Pediatric Cancer Therapeutics Development

ISBN-13: 9783031063596 / Angielski / Miękka / 2023

Opis

This book provides a comprehensive overview of the scientific, medical, regulatory, and economic considerations associated with the discovery, development, and delivery of novel therapeutics for children with cancer. Co-authored by a diverse team from academic, government, and industry backgrounds, the book describes the steps in the process from the identification of a promising therapeutic target to the evaluation of drug candidates in the various phases of clinical testing and regulatory review. Throughout, special emphasis is placed on the unique biology of pediatric malignancies and the medical and social needs of children and their families. In providing a firm grounding in the drug development process, the book will be of value to all with an interest in how medicines currently used to treat pediatric cancer were made available. This includes trainees as well as established practitioners and others participating in translational and clinical research in the academic setting.

Spis treści

Chapter 1: History of Drug Development

Chapter 2: Target Identification/Validation and Drug Discovery 

Chapter 3: The Pediatric Pre-clinical Testing Program 

Chapter 4: Non-Clinical Toxicology 

Chapter 5: Study Design and Biostatistics 

Chapter 6: Personalized Medicine and Its Impact on Study Design 

Chapter 7: Immunotherapy, including Gene and Cellular Therapy

Chapter 8: The Pharma/Biotech Model for Drug Development

Chapter 9: Economics of Cancer Therapeutics Development

Chapter 10: Clinical Trial Networks

Chapter 11: Clinical Research Organizations

Chapter 12: Role of Patients and Advocates in Cancer Therapeutics Development 

Chapter 13: International Regulatory Considerations and Initiatives 

Chapter 13: Ethical Considerations in Cancer Therapeutics Development for Life-threatening Diseases 

Chapter 14: Future Directions

O autorze

Jorge DiMartino, MD, PhD, is Chief Medical Officer and Executive Vice President of Clinical Development at Kronos Bio, a biotech company focused on drugging transcriptional dysregulation in cancer. Jorge trained in pediatrics and pediatric hematology/oncology at Stanford from 1993 to 1998. After starting his career as an academic physician scientist at Stanford and Cincinnati Children’s Hospital, Jorge moved to Industry in 2005 as one of the founding members of the Exploratory Clinical Development group at Genentech. In this role, Jorge played a key role in the early development of the hedgehog pathway inhibitor vismodegib and the BCL-2 inhibitors, navitoclax and venetoclax. Jorge left Genentech in 2011 to lead the epigenetics research efforts at Celgene. In collaboration with corporate partners Agios and Epizyme, he contributed to the design and analysis of early clinical studies of the IDH2 inhibitor enasidenib and the DOT1L inhibitor pinometostat. As Vice President of Translational Development, Jorge co-led the Protein Homeostasis and Epigenetics Thematic Center of Excellence, a vertically integrated research unit spanning drug discovery through clinical Proof of Concept. Jorge joined Kronos Bio in December of 2019.

Gregory H. Reaman, M.D. joined the Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration as the Associate Director of the Office of Oncology Drug Products in 2011. In 2017, he bacame the Associate Director for Pediatric Oncology in the FDA’s Oncology Center of Excellence. He is the Founding and Immediate Past Chair of the Children’s Oncology Group (COG) having served in this capacity from 2000 through 2010. The COG is comprised of over 200 member institutions, dedicated to clinical, translational, and epidemiology research in childhood cancer. He is a Professor of Pediatrics at The George Washington University School of Medicine and Health Sciences and a member of the Division of Hematology-Oncology at the Children’s National Medical Center in Washington, D.C., which he directed for more than 17 years.He has served on the Editorial Boards of Leukemia, Journal of Clinical Oncology, Journal of Pediatric Hematology/Oncology, Pediatric Blood and Cancer, The Oncologist, Cancer, and Physicians Data Query (PDQ), National Cancer Institute as well as www.PLWC.org (People Living with Cancer, now Cancer.net). He has served as an Associate Editor of Cancer and Leukemia and Lymphoma. He served on the Board of Directors of the American Cancer Society and chaired its Task Force on Children and Cancer. He has authored or co-authored more than 350 peer-reviewed publications and more than 25 books and book chapters. His research  interests include the biology and therapy of childhood acute leukemia and new drug development for pediatric cancer.

Franklin O. Smith, III, M.D., FAAP, FACP, is Senior Vice President at Medpace and is Professor of Medicine and Pediatrics at the University of Cincinnati College of Medicine. He graduated from the University of South Carolina School of Medicine and completed postdoctoral training in pediatrics at the University of Florida and fellowship training in pediatric hematology/oncology at the University of Washington and the Fred Hutchinson Cancer Research Center. Prior to joining Medpace, Dr. Smith was the Clinical Director at the University of Cincinnati Cancer Institute. Previously, he was the Director, Division of Hematology/Oncology at Cincinnati Children's Hospital Medical Center and Vice-Chair of the Children's Oncology Group. He has authored more than 180 medical and scientific articles, book chapters and books. Dr. Smith is active with various national and international committees and societies in the field of research and clinical care. He currently serves as President of the Board of Managers for FACT Consulting Services and faculty for the ASCO/AACR Clinical Cancer Research Workshop. Dr. Smith has also served on standing and ad hoc committees for the FDA and NIH.