? Hematologic Malignancies Program The Hematologic Malignancies (HM) Program drives basic scientific discoveries and translates them into novel therapeutics for patients with myeloid and lymphoid neoplasms. Formed in 1994, the Program has two scientific aims: 1) Develop a mechanisms-based understanding of the genetic, cellular, and biochemical processes regulating malignant hematopoiesis; and 2) Translate basic scientific discoveries into more effective and less toxic therapies for hematologic malignancies. Areas of national expertise include molecular processes (epigenetic, transcriptional, translational, signaling) underlying malignant hematopoiesis; hematopoietic stem cell biology and transplantation; molecular therapeutics; and immune-based therapies. Annual average accrual to interventional clinical trials has more than doubled this funding period compared to the prior, with heavy emphasis on investigator-initiated trials. Program members are heavy users of Shared Resources (e.g. Transgenic and Chimeric Mouse, Flow Cytometry and Cell Sorting, Human Immunology, Biostatistics and Bioinformatics, and Genomic Analysis) and have wide-ranging collaborations with members of multiple other Programs. The Program is co-led by Dr. Edward Stadtmauer, an expert in clinical trials of novel therapeutics for hematologic malignancies and blood and marrow transplantation, and Dr. Nancy Speck, an internationally known basic scientist working in the area of hematopoietic stem cells and leukemia. The Program's 29 members come from seven departments in the Perelman School of Medicine. Investments by the ACC include an innovative Hematologic Malignancies Translational Center of Excellence (TCE) co-led by Dr. Stadtmauer, as well as an emerging Lymphoid Malignancy TCE co-led by Drs. Stephen Schuster and Megan Lim. These and other investments have galvanized an already successful Program by expanding resources for laboratory, tissue banking, and clinical research and providing pilot grant funding. Scientific accomplishments this funding period include studies garnering multiple FDA approvals (CAR T cell therapy for adults with relapsed and refractory B cell non-Hodgkin's lymphoma; gilteritinib for refractory acute myeloid leukemia with FLT3 mutations; and selinexor, a first-in-class oral nuclear transport inhibitor for `penta-refractory' multiple myeloma). Basic discoveries revealed novel tumor-cell intrinsic pathways such as the demonstration that kelch-like 6 (KLHL6) functions as an E3 ubiquitin ligase, and KLHL6 mutations in lymphoma increase NF-?B signaling. Weekly seminars, collaborative grants, weekly clinical working group meetings, and an annual research retreat facilitate member interactions. Currently, members have research funding totaling $16.2M (direct), of which $5.8M is peer-reviewed and $1.9M is from the NCI. There are 20 R01-equivalents held by our members. During the project period, members published 450 cancer relevant publications, of which 32% were intra- Programmatic, 25% were inter-Programmatic and 73% were multi-institutional. The Program enrolled 1,567 subjects onto interventional trials this project period, and 2,162 subjects onto non-interventional trials.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Center Core Grants (P30)
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Subcommittee I - Transistion to Independence (NCI)
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University of Pennsylvania
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Shroff, Rachna T; Hendifar, Andrew; McWilliams, Robert R et al. (2018) Rucaparib Monotherapy in Patients With Pancreatic Cancer and a Known Deleterious BRCA Mutation. JCO Precis Oncol 2018:
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