Molecular Therapeutics Research Program

摘要：分子治疗计划 分子治疗(MT)计划致力于评估癌症靶点，开发新的 疗法，并通过高度集成的基础科学和翻译设计更有效的递送系统 活动。该项目由综合化学生物学中心主任斯蒂芬·弗莱领导 药物发现(CICBDD)和弗雷德·埃谢尔曼杰出教授，加里·约翰逊，凯南 药理学系特聘教授。MT计划由42名成员组成，他们 与四所学校和16个系有联系。在上一个资助期内，计划成员有 发表了675篇与癌症相关的文章。MT具有高度的协作性。这些论文中有16%是方案内论文 33%是跨计划的(43%是协作性的)。2019年，我们的计划成员共获得了 2090万美元(直接费用)与癌症相关的外部资金，包括来自NCI的640万美元(直接费用) 1300万美元的其他同行资金。MT项目由在五个广泛领域拥有专业知识的调查人员组成： 化学和结构生物学；药物发现和开发；药物输送和纳米技术； 系统药理学；和致癌信号。该项目中的许多调查人员都很活跃 与其他LCCC项目合作，利用LCCC的共享资源并为其提供指导。这些 相互作用使发现和发展有希望的生物的许多科学步骤成为可能 治疗。2015年，MT被评为“杰出的”，指出“这个项目的小弱点是，相对 在LCCC，前景看好的疗法很少进入研究人员发起的临床试验。“既然是这样 回顾，北卡罗来纳大学和其他机构已经根据MT的发现启动了多项临床研究， 正如我们在对先前批评的回应中所概述的那样。未来计划建立在可用的独特翻译资源的基础上 由于埃谢尔曼创新研究所和顶峰山的成立，带来了超过100美元的收入 100万美元，用于将以北卡罗来纳大学为基础的发现转化为患者。细胞和生物疗法将是 临床免疫治疗计划的GMP设施的扩展使MT成员的未来重点。 通过这些努力，MT将继续加快发现新的癌症疗法，并通过 临床研究和乳腺癌项目，设计和执行我们的翻译和治疗试验 病人。

ABSTRACT: MOLECULAR THERAPEUTICS PROGRAM The Molecular Therapeutics (MT) program is dedicated to evaluating cancer targets, developing novel therapies, and devising more effective delivery systems via highly integrated basic science and translational activities. The Program is led by Stephen Frye, Director of the Center for Integrative Chemical Biology and Drug Discovery (CICBDD) and Fred Eshelman Distinguished Professor, and Gary Johnson, Kenan Distinguished Professor in the Department of Pharmacology. The MT Program consists of 42 members who are associated with four schools and 16 departments. During the last funding period, program members have published 675 cancer-related articles. MT is highly collaborative. 16% of these papers are intra-programmatic and 33% are inter-programmatic (43% collaborative). In 2019, our program members held grants totaling $20.9M (direct cost) in cancer-relevant extramural funding, including $6.4M (direct costs) from the NCI and $13.0M other peer funding. The MT program is comprised of investigators with expertise in five broad areas: Chemical and Structural Biology; Drug Discovery and Development; Drug Delivery and Nanotechnology; Systems Pharmacology; and Oncogenic Signaling. Many investigators in the program have active collaborations with other LCCC programs, using and providing direction for LCCC's shared resources. These interactions enable many of the scientific steps needed for the discovery and development of promising therapies. In 2015, MT was rated “outstanding” stating “the minor weakness of this program is that, relatively few of the promising therapeutics have moved into investigator initiated clinical trials at the LCCC.” Since this review, multiple clinical studies have been initiated at UNC and other institutions based on discoveries in MT, as outlined in our response to the prior critique. Future plans build on unique translational resources available due to the creation of the Eshelman Institute for Innovation and Pinnacle Hill, which bring more than $100 million to progress UNC-based discoveries into patients. Cellular and biologic therapies will be an area of future focus for MT members enabled by the expansion of the Clinical Immunotherapy Program's GMP facility. Through these efforts, MT will continue to accelerate discovery of new cancer therapeutics and, with the Clinical Research and Breast Cancer Programs, design and execute translational and therapeutic trials in our patients.