Du Wei
Leukemia
Du Wei, PhD, MD
Pittsburg, PA
United States
University of Pittsburgh
My research is centered on pathophysiology of hematologic diseases such as bone marrow failure and leukemia, with specific focus on hematopoietic stem cell (HSC), DNA damage and immune response, and tumor microenvironment. I have been investigating HSC-BM niche interaction and HSC regeneration under conditions of injury and aging; and have identified functional interactions between certain factors implicated in cell proliferation, polarity, adhesion/migration, stem cell metabolism and aging. My work has led to 53 peer-reviewed scientific papers in high-impact scientific journals, including Blood, JCI, Nat Comm and Leukemia. I laid the groundwork for my long-term research by developing cell and animal models as well as effective assays for identifying mechanisms underlying critical hematological diseases as PI on several NIH- and private-funded grants. I am confident that both myself as a PI and my innovative research are highly competitive in future success in grants and publications.
Program Name(s)
Career Development Program
Project Title
David Avigan, MD
Boston, MA
United States
Beth Israel Deaconess Medical Center
Dr. Avigan is the Director of Beth Israel Deaconess Cancer Center; Chief of the Division of Hematology/Hematologic Malignancies, and Professor of Medicine at Harvard Medical School. He leads a large program for translational and clinical investigation in cellular immunotherapy for blood cancers. He has developed a personalized tumor vaccine and conducted a series of clinical trials to define safety, immunologic response and clinical efficacy of vaccination across the country. He has established pre-clinical models studying the immune microenvironment in patients undergoing CAR T and bispecific T cell engager therapy. We have developed transcriptomic platforms to identify biomarkers predictive of response to CAR T cell demonstrating the critical contribution of secondary activation of adaptive immune mechanisms. We led a consortium to study the integration of vaccine and CAR T cell therapy to promote durable CAR T cell persistence and disease regression.
Program Name(s)
Discovery
Project Title
Dimericon
Blood cancers
Dimericon, LLC
Zurich,
Switzerland
TAP Partner
Dimericon is a private biotech company focused on exploring crosslinked helix dimers (Dimericons) as therapeutics and templates for small molecule development. Dimericon’s technology targets hard-to-drug intracellular protein-protein interactions using rationally designed mimetics of helix dimers. The Seed round of financing will support preclinical studies to further develop the current cFLIP inhibitor lead compound, DMRX1004, to be an IND ready clinical candidate in hematological malignancies.
Program Name(s)
Therapy Acceleration Program
Project Title
Supporting development of dimericons (crosslinked helix dimers) for blood cancers
Elliot Stieglitz
CMML
Elliot Stieglitz, MD
San Francisco, CA
United States
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
Jing Yang
Myeloma
Jing Yang, PhD
Houston, TX
United States
Houston Methodist Research Institute
My research focuses on the translation of bench works into new therapeutic approaches/strategies for multiple myeloma and other blood cancers. One of my research goals is to study the mechanism underlying the pathogenesis of myeloma and its associated tumor microenvironment. I have been investigating the interaction of myeloma cells with bone marrow adipocytes, bone cells, and the signaling pathways associated with obesity, inflammation, chemo-, immune-therapeutic resistance, and bone disease, which are the major hurdles in treating myeloma. As a PI, I have managed many projects funded by extramural grants, private funds, and industry alliances. Some highlights include the NIH/NCI R01s and funding received from ASH and ACS. I have an excellent publication track record with peer-review articles that are in top-tier journals with high impact factors (e.g. Cell Metabolism, Science Translational Medicine, Cancer Cell). My research experience shows my capacity to be a PI for this Project.
Program Name(s)
Translational Research Program
Project Title
Targeting acetyl-CoA synthetase 2 to remodel obesity-evoked inflammatory microenvironment in myeloma
Martin Carroll, MD
Philadelphia, PA
United States
Perelman School of Medicine at the University of Pennsylvania
Dr. Carroll is a physician scientist who has been studying leukemia biology for 3 decades. Until recently Dr. Carroll saw patients with blood cancers at the Philadelphia Veterans Administration Hospital but is now focused solely on research to improve therapy for AML. He has performed that research at the Univeristy of Pennsylvania since 1998. Dr. Carroll has had a long term commitment to building tools to enhance the understanding of human AML. These tools have included development of a large tissue bank of patient samples. He has also lead the field in development and application of the study of AML in immunocompromised mice. His work in xenograft AML models lead to the characterization of chemotherapy resistance as not always being dependent on leukemic stem cells but on metabolic adaptations of the cells to chemotherapy. Dr. Carroll has been involved in development of previous novel therapies for AML and continues to focus on developing safer and more effective treatments.
Program Name(s)
Specialized Center of Research Program
Project Title
Qian Zhang, PhD
New York, NY
United States
Memorial Sloan Kettering Cancer Research
Qian Zhang is a postdoctoral fellow in the Dr. Abdel-Wahab’s lab at Memorial Sloan Kettering Cancer Center. She developed synthetic introns to selectively targeting U2AF1 mutated myeloid cells. U2AF1 mutations characterize high risk forms of MDS and are also common in elderly patients with AML. She aims to optimize these synthetic introns and develop novel lipid nanoparticles delivery of the synthetic introns into mice. Qian received her Ph.D. training in the Dr. Krainer lab at Cold spring harbor laboratory, where she developed novel RNA therapeutics to target a driver mutation of a deadly pediatric brain tumor known as Diffuse Intrinsic Pontine Gliomas. This work was published in Science Translational Medicine with Qian as a first author. She also contributed to the development of innovative RNA therapeutics to modulate RNA splicing in cystic fibrosis. Qian is a key coauthor for these studies that were published in papers in Nucleic Acids Research, PNAS, and Nature Communications.
Program Name(s)
Career Development Program
Project Title
Synthetic introns to target U2AF1 mutant leukemias and dissect molecular basis for mis-splicing
Stephen Gottschalk
pediatric leukemia and CAR-T
Stephen Gottschalk, MD
Memphis, TN
United States
St. Jude Children's Research Hospital
I am a pediatric oncologist, who is interested in using the patient’s immune system to fight cancer. I grew up in Germany and moved to the US in 1992 after obtaining my medical degree. After completing my training in pediatric and oncology, I became faculty at the Cancer Center of Texas Children’s Hospital and the Center for Cell and Gene Therapy at Baylor College of Medicine. In the laboratory I focused on developing immune cell therapies for cancer patients, which I translated with my research team into the clinic. In addition, I managed pediatric cancer patients, who underwent bone marrow transplantation, and participated in numerous clinical studies. In 2017 I became Chair of the Department of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children’s Research Hospital. The department focuses on evaluating novel transplant and cell therapy approaches using genetic engineering approaches to augment the ability of the immune system to fight cancer.
Program Name(s)
Translational Research Program
Project Title
CD70-directed CAR T-cell therapy for the treatment of relapsed/refractory pediatric AML
Manyi Wei
acute megakaryoblastic leukemia
Manyi Wei, PhD
New Haven, CT
United States
Yale
Dr. Manyi Wei gained his B.S. degree at China Pharmaceutical University in Nanjing. Subsequently, he completed his Ph.D. training in Cell Biology at the Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences. During his Ph.D. research, Dr. Wei focused on the regulatory role of long non-coding RNA in gene expression and its impact on neuronal function. Dr. Wei has always nurtured a particular interest in understanding and developing novel treatment for leukemia. After his Ph.D. training, Dr. Wei joined the laboratory of Dr. Stephanie Halene at Yale Cancer Center and Yale University School of Medicine. He immediately began exciting work on RNA modifications and their functions in the initiation and progression of acute myeloid leukemia and myelodysplastic syndromes and importantly on novel therapeutic approaches exploiting cell intrinsic and extrinsic effects of targeted inhibitors of RNA methylation. His goal is to contribute to the cure of leukemia.
Program Name(s)
Career Development Program
Project Title
Britta Will
Leukemia and pre-leukemia
Britta Will, PhD
Bronx, NY
United States
Albert Einstein College of Medicine
Dr. Will has been a group leader at the Albert Einstein College of Medicine since 2016. She obtained her Ph.D. in Cell Biology from the University of Freiburg, Germany and underwent further training in hematology/oncology with Dr. Ulrich Steidl. With a passion for and through the lens of stem cell biology, Britta’s laboratory seeks to discover novel therapeutic options for patients with myeloid malignancies. Current research concentrates on two largely uncharted territories in blood stem cell aging and leukemic stem cell maintenance - iron homeostasis and highly selective autophagy. Dr. Will also serves as co-leader of the Stem Cell and Cancer Biology Program and directs the Cancer Stem Cell Pharmacodynamics Unit at the NCI-designated Montefiore-Einstein Cancer Center. She is the recipient of prestigious young investigator awards, including from the Pershing Square Sohn Cancer Research Alliance, Gilead, AAMDSIF, Feldstein Medical Foundation, and the Leukemia Research Foundation.
Program Name(s)
Career Development Program
Project Title
Therapeutically actionable molecular safeguards in leukemic stem cells
Lorenzo Falchi, MD
New York, NY
United States
Memorial Sloan Kettering Cancer Center
Dr. Falchi graduated Cum Laude from the University of Perugia, Italy. He completed a postdoctoral fellowship at MD Anderson Cancer Center, residency at Yale-New Haven Hospital, and Hematology/Oncology fellowship at Columbia University Medical Center. He is currently an Assistant Attending in the Lymphoma and Cellular Therapy Services at Memorial Sloan Kettering Cancer Center.
Dr. Falchi’s research focuses on the use of immunotherapy to treat B-cell NHL. He leads multiple such trials nationally and internationally. He authored or co-authored over 75 articles published in peer-reviewed journals, numerous abstracts, and several book chapters.
Dr. Falchi is Co-Editor-In-Chief of Oncoimmunology, Editorial Board Member for Blood Advances, and recurrent peer-reviewer for high-profile journals. He is recipient of several awards, including a Lymphoma Research Foundation Career Development Award. He is an active member of several scientific societies, including the American Society of Hematology.
Program Name(s)
Career Development Program
Project Title
Stephen Nimer
myeloid cancer biology
Stephen Nimer, MD
Coral Gables, FL
United States
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