcomes by race/ethnicity and age in ALL and HL. Increasingly, I am working to identify barriers to clinical trial participation among diverse populations, and on leveraging the clinical trial infrastructure to collect prospective data on social determinants of health.

Program Name(s)

Career Development Program

Project Title

Leveraging cancer registries, clinical trials, and community partnerships to address disparities in pediatric, adolescent, and young adult lymphoma

The University of Melbourne

Professor Jane Oliaro is an internationally recognized cancer immunologist motivated to translate scientific discoveries into the clinic. Her research experience spans the fields of T cell biology and tumor immunology, with a focus on the application of new technology to identify novel therapies to enhance immunotherapy for the treatment of cancer. She has a proven track record of undertaking successful collaborative research leading to high quality publications in influential journals including Science, Immunity, Science Immunology; successful funding and awards (including 9 CIA project grants, NHMRC fellowship, NHMRC ‘Ten of the Best’ Research Project Award and NHMRC Inaugural Achievement Award) and over 15 conference invitations in the past 5 years. As Chief Scientist for the Centre of Excellence in Cellular Immunotherapy Translation Laboratory at the Peter MacCallum Cancer Centre, she leads a preclinical program focused on the development of novel cell-based immunotherapies for translation into pilot clinical trials.

Program Name(s)

Translational Research Program

Project Title

Improving CAR-T cell therapy outcomes for patients with for aggressive lymphoma and multiple myeloma

MD Anderson Cancer Center

Dr. Meng Li is an Assistant Professor at the Department of Health Services Research at the UT MD Anderson Cancer Center and a nonresident fellow at the Leonard D. Schaeffer Center for Health Policy & Economics at the University of Southern California. Dr. Li completed her PhD in Pharmaceutical Outcomes Research and Policy at University of Washington, where co-PI, Dr. Flowers also completed his MS. Dr. Li’s research uses various data sources including population-based cancer registries, nationwide administrative claims databases, national survey data, and data from clinical trials and observational series to examine disease burden, factors that influence health care utilization, the effect of health care utilization on health outcomes, and the cost-sharing burden of prescription drugs. In particular, Her research has used Optum and SEER-Medicare data to examine disparity in initiation of immunotherapies among lung cancer and melanoma patients, the association between spending on several types of treatment and outcomes in breast cancer, and catastrophic spending among cancer patients on oral targeted anticancer medicines. Dr. Li has also developed methods to quantify novel elements of value for medical technologies in economic evaluations, which has important implications on the practice of value assessment. She meets regularly and collaborates with Drs. Shih and Flowers. Dr. Li’s research work has been published in top clinical, health economics, and health policy journals.

Program Name(s)

Equity in Access

Project Title

Investigating the Impact of Insurance Coverage on Access to Care and Outcomes among Lymphoma Patients

University of California, San Francisco

I am a postdoctoral researcher at UCSF, aiming to advance proteomic technologies for target discovery and redefine immunotherapy in blood cancers. In Dr. Arun Wiita's lab, I focus on identifying novel surface antigens in hematological malignancies like AML and multiple myeloma. By combining proteomics with immunotherapy strategies, I aim to bridge scientific discovery and clinical applications, improving outcomes for patients. My PhD in clinical proteomics from IIT-Bombay provided a foundation in biomarker discovery and translational research. I specialize in mass spectrometry-based proteomics, applying quantitative and targeted approaches to explore cancer proteomes and identify "proteoforms" as therapeutic targets. Building on promising data, my research explores surface protein post-translational modifications as a new class of immunotherapy targets, positioning me to become an independent investigator developing breakthrough therapies for blood cancers.

Program Name(s)

Career Development Program

Project Title

“Open-Surfaceomics” for Identifying Novel Surface PTMs as Immunotherapy Targets in AML

Dana-Farber Cancer Institute

Dr. Carrasco earned his MD and PhD degrees at the University of Chile. Motivated by his desire to practice medicine at the highest level, he immigrated to the USA and pursued a residency in anatomic pathology at the Massachusetts General Hospital followed by a fellowship in hematopathology at the Brigham and Women’s Hospital. In recognition to his research accomplishments in the field of multiple myeloma (MM) during his postdoctoral training he was recruited to the Dana-Farber Cancer Institute to develop an independent laboratory research program in MM. Dr. Carrasco is current Professor in Pathology at Harvard Medical School. His principal area of excellence is laboratory and clinical investigation focusing on understanding the roles of the Wnt/b-catenin signaling pathway in MM pathogenesis, with the threefold intent of (i) identifying novel therapeutic targets, (ii) developing novel targeted therapies, and (iii) developing animal models for preclinical and clinical intervention.

Program Name(s)

Translational Research Program

Project Title

Developing selective inhibitors of the b-catenin/BCL9 transcriptional complex for myeloma therapy