Abramson Cancer Center BMT/CTN Core Clinical Center

项目摘要：宾夕法尼亚大学艾布拉姆森分校的血液和骨髓移植计划（BMTP） 癌症中心（ACC）是美国最大和最古老的癌症中心之一。该活动计划看到 所有年龄，性别和种族的成年人，并进行各种形式的造血细胞 移植（HCT），包括自体、同种异体（清髓性和降低强度） 和脐带血，匹配的和不匹配的，包括单倍相同的，相关的和不相关的，和 细胞疗法，包括供体淋巴细胞输注， 修饰的自体和同种异体T细胞疗法，如嵌合抗原受体（CAR） 和T细胞受体试验来治疗各种疾病。Edward Stadtmauer博士是 这个核心中心自15年前成立以来，也是首席， 血液肿瘤（HM）部门和ACC HM研究项目的共同负责人， 因此，BMT CTN临床试验的患者是直接的，这已经反映在 我们中心的强劲增长大卫波特博士与斯塔特莫尔博士密切合作， 20年来一直从事细胞免疫治疗。378名患者来自宾夕法尼亚大学 BMT CTN;在超过140个参与中心中排名第五。佩恩已经证明了 在网络中担任领导职务，是9个礼宾小组的成员（4个小组的研究主席）， 3个行政和技术委员会。宾夕法尼亚大学的调查人员是成功的关键， 骨髓瘤系列临床试验。ACC BMTP始终非常活跃，有799例HCT 在2013-2015年进行; 536例自体，263例同种异体。BMTP得到了众多 世界一流的ACC研究资源，包括我们的ACC本身，被评为“卓越”的NCI CCC在2015年，癌症免疫治疗中心由我们的先驱细胞免疫生物学家领导 博士Carl June和PENN-CHOP血液中心专注于非恶性血液疾病 由Charles Abrams管理，他是著名的血液学家，现任ASH主席。我们的研究 建议，“自体HCT随后抗- BCMA +/-anti-CD 19 CAR自体T细胞治疗高危型Mycobacteria。选择 在宾夕法尼亚大学的许多替代方案中，以展示我们的专业知识，基于我们自己的 试点研究工作，填补了一个主要的临床需求（改善高风险患者的结果 骨髓瘤），并可以及时完成。这些强大的临床研究的属性， 患者护理，该领域的思想领袖以及对BMT的热情和成功的记录 CTN试验使Penn成为领导新型细胞疗法开发和评估的独特位置 并迅速传播结果以使需要HCT治疗的患者受益。

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.