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.
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.
In recent years, significant progress has been made in the clinical development and use of various types of cancer immunotherapy, all of which rely on the immune system to fight cancer. The majority of new cancer drug applications submitted to the Food and Drug Administration (FDA) are for immunotherapies or combinations involving immunotherapies. One type of immunotherapy is an immune checkpoint inhibitor. Cells in the human body have proteins that regulate the immune system response to foreign invaders (e.g., cancer cells, microorganisms). However, cancer cells can coopt these "checkpoint" proteins and thwart the immune system's ability to recognize and attack cancer cells. To help promote an immune response to cancer, researchers have developed immune checkpoint inhibitors that enable T-cells to recognize cancer cells as foreign and to prevent deactivation of an immune system response. To examine the challenges and opportunities to develop combination cancer therapies that include immune checkpoint inhibitors, the National Cancer Policy Forum held a workshop on July 16â€"17, 2018, in Washington, DC. This workshop convened stakeholders with a broad range of expertise, including cancer researchers, clinicians, patient advocates, and representatives from industry, academia, and government. This publication summarizes the presentations and discussions from the workshop.
A guide to state-of-the-art cancer immunotherapy in translational cancer research A volume in the Translational Oncology series, Immunotherapy in Translational Cancer Research explores the recent developments in the role that immunotherapy plays in the treatment of a wide range of cancers. The editors present key concepts, illustrative examples, and suggest alternative strategies in order to achieve individualized targeted therapy. Comprehensive in scope, Immunotherapy in Translational Cancer Research reviews the relevant history, current state, and the future of burgeoning cancer-fighting therapies. The book also includes critical information on drug development, clinical trials, and governmental resources and regulatory issues. Each chapter is created to feature: development of the immunotherapy; challenges that have been overcome in order to scale up and undertake clinical trials; and clinical experience and application of research. This authoritative volume is edited by a team of noted experts from MD Anderson Cancer Center, the world’s foremost cancer research and care center and: Offers a comprehensive presentation of state-of-the-art cancer immunotherapy research that accelerates the pace of clinical cancer care Filled with the concepts, examples, and approaches for developing individualized therapy Explores the breath of treatments that reflect the complexity of the immune system itself Includes contributions from a panel international experts in the field of immunotherapy Designed for physicians, medical students, scientists, pharmaceutical executives, public health and public policy government leaders and community oncologists, this essential resource offers a guide to the bidirectional interaction between laboratory and clinic immunotherapy cancer research.
“Drs. Butterfield, Kaufman and Marincola on behalf of the SITC have created a comprehensive must-have resource covering the basic and translational science behind approved and investigational immune therapies, and disease-by-disease clinical application of the therapies, written by leaders in the field. The textbook will be highly valuable reading for those just entering the field and for experienced scientists and clinicians looking to expand their knowledge base.” —Mario Sznol, MD, Professor of Medicine (Medical Oncology), Yale School of Medicine, New Haven, Connecticut “Given the important and exciting advances in cancer immunotherapy, it is not surprising that there is no dearth of books on the subject. Despite the competition, however, this book is a unique masterpiece and a must-have comprehensive resource for anyone interested in this area and for every medical library.” Score: 100, 5 Stars, Doody’s Medical Reviews Cancer Immunotherapy Principles and Practice, from the Society for Immunotherapy of Cancer (SITC), is the authoritative reference on cancer immunobiology and the immunotherapy treatments that harness the immune system to combat malignant disease. Featuring five sections and over 50 chapters covering the Basic Principles of Tumor Immunology, Cancer Immunotherapy Targets and Classes, Immune Function in Cancer Patients, Disease-Specific Treatments and Outcomes, and Regulatory Aspects of Cancer Immunotherapy, this book covers all major topics that have shaped the development of immunotherapy and propelled it to its current place at the forefront of cancer treatment innovation. This volume is a comprehensive resource for oncologists and fellows, immunologists, cancer researchers, and related practitioners seeking understanding of the basic science and clinical applications of cancer immunotherapy. As well as presenting the evidence for immune-based cancer treatment, it positions immunotherapy in the context of other available cancer treatments and provides data on response rates, risks, and toxicities across a variety of diseases. Filled with detailed tables, and instructive illustrations, as well as key points for quick reference, Cancer Immunotherapy Principles and Practice simplifies a challenging and dynamic subject. KEY FEATURES Clearly summarizes the basic principles and research supporting cancer immunotherapy clinical translation Contains expert guidance and treatment strategies for all immunotherapy classes and agents, including cell-based therapies, monoclonal antibodies, cytokine therapies, checkpoint inhibitors, oncolytic viruses, adjuvant approaches, and treatment combinations Includes expert perspectives from leading authorities in the field Provides information on all FDA-approved immunotherapies, including clinical management and outcome data Discusses clinical aspects of immunotherapy for individual cancer types, including melanoma and other skin cancers, lung cancers, gynecologic cancers, gastrointestinal cancers, hematologic cancers, genitourinary cancers, head and neck cancers, sarcomas, brain and other CNS cancers, breast cancer, and pediatric malignancies. Explains regulatory aspects behind the development and approval of immunotherapy drugs
A long-held goal in oncology has been to develop therapies that target the specific abnormalities in each patient's cancer rather than simply treating cancers based on the tissue of origin. In the past decade, advances in technology have enabled researchers to relatively quickly and inexpensively determine, in minute detail, the genetic makeup of tumors. Although relatively few targeted cancer therapies are currently available in the clinic and it is not yet clear whether all cancers are driven by genetic changes that can be targeted, there is widespread optimism in the cancer community that this new ability to assess the genetic abnormalities in tumors will ultimately lead to better cancer treatments and improved patient outcomes. Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies is the summary of a workshop convened in November 2014 by the Institute of Medicine's National Cancer Policy Forum to discuss recent trends in the development and implementation of molecularly targeted cancer therapies and explore potential policy actions to address specific challenges. This report highlights the presentations and discussions at the workshop.
One of the challenges in treating cancer is the disease's complexity and variation among patients. Cancer manifests differently in each patient, so treatments that are effective in one patient may not be effective in another. As cancer care becomes more personalized, subpopulations of individuals will be given preventive or therapeutic interventions based on their susceptibility to a particular disease or their predicted response to a specific treatment. However, before the use of personalized cancer care can reach its full potential, the health care system must resolve a number of technological, regulatory, and reimbursement issues. To explore these policy challenges, the National Cancer Policy Forum held the workshop Policy Issues in the Development of Personalized Medicine in Oncology in June 2009. Experts provided presentations on the current state of personalized medicine technology, as well as issues in the validation of, regulation of, and reimbursement for the predictive tests that underpin personalized medicine. Participants discussed the obstacles and possible solutions to further developing and using personalized medicine technologies. This document summarizes the workshop.
In the nearly 40 years since implementation of federal regulations governing the protection of human participants in research, the number of clinical studies has grown exponentially. These studies have become more complex, with multisite trials now common, and there is increasing use of archived biospecimens and related data, including genomics data. In addition, growing emphasis on targeted cancer therapies requires greater collaboration and sharing of research data to ensure that rare patient subsets are adequately represented. Electronic records enable more extensive data collection and mining, but also raise concerns about the potential for inappropriate or unauthorized use of data, bringing patient protections into a new landscape. There are also long-standing concerns about the processes and forms used to obtain informed consent from patients participating in clinical studies. These changes and challenges raise new ethical and practical questions for the oversight of clinical studies, and for protecting patients and their health information in an efficient manner that does not compromise the progress of biomedical research. Contemporary Issues for Protecting Patients in Cancer Research is the summary of a workshop convened by the National Cancer Policy Forum of the Institute of Medicine in February 2014 to explore contemporary issues in human subjects protections as they pertain to cancer research, with the goal of identifying potential relevant policy actions. Clinical researchers, government officials, members of Institutional Review Boards, and patient advocates met to discuss clinical cancer research and oversight. This report examines current regulatory provisions that may not adequately protect patients or may be hindering research, and discusses potential strategies and actions to address those challenges.
The use of immune modulator therapeutics, a type of immunotherapy enhancing the body immune system response to cancer, was perceived as the beginning of a new era in cancer care. While still important and frequently used, some of these therapeutics produce uneven response rates, disease resistance, and serious side effects. The National Academies National Cancer Policy Forum, in collaboration with the Forum on Drug Discovery, Development, and Translation, hosted a public workshop to discuss challenges related to immunotherapy treatment resistance, as well as potential policy opportunities to improve the development of immunotherapies for cancer treatment.
Many ongoing changes are likely to have an impact on cancer research and care. For example, technological advances are rapidly changing the way cancer research is conducted, and the recently passed healthcare reform legislation has many implications for cancer care. Technological advances are altering the way cancer research is conducted and cancer care is delivered, and the recently passed healthcare reform legislation has many implications for cancer care. There is a growing emphasis on molecularly targeted therapies, information technology (IT), and patient-centered care, and clinical cancer research has become a global endeavor. At the same time, there are concerns about shrinking research budgets and escalating costs of cancer care. Considering such changes, the National Cancer Policy Forum (NCPF) of the Institute of Medicine held a National Cancer Policy Summit on October 25, 2010. The Summit convened key leaders in the cancer community to identify and discuss the most pressing policy issues in cancer research and cancer care. The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care is a summary of the summit. The report explores policy issues related to cancer research, the implementation of healthcare reform, delivery of cancer care, and cancer control and public health needs. Expert participants suggested many potential actions to provide patient-centered cancer care, to foster more collaboration, and to achieve other goals to improve research and care.
