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

点击翻译按钮获取中文摘要

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.