The Challenge of New Therapeutic Approaches for Unmet Therapeutic Needs

The Challenge of New Therapeutic Approaches for Unmet Therapeutic Needs PDF Author: Arianna Carolina Rosa
Publisher: Frontiers Media SA
ISBN: 2889661784
Category : Science
Languages : en
Pages : 185

Book Description
This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact.

Improving and Accelerating Therapeutic Development for Nervous System Disorders

Improving and Accelerating Therapeutic Development for Nervous System Disorders PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309292492
Category : Medical
Languages : en
Pages : 107

Book Description
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.

Cell and Gene Therapies

Cell and Gene Therapies PDF Author: Miguel-Angel Perales
Publisher: Springer
ISBN: 3319543687
Category : Medical
Languages : en
Pages : 286

Book Description
In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Treating Infectious Diseases in a Microbial World

Treating Infectious Diseases in a Microbial World PDF Author: National Research Council
Publisher: National Academies Press
ISBN: 0309180686
Category : Medical
Languages : en
Pages : 102

Book Description
Humans coexist with millions of harmless microorganisms, but emerging diseases, resistance to antibiotics, and the threat of bioterrorism are forcing scientists to look for new ways to confront the microbes that do pose a danger. This report identifies innovative approaches to the development of antimicrobial drugs and vaccines based on a greater understanding of how the human immune system interacts with both good and bad microbes. The report concludes that the development of a single superdrug to fight all infectious agents is unrealistic.

Revisiting Seed and Soil: A New Approach to Target Hibernating Dormant Tumor Cells, 2nd edition

Revisiting Seed and Soil: A New Approach to Target Hibernating Dormant Tumor Cells, 2nd edition PDF Author: Dalit Barkan
Publisher: Frontiers Media SA
ISBN: 2832512542
Category : Medical
Languages : en
Pages : 158

Book Description
Metastasis is the major cause of mortality in cancer patients. Metastases can be present at the time of diagnosis or can occur years or decades after the removal of the primary tumor and treatment. This long latency in the manifestation of recurrent metastatic disease is explained clinically by the persistence of quiescent tumor cells that disseminated early in the course of the disease from the primary tumor to select distant organs. These residing disseminated tumor cells (DTCs) at distant organs lay dormant and asymptomatic until reawakened to form overt metastases. Importantly, the quiescent nature of these “hibernating” DTCs facilitates their resistance to conventional therapies that target actively dividing tumor cells. Therefore, unraveling the biology of dormancy and reactivation of the residing DTCs to life-threatening lesions is of utmost importance in order to develop new therapeutic strategies to prevent the recurrent metastatic disease from ever emerging or to better treat these recurrent cancers. The mechanisms underlying the biology of tumor dormancy and their reactivation to overt metastases are just beginning to emerge thanks to a growing appreciation of the potentially chronic nature of some cancers and the development of experimental model systems for their study. In this Research Topic, we will follow the journey of circulating tumor cells (CTCs) dispatching from the primary site until their successful lodging into a new and foreign site to become DTCs. We will explore the intrinsic mechanisms along with microenvironmental cues and niches that they encounter during their journey that may dictate their fate.

Medical devices made of substances for human health: A challenge in terms of efficacy, safety and sustainability

Medical devices made of substances for human health: A challenge in terms of efficacy, safety and sustainability PDF Author: Juan L. Tamargo
Publisher: Frontiers Media SA
ISBN: 2832531040
Category : Science
Languages : en
Pages : 66

Book Description


The Treatment of Epilepsy

The Treatment of Epilepsy PDF Author: Simon Shorvon
Publisher: John Wiley & Sons
ISBN: 1118936981
Category : Medical
Languages : en
Pages : 1072

Book Description
Highly Commended at the British Medical Association Book Awards 2016 The Treatment of Epilepsy, fourth edition, is a comprehensive reference and clinical guide to the pharmacological, medical and surgical options available in the treatment of epilepsy. The text is compiled by a group of internationally renowned editors and contributors and is now in full color and extensively illustrated The first two sections cover the background to, and principles of, treatment in different clinical situations Section three comprises a series of systematic reviews of contemporary drug therapy, devoting one chapter to each anti-epileptic drug and covering all clinically-relevant aspects Section four focuses on the surgical options, devoting individual chapters to each of the modalities of presurgical assessment and to each surgical operation or approach This 4th edition is extensively revised incorporating the many recent developments in therapy, and comprises 81 chapters from world experts from 18 countries

Medications for Opioid Use Disorder Save Lives

Medications for Opioid Use Disorder Save Lives PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309486483
Category : Medical
Languages : en
Pages : 175

Book Description
The opioid crisis in the United States has come about because of excessive use of these drugs for both legal and illicit purposes and unprecedented levels of consequent opioid use disorder (OUD). More than 2 million people in the United States are estimated to have OUD, which is caused by prolonged use of prescription opioids, heroin, or other illicit opioids. OUD is a life-threatening condition associated with a 20-fold greater risk of early death due to overdose, infectious diseases, trauma, and suicide. Mortality related to OUD continues to escalate as this public health crisis gathers momentum across the country, with opioid overdoses killing more than 47,000 people in 2017 in the United States. Efforts to date have made no real headway in stemming this crisis, in large part because tools that already existâ€"like evidence-based medicationsâ€"are not being deployed to maximum impact. To support the dissemination of accurate patient-focused information about treatments for addiction, and to help provide scientific solutions to the current opioid crisis, this report studies the evidence base on medication assisted treatment (MAT) for OUD. It examines available evidence on the range of parameters and circumstances in which MAT can be effectively delivered and identifies additional research needed.

The Drug Development Paradigm in Oncology

The Drug Development Paradigm in Oncology PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309457971
Category : Medical
Languages : en
Pages : 145

Book Description
Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of therapies in the drug development pipeline, including targeted therapies and associated biomarker tests that can select which patients are most likely to respond, and immunotherapies that harness the body's immune system to destroy cancer cells. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit and clearer distinctions between efficacy and toxicity at an earlier stage of development. However, there is a concern that the traditional processes for cancer drug development, evaluation, and regulatory approval could impede or delay the use of these promising cancer treatments in clinical practice. This has led to a number of effortsâ€"by patient advocates, the pharmaceutical industry, and the Food and Drug Administration (FDA)â€"to accelerate the review of promising new cancer therapies, especially for cancers that currently lack effective treatments. However, generating the necessary data to confirm safety and efficacy during expedited drug development programs can present a unique set of challenges and opportunities. To explore this new landscape in cancer drug development, the National Academies of Sciences, Engineering, and Medicine developed a workshop held in December 2016. This workshop convened cancer researchers, patient advocates, and representatives from industry, academia, and government to discuss challenges with traditional approaches to drug development, opportunities to improve the efficiency of drug development, and strategies to enhance the information available about a cancer therapy throughout its life cycle in order to improve its use in clinical practice. This publication summarizes the presentations and discussions from the workshop.

Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders

Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309373263
Category : Medical
Languages : en
Pages : 107

Book Description
The Institute of Medicine (IOM) Forum on Neuroscience and Nervous System Disorders, in collaboration with the IOM Forum on Drug Discovery, Development, and Translation, convened a workshop on January 20-21, 2015, to explore policy changes that might increase private sector investment in research and development innovation that fills unmet medical needs for central nervous system (CNS) disorders. Workshop participants strategized about how to incentivize companies to fortify their CNS drug development programs, shrinking obstacles that currently deter ventures. Representatives from academia, government agencies, patient groups, and industry gathered to share information and viewpoints, and to brainstorm about budget-neutral policy changes that could help widen the pipeline toward drugs that address unmet needs for CNS disorders. This report summarizes the presentations and discussion of the workshop.