Pediatric and Adult Translational Cancer Drug Discovery and Development Training Program (PACT-D3)

摘要 这一培训计划对我们机构来说是独一无二的，培养了下一代基础和临床研究人员 世卫组织将重点放在癌症的战略性和综合性药物发现和开发上。本节目 利用强大的基础设施和机构承诺为研究生实习生提供培训 从事儿科和成人药物发现和开发工作。增加了一个儿科癌症病灶 解决未得到满足的需求以及NCI科学委员会确定的NCI的主要重点 顾问和由此产生的儿童癌症数据(CCDI)倡议。丰富的成人经验和儿科经验 研究人员在这款T32中独一无二地被用来开发儿科药物发现的新方法。那里 目前没有侧重于儿童和成人癌症药物发现和开发的NCI T32 训练。癌症仍然是儿童疾病相关死亡的主要原因。对于许多孩子来说， 他们的恶性实体瘤复发的经验是很少的根治治疗选择。儿科的努力 将与多机构的“发育和高度活跃的RAS肿瘤(DHART)”相吻合 多PI Wade Clapp博士专注于神经纤维瘤病1型(NF1)--人类最常见的遗传癌 易感综合症。在这种孢子中的发现已经导致了对儿科患者的新的临床试验，这是 预计将需要开发超出孢子范围的更多新药。此外, NIH/NICHD资助的P50，“儿科发育药理学研究的专门中心”是 利用精确基因组学策略和体内建模设计安全有效的联合疗法 对于复发的实体瘤，如骨肉瘤和其他潜在药物开发的新靶点是 被指认了。这突出了我们识别新药靶点和开发新药的重点和机会 专为儿科病人而设。该计划将得到来自其他计划的协作努力的支持，网址为 伊利诺伊大学医学院(IUSM)包括NCI指定的伊利诺伊州西蒙综合癌症中心 (IUSCCC)；嵌入IUSCCC的抗癌药物研发(CDDD)中心 实验和发展治疗(EDT)计划；加速治疗的目标参与 阿尔茨海默病(Treat-AD)中心的发展和印第安纳州CTSI临床前创新 “坦克”计划。我们将重点将研究生作为实习生，他们将从两个集中的 监督研究生招生并指导学员选择导师和家庭的计划 各部门。这两个项目是印第安纳州生物医学门户(IBMG)项目，该项目招生约36人 每年研究生人数和每年招收约10名学生的MSTP。18位导师密切参与了 这一计划和学校的药物开发努力。PACT-D3的目标是专注于培养下一个 一代对儿科和成人抗癌药物感兴趣的多样化、基础和转化型研究人员 发现和发展。

Abstract This training program is unique for our institution, creating the next generation of basic and clinical investigators who focus their efforts on strategic and comprehensive drug discovery and development in cancer. This program capitalizes on a strong infrastructure and institutional commitment to the training of graduate student trainees for careers in pediatric and adult drug discovery and development. The addition of a pediatric cancer focus addresses an unmet need as well as a major focus of NCI as determined by the NCI's board of scientific advisors and the resulting Childhood Cancer Data (CCDI) initiative. The wealth of adult experience and pediatric researchers is uniquely being used in this T32 to develop novel approaches in pediatric drug discovery. There are no current NCI T32s with an emphasis on both pediatric and adult cancer drug discovery and development training. Cancer remains the leading cause of disease-related death in children. For the many children who experience relapses of their malignant solid tumors curative treatment options are scarce. The pediatric efforts will dovetail with the multi-institutional “Developmental and HyperActive Ras Tumor (DHART)” SPORE led by multi-PI Dr. Wade Clapp focusing on neurofibromatosis type 1 (NF1)—the most common human genetic cancer predisposition syndrome. Findings in this SPORE have led to new clinical trials for pediatric patients, and it is anticipated additional new drugs will need to be developed that is beyond the scope of the SPORE. In addition, the NIH/NICHD-funded P50, “Specialized Centers in Research in Pediatric Developmental Pharmacology” is using precision genomics strategies and in vivo modeling to design efficacious and safe combination therapies for relapsed solid tumors such as osteosarcoma and additional new targets for potential drug development are being identified. This highlights our focus and opportunity to identify new drug targets and develop new drugs specifically for pediatric patients. This program will be supported by collaborative efforts from other programs at the IU School of Medicine (IUSM) including the NCI designated IU Simon Comprehensive Cancer Center (IUSCCC); the Cancer Drug Discovery and Development (CDDD) center which is embedded in the IUSCCC Experimental and Developmental Therapeutics (EDT) program; the TaRget Engagement to Accelerate Therapy Development for Alzheimer's Disease (TREAT-AD) Center and the Indiana CTSI Preclinical Innovation “Think Tank” program. We will focus on graduate students as trainees, who will be recruited from two centralized programs that oversee graduate student recruitment and guide trainees in selecting mentors and home departments. These two programs are the Indiana BioMedical Gateway (IBMG) program, which enrolls ~36 graduate students per year and MSTP which enrolls ~10 per year. Eighteen mentors are intimately involved in this program and drug development efforts at the school. The goal of PACT-D3 is focused on grooming the next generation of diverse, basic, and translational researchers with interests in pediatric and adult cancer drug discovery and development.