Abramson Cancer Center BMT/CTN Core Clinical Center

项目概述：宾大艾布拉姆森大学血液和骨髓移植项目（BMTP）

Project Summary: The Blood and Marrow Transplant Program (BMTP) of the Penn Abramson Cancer Center (ACC) is among the largest and oldest in the nation. This active program sees adults of all ages, sexes and ethnic origins and conducts all forms of hematopoietic cell transplantation (HCT) including autologous, allogeneic (myeloablative and reduced intensity) and cord blood, matched and mismatched including haplo-identical, related and unrelated, and cellular therapies including donor lymphocyte infusions and has been a pioneer in gene modified autologous and allogeneic T cell therapies such as chimeric antigen receptor (CAR) and T cell receptor trials for a myriad of diseases. Dr. Edward Stadtmauer has been the PI of this Core Center since the founding of the Network 15 years ago and is also the Chief, Hematologic Malignancies (HM) Section and Co-Leader of the ACC HM Research Program and so access to patients for BMT CTN clinical trials is straightforward and this has been reflected in the strong accrual from our center. Dr. David Porter works very closely with Dr Stadtmauer and has directed Cellular Immunotherapy for 20 years. 378 patients have been accrued from Penn to BMT CTN; 5th largest of >140 centers participating. Penn has demonstrated intellectual leadership in the Network with membership on 9 protocol teams (study chairs for 4) and chair of 3 administrative and technical committees. Penn investigators were key to the highly successful myeloma series of clinical trials. ACC BMTP remains consistently very active with 799 HCTs conducted in 2013-2015; 536 autologous, 263 allogeneic. The BMTP is supported by numerous world-class ACC research resources including our ACC itself, ranked `Exceptional' as a NCI CCC in 2015, the Center for Cancer Immunotherapy led by our pioneer cellular immunobiologist Dr. Carl June, and the PENN-CHOP Blood Center focused on non-malignant blood disorders run by Charles Abrams a renowned hematologist and current President ASH. Our research proposal, “A RANDOMIZED PHASE II STUDY OF AUTOLOGOUS HCT FOLLOWED BY anti- BCMA +/- anti-CD19 CAR AUTOLOGOUS T CELLS FOR HIGH-RISK MYELONA.” was chosen among many alternatives from Penn to demonstrate an area of our expertise, based on our own pilot study work, fill a major clinical need (improvement of outcome for patients with high-risk myeloma) and can be completed in a timely fashion. These attributes of strong clinical research, patient care, thought leaders in the field and a documented enthusiasm for and success in BMT CTN trials uniquely position Penn to lead development and evaluation of novel cell therapies and rapidly disseminate results to benefit patients in need of HCT therapy.