Download or read book Unique Aspects of the Design of Phase I II Clinical Trials of Stem Cell Therapy written by Ivonne H. Schulman and published by . This book was released on 2018 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: This chapter will review the unique aspects and limitations of the design of phase I/II (safety and efficacy) clinical trials of stem cell therapy. Although the classical pharmacologic principles applicable to drugs are not applicable to biologic (live cell) therapeutic agents, an important stage in the development of any new therapeutic agent is the establishment of an optimal dosage and delivery route. This can be particularly challenging when the treatment is a biologic agent, such as stem cells, that may exert its therapeutic effects via complex or poorly understood mechanisms. To date, clinical studies have shown inconsistent findings regarding the relationship between cell dose and clinical outcomes. This can be at least partially attributed to variations in donor cell type, source, characteristics, dosing/concentration, delivery route, underlying mechanisms of action, and efficacy endpoints tested. The current recommendations will be reviewed herein to give new investigators a general understanding of the unique issues that need to be considered and addressed when designing a stem cell therapy phase I/II clinical trial.

Download or read book The Management of Clinical Trials written by Hesham Abdeldayem and published by BoD – Books on Demand. This book was released on 2018-06-06 with total page 91 pages. Available in PDF, EPUB and Kindle. Book excerpt: This concise book is addressed to researchers, clinical investigators, as well as practicing physicians and surgeons who are interested in the fields of clinical research and trials. It covers some important topics related to clinical trials including an introduction to clinical trials, some aspects concerning clinical trials in pediatric age group, and the unique aspects of the design of clinical trials on stem cell therapy.

Download or read book Bayesian Designs for Phase I II Clinical Trials written by Ying Yuan and published by CRC Press. This book was released on 2017-12-19 with total page 310 pages. Available in PDF, EPUB and Kindle. Book excerpt: Reliably optimizing a new treatment in humans is a critical first step in clinical evaluation since choosing a suboptimal dose or schedule may lead to failure in later trials. At the same time, if promising preclinical results do not translate into a real treatment advance, it is important to determine this quickly and terminate the clinical evaluation process to avoid wasting resources. Bayesian Designs for Phase I–II Clinical Trials describes how phase I–II designs can serve as a bridge or protective barrier between preclinical studies and large confirmatory clinical trials. It illustrates many of the severe drawbacks with conventional methods used for early-phase clinical trials and presents numerous Bayesian designs for human clinical trials of new experimental treatment regimes. Written by research leaders from the University of Texas MD Anderson Cancer Center, this book shows how Bayesian designs for early-phase clinical trials can explore, refine, and optimize new experimental treatments. It emphasizes the importance of basing decisions on both efficacy and toxicity.

Download or read book Exploring Novel Clinical Trial Designs for Gene Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Download or read book Mesenchymal Stem Cell Therapy written by Lucas G. Chase and published by Springer Science & Business Media. This book was released on 2012-12-12 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over the past decade, significant efforts have been made to develop stem cell-based therapies for difficult to treat diseases. Multipotent mesenchymal stromal cells, also referred to as mesenchymal stem cells (MSCs), appear to hold great promise in regards to a regenerative cell-based therapy for the treatment of these diseases. Currently, more than 200 clinical trials are underway worldwide exploring the use of MSCs for the treatment of a wide range of disorders including bone, cartilage and tendon damage, myocardial infarction, graft-versus-host disease, Crohn’s disease, diabetes, multiple sclerosis, critical limb ischemia and many others. MSCs were first identified by Friendenstein and colleagues as an adherent stromal cell population within the bone marrow with the ability to form clonogenic colonies in vitro. In regards to the basic biology associated with MSCs, there has been tremendous progress towards understanding this cell population’s phenotype and function from a range of tissue sources. Despite enormous progress and an overall increased understanding of MSCs at the molecular and cellular level, several critical questions remain to be answered in regards to the use of these cells in therapeutic applications. Clinically, both autologous and allogenic approaches for the transplantation of MSCs are being explored. Several of the processing steps needed for the clinical application of MSCs, including isolation from various tissues, scalable in vitro expansion, cell banking, dose preparation, quality control parameters, delivery methods and numerous others are being extensively studied. Despite a significant number of ongoing clinical trials, none of the current therapeutic approaches have, at this point, become a standard of care treatment. Although exceptionally promising, the clinical translation of MSC-based therapies is still a work in progress. The extensive number of ongoing clinical trials is expected to provide a clearer path forward for the realization and implementation of MSCs in regenerative medicine. Towards this end, reviews of current clinical trial results and discussions of relevant topics association with the clinical application of MSCs are compiled in this book from some of the leading researchers in this exciting and rapidly advancing field. Although not absolutely all-inclusive, we hope the chapters within this book can promote and enable a better understanding of the translation of MSCs from bench-to-bedside and inspire researchers to further explore this promising and quickly evolving field.

Download or read book Human Embryonic Stem Cells written by Arlene Chiu and published by Springer Science & Business Media. This book was released on 2003-08 with total page 488 pages. Available in PDF, EPUB and Kindle. Book excerpt: A discussion of all the key issues in the use of human pluripotent stem cells for treating degenerative diseases or for replacing tissues lost from trauma. On the practical side, the topics range from the problems of deriving human embryonic stem cells and driving their differentiation along specific lineages, regulating their development into mature cells, and bringing stem cell therapy to clinical trials. Regulatory issues are addressed in discussions of the ethical debate surrounding the derivation of human embryonic stem cells and the current policies governing their use in the United States and abroad, including the rules and conditions regulating federal funding and questions of intellectual property.

Download or read book Stem Cells and the Future of Regenerative Medicine written by Institute of Medicine and published by National Academies Press. This book was released on 2002-01-25 with total page 112 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recent scientific breakthroughs, celebrity patient advocates, and conflicting religious beliefs have come together to bring the state of stem cell researchâ€"specifically embryonic stem cell researchâ€"into the political crosshairs. President Bush's watershed policy statement allows federal funding for embryonic stem cell research but only on a limited number of stem cell lines. Millions of Americans could be affected by the continuing political debate among policymakers and the public. Stem Cells and the Future of Regenerative Medicine provides a deeper exploration of the biological, ethical, and funding questions prompted by the therapeutic potential of undifferentiated human cells. In terms accessible to lay readers, the book summarizes what we know about adult and embryonic stem cells and discusses how to go about the transition from mouse studies to research that has therapeutic implications for people. Perhaps most important, Stem Cells and the Future of Regenerative Medicine also provides an overview of the moral and ethical problems that arise from the use of embryonic stem cells. This timely book compares the impact of public and private research funding and discusses approaches to appropriate research oversight. Based on the insights of leading scientists, ethicists, and other authorities, the book offers authoritative recommendations regarding the use of existing stem cell lines versus new lines in research, the important role of the federal government in this field of research, and other fundamental issues.

Download or read book Stem Cell Therapies written by Adam C. Berger and published by National Academy Press. This book was released on 2014-06-18 with total page 108 pages. Available in PDF, EPUB and Kindle. Book excerpt: Stem cells offer tremendous promise for advancing health and medicine. Whether being used to replace damaged cells and organs or else by supporting the body's intrinsic repair mechanisms, stem cells hold the potential to treat such debilitating conditions as Parkinson's disease, diabetes, and spinal cord injury. Clinical trials of stem cell treatments are under way in countries around the world, but the evidence base to support the medical use of stem cells remains limited. Despite this paucity of clinical evidence, consumer demand for treatments using stem cells has risen, driven in part by a lack of available treatment options for debilitating diseases as well as direct-to-consumer advertising and public portrayals of stem cell-based treatments. Clinics that offer stem cell therapies for a wide range of diseases and conditions have been established throughout the world, both in newly industrialized countries such as China, India, and Mexico and in developed countries such as the United States and various European nations. Though these therapies are often promoted as being established and effective, they generally have not received stringent regulatory oversight and have not been tested with rigorous trials designed to determine their safety and likely benefits. In the absence of substantiated claims, the potential for harm to patients - as well as to the field of stem cell research in general - may outweigh the potential benefits. To explore these issues, the Institute of Medicine, the National Academy of Sciences, and the International Society for Stem Cell Research held a workshop in November 2013. "Stem Cell Therapies" summarizes the workshop. Researchers, clinicians, patients, policy makers, and others from North America, Europe, and Asia met to examine the global pattern of treatments and products being offered, the range of patient experiences, and options to maximize the well-being of patients, either by protecting them from treatments that are dangerous or ineffective or by steering them toward treatments that are effective. This report discusses the current environment in which patients are receiving unregulated stem cell offerings, focusing on the treatments being offered and their risks and benefits. The report considers the evidence base for clinical application of stem cell technologies and ways to assure the quality of stem cell offerings.

Download or read book Chronic Graft Versus Host Disease written by Georgia B. Vogelsang and published by Cambridge University Press. This book was released on 2009-04-20 with total page 427 pages. Available in PDF, EPUB and Kindle. Book excerpt: Chronic graft versus host disease (GVHD) is the most common complication of allogenic bone marrow transplantation. Because of the protracted clinical course of chronic GVHD, transplant centers and hematology/oncology offices are inadequately equipped to manage these immuno-incompetent patients with a multi-system disorder. Practitioners need to be able to recognize and effectively manage chronic GVHD as a late effect of more than half of allogenic transplantations. The text is oriented for the clinician, with chapters covering staging, organ site and system-specific manifestations, treatment options, and supportive care. Drs Georgia B. Vogelsang and Steven Z. Pavletic have been pioneers in the recognition of the multi-organ complexity of this disease and have gathered the input of a variety of subspecialist physicians for this book. This book fills the gap in practical literature on chronic GVHD, providing a comprehensive, up-to-date, and clinically relevant resource for anyone who deals with cancer patients post-transplant.

Download or read book Sharing Publication Related Data and Materials written by National Research Council and published by National Academies Press. This book was released on 2003-04-17 with total page 240 pages. Available in PDF, EPUB and Kindle. Book excerpt: Biologists communicate to the research community and document their scientific accomplishments by publishing in scholarly journals. This report explores the responsibilities of authors to share data, software, and materials related to their publications. In addition to describing the principles that support community standards for sharing different kinds of data and materials, the report makes recommendations for ways to facilitate sharing in the future.

Download or read book Stem Cells written by Mariusz Z. Ratajczak and published by Springer Nature. This book was released on 2020-01-02 with total page 410 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since different types of stem cells for therapeutic applications have recently been proposed, this timely volume explores various sources of stem cells for tissue and organ regeneration and discusses their advantages and limitations. Also discussed are pros and cons for using embryonic stem cells, induced pluripotent stem cells, and adult stem cells isolated from postnatal tissues. Different types of adult stem cells for therapeutic applications are also reviewed, including hematopoietic stem cells, epidermal stem cells, endothelial progenitors, neural stem cells, mesenchymal stem cells, and very small embryonic-like stem cells. This book also addresses paracrine effects of stem cells in regenerative medicine that are mediated by extracellular microvesicles and soluble secretome. Finally, potential applications of stem cells in cardiology, gastroenterology, neurology, immunotherapy, and aging are presented. This is an ideal book for students and researchers working in the stem cell research field.

Download or read book Policy Issues in the Clinical Development and Use of Immunotherapy for Cancer Treatment written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2016-12-10 with total page 135 pages. Available in PDF, EPUB and Kindle. Book excerpt: Immunotherapy is a form of cancer therapy that harnesses the body's immune system to destroy cancer cells. In recent years, immunotherapies have been developed for several cancers, including advanced melanoma, lung cancer, and kidney cancer. In some patients with metastatic cancers who have not responded well to other treatments, immunotherapy treatment has resulted in complete and durable responses. Given these promising findings, it is hoped that continued immunotherapy research and development will produce better cancer treatments that improve patient outcomes. With this promise, however, there is also recognition that the clinical and biological landscape for immunotherapies is novel and not yet well understood. For example, adverse events with immunotherapy treatment are quite different from those experienced with other types of cancer therapy. Similarly, immunotherapy dosing, therapeutic responses, and response time lines are also markedly different from other cancer therapies. To examine these challenges and explore strategies to overcome them, the National Academies of Sciences, Engineering, and Medicine held a workshop in February and March of 2016. This report summarizes the presentations and discussions from the workshop.

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Download or read book Preclinical Safety Evaluation of Biopharmaceuticals written by Joy A. Cavagnaro and published by John Wiley & Sons. This book was released on 2013-03-07 with total page 1012 pages. Available in PDF, EPUB and Kindle. Book excerpt: "The goal is to provide a comprehensive reference book for the preclinicaldiscovery and development scientist whose responsibilities span target identification, lead candidate selection, pharmacokinetics, pharmacology, and toxicology, and for regulatory scientists whose responsibilities include the evaluation of novel therapies." —From the Afterword by Anthony D. Dayan Proper preclinical safety evaluation can improve the predictive value, lessen the time and cost of launching new biopharmaceuticals, and speed potentially lifesaving drugs to market. This guide covers topics ranging from lead candidate selection to establishing proof of concept and toxicity testing to the selection of the first human doses. With chapters contributed by experts in their specific areas, Preclinical Safety Evaluation of Biopharmaceuticals: A Science-Based Approach to Facilitating Clinical Trials: Includes an overview of biopharmaceuticals with information on regulation and methods of production Discusses the principles of ICH S6 and their implementation in the U.S., Europe, and Japan Covers current practices in preclinical development and includes a comparison of safety assessments for small molecules with those for biopharmaceuticals Addresses all aspects of the preclinical evaluation process, including: the selection of relevant species; safety/toxicity endpoints; specific considerations based upon class; and practical considerations in the design, implementation, and analysis of biopharmaceuticals Covers transitioning from preclinical development to clinical trials This is a hands-on, straightforward reference for professionals involved in preclinical drug development, including scientists, toxicologists, project managers, consultants, and regulatory personnel.

Download or read book Improving and Accelerating Therapeutic Development for Nervous System Disorders written by Institute of Medicine and published by National Academies Press. This book was released on 2014-02-06 with total page 107 pages. Available in PDF, EPUB and Kindle. Book excerpt: Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

Download or read book Clinical Trials written by Tom Brody and published by Academic Press. This book was released on 2016-02-19 with total page 897 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials. Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and more Extensively covers the "study schema" and related features of study design Incorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trials Includes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers

Download or read book Stem Cells From Hype to Real Hope written by Khawaja Husnain Haider and published by Walter de Gruyter GmbH & Co KG. This book was released on 2018-12-17 with total page 340 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is a compilation of the bench experience of leading experts from various research labs involved in the cutting edge area of research. The authors describe the use of stem cells both as part of the combinatorial therapeutic intervention approach and as tools (disease model) during drug development, highlighting the shift from a conventional symptomatic treatment strategy to addressing the root cause of the disease process. The book is a continuum of the previously published book entitled "Stem Cells: from Drug to Drug Discovery" which was published in 2017.