Accelerated Path to Cures


Book Description

Accelerated Path to Cures provides a transformative perspective on the power of combining advanced computational technologies, modeling, bioinformatics and machine learning approaches with nonclinical and clinical experimentation to accelerate drug development. This book discusses the application of advanced modeling technologies, from target identification and validation to nonclinical studies in animals to Phase 1-3 human clinical trials and post-approval monitoring, as alternative models of drug development. As a case of successful integration of computational modeling and drug development, we discuss the development of oral small molecule therapeutics for inflammatory bowel disease, from the application of docking studies to screening new chemical entities to the development of next-generation in silico human clinical trials from large-scale clinical data. Additionally, this book illustrates how modeling techniques, machine learning, and informatics can be utilized effectively at each stage of drug development to advance the progress towards predictive, preventive, personalized, precision medicine, and thus provide a successful framework for Path to Cures.




Accelerated Path to Cures


Book Description

Accelerated Path to Cures provides a transformative perspective on the power of combining advanced computational technologies, modeling, bioinformatics and machine learning approaches with nonclinical and clinical experimentation to accelerate drug development. This book discusses the application of advanced modeling technologies, from target identification and validation to nonclinical studies in animals to Phase 1-3 human clinical trials and post-approval monitoring, as alternative models of drug development. As a case of successful integration of computational modeling and drug development, we discuss the development of oral small molecule therapeutics for inflammatory bowel disease, from the application of docking studies to screening new chemical entities to the development of next-generation in silico human clinical trials from large-scale clinical data. Additionally, this book illustrates how modeling techniques, machine learning, and informatics can be utilized effectively at each stage of drug development to advance the progress towards predictive, preventive, personalized, precision medicine, and thus provide a successful framework for Path to Cures.




21st Century Cures


Book Description




Chasing My Cure


Book Description

LOS ANGELES TIMES AND PUBLISHERS WEEKLY BESTSELLER • The powerful memoir of a young doctor and former college athlete diagnosed with a rare disease who spearheaded the search for a cure—and became a champion for a new approach to medical research. “A wonderful and moving chronicle of a doctor’s relentless pursuit, this book serves both patients and physicians in demystifying the science that lies behind medicine.”—Siddhartha Mukherjee, New York Times bestselling author of The Emperor of All Maladies and The Gene David Fajgenbaum, a former Georgetown quarterback, was nicknamed the Beast in medical school, where he was also known for his unmatched mental stamina. But things changed dramatically when he began suffering from inexplicable fatigue. In a matter of weeks, his organs were failing and he was read his last rites. Doctors were baffled by his condition, which they had yet to even diagnose. Floating in and out of consciousness, Fajgenbaum prayed for a second chance, the equivalent of a dramatic play to second the game into overtime. Miraculously, Fajgenbaum survived—only to endure repeated near-death relapses from what would eventually be identified as a form of Castleman disease, an extremely deadly and rare condition that acts like a cross between cancer and an autoimmune disorder. When he relapsed while on the only drug in development and realized that the medical community was unlikely to make progress in time to save his life, Fajgenbaum turned his desperate hope for a cure into concrete action: Between hospitalizations he studied his own charts and tested his own blood samples, looking for clues that could unlock a new treatment. With the help of family, friends, and mentors, he also reached out to other Castleman disease patients and physicians, and eventually came up with an ambitious plan to crowdsource the most promising research questions and recruit world-class researchers to tackle them. Instead of waiting for the scientific stars to align, he would attempt to align them himself. More than five years later and now married to his college sweetheart, Fajgenbaum has seen his hard work pay off: A treatment he identified has induced a tentative remission and his novel approach to collaborative scientific inquiry has become a blueprint for advancing rare disease research. His incredible story demonstrates the potency of hope, and what can happen when the forces of determination, love, family, faith, and serendipity collide. Praise for Chasing My Cure “A page-turning chronicle of living, nearly dying, and discovering what it really means to be invincible in hope.”—Angela Duckworth, #1 New York Times bestselling author of Grit “[A] remarkable memoir . . . Fajgenbaum writes lucidly and movingly . . . Fajgenbaum’s stirring account of his illness will inspire readers.”—Publishers Weekly




Cure


Book Description

A rigorous, sceptical, deeply reported look at the new science behind the mind's extraordinary ability to heal the body. Have you ever felt a surge of adrenaline after narrowly avoiding an accident? Salivated at the sight (or thought) of a sour lemon? Felt turned on just from hearing your partner's voice? If so, then you've experienced how dramatically the workings of your mind can affect your body. Yet while we accept that stress or anxiety can damage our health, the idea of 'healing thoughts' was long ago hijacked by New Age gurus and spiritual healers. Recently, however, serious scientists from a range of fields have been uncovering evidence that our thoughts, emotions, and beliefs can ease pain, heal wounds, fend off infection and heart disease, even slow the progression of AIDS and some cancers. In Cure, award-winning science writer Jo Marchant travels the world to meet the physicians, patients and researchers on the cutting edge of this new world of medicine. We learn how meditation protects against depression and dementia, how social connections increase life expectancy, and how patients who feel cared for recover from surgery faster. We meet Iraq war veterans who are using a virtual arctic world to treat their burns and children whose ADHD is kept under control with half the normal dose of medication. We watch as a transplant patient uses the smell of lavender to calm his hostile immune system and an Olympic runner shaves vital seconds off his time through mind-power alone. Drawing on the very latest research, Marchant explores the vast potential of the mind's ability to heal, acknowledges its limitations, and explains how we can make use of the findings in our own lives. ‘A thought-provoking exploration of how the mind affects the body and can be harnessed to help treat physical illness, by an award-winning science journalist.’ Best Books of 2016, Australian Financial Review ‘A thought-provoking exploration.’ Best Books of 2016, Economist







Translational Medicine


Book Description

Translational Medicine: Optimizing Preclinical Safety Evaluation of Biopharmaceuticals provides scientists responsible for the translation of novel biopharmaceuticals into clinical trials with a better understanding of how to navigate the obstacles that keep innovative medical research discoveries from becoming new therapies or even making it to clinical trials. The book includes sections on protein-based therapeutics, modified proteins, oligonucleotide-based therapies, monoclonal antibodies, antibody–drug conjugates, gene and cell-based therapies, gene-modified cell-based therapies, combination products, and therapeutic vaccines. Best practices are defined for efficient discovery research to facilitate a science-based, efficient, and predictive preclinical development program to ensure clinical efficacy and safety. Key Features: Defines best practices for leveraging of discovery research to facilitate a development program Includes general principles, animal models, biomarkers, preclinical toxicology testing paradigms, and practical applications Discusses rare diseases Discusses "What-Why-When-How" highlighting different considerations based upon product attributes. Includes special considerations for rare diseases About the Editors Joy A. Cavagnaro is an internationally recognized expert in preclinical development and regulatory strategy with an emphasis on genetic medicines.. Her 40-year career spans academia, government (FDA), and the CRO and biotech industries. She was awarded the 2019 Arnold J Lehman Award from the Society of Toxicology for introducing the concept of science-based, case-by-case approach to preclinical safety evaluation, which became the foundation of ICH S6. She currently serves on scientific advisory boards for advocacy groups and companies and consults and lectures in the area of preclinical development of novel therapies. Mary Ellen Cosenza is a regulatory toxicology consultant with over 30 years of senior leadership experience in the biopharmaceutical industry in the U.S., Europe, and emerging markets. She has held leadership position in both the American College of Toxicology (ACT) and the International Union of Toxicology (IUTOX) and is also an adjunct assistant professor at the University of Southern California where she teaches graduate-level courses in toxicology and regulation of biologics.




Second Generation Cell and Gene-Based Therapies


Book Description

Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies




Computational Methods in Drug Discovery and Repurposing for Cancer Therapy


Book Description

Computational Methods in Drug Discovery and Repurposing for Cancer Therapy provides knowledge about ongoing research as well as computational approaches for drug discovery and repurposing for cancer therapy. The book also provides detailed descriptions about target molecules, pathways, and their inhibitors for easy understanding and applicability. The book discusses tools and techniques such as integrated bioinformatics approaches, systems biology tools, molecular docking, computational chemistry, artificial intelligence, machine learning, structure-based virtual screening, biomarkers, and transcriptome; those are discussed in the context of different cancer types, such as colon, pancreatic, glioblastoma, endometrial, and retinoblastoma, among others. This book is a valuable resource for researchers, students, and members of the biomedical and medical fields who want to learn more about the use of computational modeling to better tailor the treatment for cancer patients. Discusses in silico remodeling of effective phytochemical compounds for discovering improved anticancer agents for substantial/significant cancer therapy Covers potential tools of bioinformatics that are applied toward discovering new targets by drug repurposing and strategies to cure different types of cancers Demonstrates the significance of computational and artificial intelligence approaches in anticancer drug discovery Explores how these various advances can be integrated into a precision and personalized medicine approach that can eventually enhance patient care




Laser-Driven Particle Acceleration Towards Radiobiology and Medicine


Book Description

This book deals with the new method of laser-driven acceleration for application to radiation biophysics and medicine. It provides multidisciplinary contributions from world leading scientist in order to assess the state of the art of innovative tools for radiation biology research and medical applications of ionizing radiation. The book contains insightful contributions on highly topical aspects of spatio-temporal radiation biophysics, evolving over several orders of magnitude, typically from femtosecond and sub-micrometer scales. Particular attention is devoted to the emerging technology of laser-driven particle accelerators and their application to spatio-temporal radiation biology and medical physics, customization of non-conventional and selective radiotherapy and optimized radioprotection protocols.