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

摘要 该培训计划是我们机构的独特之处，培养下一代基础和临床研究人员 他们专注于癌症的战略性和全面的药物发现和开发。这个程序 利用强大的基础设施和机构承诺培训研究生学员， 在儿科和成人药物发现和开发的职业生涯。增加了儿科癌症重点 解决了一个未满足的需求，以及由NCI的科学委员会确定的NCI的主要重点 儿童癌症数据（Childhood Cancer Data，CCDI）。丰富的成人经验和儿科经验 研究人员在这个T32中被独特地用于开发儿科药物发现的新方法。那里 目前没有NCI T32，重点是儿科和成人癌症药物的发现和开发 训练癌症仍然是儿童疾病相关死亡的主要原因。对于那些 经验复发的恶性实体瘤治愈性治疗选择是稀缺的。儿科的努力 将与多机构的“发育和过度活跃的Ras肿瘤（DHART）”SPORE相吻合， Wade Clapp博士专注于1型神经纤维瘤病（NF 1）-最常见的人类遗传性癌症 易感综合征在这个孢子的发现导致了新的临床试验的儿科患者，它是 预计将需要开发超出SPORE范围的其他新药。此外，本发明还提供了一种方法， NIH/NICHD资助的P50“儿科发育药理学研究专业中心”是 使用精确的基因组学策略和体内建模来设计有效和安全的联合疗法 对于复发性实体瘤如骨肉瘤和潜在药物开发的其他新靶点， 被识别。这凸显了我们确定新药靶点和开发新药的重点和机会 特别是儿科患者。该计划将得到其他计划的合作支持， IU医学院（IUSM），包括NCI指定的IU西蒙综合癌症中心 癌症药物发现和开发（CDDD）中心，该中心嵌入IUSCCC 实验和发展治疗学（EDT）计划; TaRget参与加速治疗 阿尔茨海默病（AD）开发中心和印第安纳州CTSI临床前创新“Think 坦克”计划。我们将重点培养研究生作为培训生，他们将从两个集中的 监督研究生招聘并指导学员选择导师和家庭的计划 部门这两个项目是印第安纳州生物医学门户（IBMG）项目，该项目招收了约36名学生。 每年招收约10名研究生和MSTP。十八位导师密切参与 这个项目和药物开发在学校的努力。PACT-D3的目标是培养下一个 培养对儿科和成人癌症药物感兴趣的多样化、基础和转化研究人员 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.