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.

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 this book we provide insights into liver – cancer and immunology. Experts in the field provide an overview over fundamental immunological questions in liver cancer and tumorimmunology, which form the base for immune based approaches in HCC, which gain increasing interest in the community due to first promising results obtained in early clinical trials. Hepatocellular carcinoma (HCC) is the third most common cause of cancer related death in the United States. Treatment options are limited. Viral hepatitis is one of the major risk factors for HCC, which represents a typical “inflammation-induced” cancer. Immune-based treatment approaches have revolutionized oncology in recent years. Various treatment strategies have received FDA approval including dendritic cell vaccination, for prostate cancer as well as immune checkpoint inhibition targeting the CTLA4 or the PD1/PDL1 axis in melanoma, lung, and kidney cancer. Additionally, cell based therapies (adoptive T cell therapy, CAR T cells and TCR transduced T cells) have demonstrated significant efficacy in patients with B cell malignancies and melanoma. Immune checkpoint inhibitors in particular have generated enormous excitement across the entire field of oncology, providing a significant benefit to a minority of patients.

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.

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.

Immune Checkpoint Inhibitors - New Insights and Recent Progress explores a vast array of subjects related to immune checkpoint inhibitors and presents novel insights in this emerging field. Chapters address such topics as mechanistic approaches of emerging immune checkpoint inhibitors, their role in clinical and pre-clinical trials, the manipulation of the system by immune-related adverse events that hinder the utility of these immune molecules, and the predictive and prognostic aspects of these molecules as biomarkers of response in immunotherapy. The book is useful for students, clinicians, and scientists to gain updated information on managing patients treated with immune checkpoint inhibitors.

Advances in biomedical research have increased our understanding of the complex nature of disease and the interaction of multiple molecular pathways involved in cancer. Combining investigational products early in their development is thought to be a promising strategy for identifying effective therapies. The IOM's National Cancer Policy Forum held a workshop to discuss challenges and identify potential solutions to improve collaboration and advance the development of combination investigational cancer therapies.