NCI-Related Cancer Moonshot Activities
Through the Cancer MoonshotSM, many new collaborations and activities have been launched that address barriers and opportunities in cancer research. NCI is involved in a number of these activities.
NCI-Related Activities Underway
National Cancer Institute Formulary
Leveraging lessons learned through the NCI-MATCH Trial, in which agents from different companies are tested alone or in combination under a single study, the institute is forging a public–private partnership with pharmaceutical and biotechnology companies to expedite cancer researchers’ access to investigational agents and approved drugs. Researchers from NCI-Designated Cancer Centers will be able to apply for access to agents from the available formulary list and test them in new preclinical or clinical studies, including combination studies of formulary agents from different companies. The NCI Formulary will alleviate the need to negotiate with each company independently for individual research projects, a process that can take as long as 18 months. Ultimately this approach will expedite the start of clinical trials and bring new treatment options to cancer patients faster.
Making Clinical Research Trials More Accessible to Cancer Patients
NCI, in partnership with the White House Presidential Innovation Fellows, is redesigning how patients and oncologists learn about and find information on cancer clinical trials. The goal is to ensure that patients and their care teams have access to the information they need at the right time, as well as to strengthen participation in cancer research studies to help accelerate medical discoveries and treatments for cancer. The first phase will make cancer clinical trials data hosted on cancer.gov available through an application programming interface (API) for advocacy groups, academia, and others in the cancer ecosystem. The new cancer clinical trial API will enable third-party innovators, including Smart Patients, Synapse, Cure Forward, and Antidote, to build applications, integrations, search tools, and digital platforms tailored to individual communities that bring clinical trial information to more providers, patients, and their family members.
Strategic Computing Partnership between the Department of Energy and the National Cancer Institute to Accelerate Precision Oncology
The Department of Energy (DOE), in partnership with NCI, has launched three new pilot projects focused on bringing together nearly 100 cancer researchers, care providers, computer scientists, and engineers to apply the nation's most advanced supercomputing capabilities to analyze data from preclinical models in cancer, molecular interaction data for RAS, and cancer surveillance data. The effort spans four DOE National Laboratories—Argonne, Los Alamos, Lawrence Livermore, and Oak Ridge—in conjunction with the NCI Frederick National Laboratory for Cancer Research. By joining these forces under a coordinated effort, these new projects will refine our understanding of the mechanisms leading to cancer development and thereby accelerate the development of promising therapies that are more effective and less toxic.
The CANcer Distributed Learning Environment (CANDLE) DOE exascale computing project will deliver essential new computing capabilities that will support the three pilot projects, while providing insight into techniques that are essential to understand, accelerate, and inform the design of future computing solutions in precision oncology.
Preclinical Research Partnership to Evaluate the Potential of Particle Beam Radiotherapy
The National Aeronautics and Space Administration (NASA) and NCI are establishing a new collaboration to study the biological effects of particle beam radiotherapy, a novel technology that may deliver a more targeted dose of radiation to tumor cells. Currently, NCI supports several efforts in this area, including comparing the efficacy of carbon ion therapy for the treatment of pancreatic cancer, and NASA is studying the biological effects of a wide range of heavy ions to develop countermeasures for protecting astronauts from the space radiation environment. Under this new partnership, agencies will share data and biospecimens to assess the biological effects of particle beam radiotherapy and evaluate its potential value as a new approach to fighting cancer.
Creation of an Open Access Resource for Sharing Cancer Data via the Genomic Data Commons
As part of the Cancer Moonshot and the Precision Medicine Initiative®, data from Foundation Medicine is more than doubling the total number of patients represented within NCI's Genomic Data Commons (GDC), bringing its total to more than 32,000 patients. At its launch in early June, the GDC already shared more than 5 petabytes of raw unprocessed genomic data from large research projects on more than 30 tumor types from more than 14,500 patients, along with associated clinical data (e.g., clinical diagnosis, treatment history, and survival data). The GDC provides a foundational system for broad sharing and analysis of cancer genomic data, which is critical for advancing the field of precision medicine and improving the care of cancer patients.
The GDC is complemented by the NCI Genomics Cloud Pilots, which were recently funded for an additional year with the goal of co-locating the computational tools developed by the three Cloud Pilots with the GDC to create a cohesive model for large-scale genomic data management and analysis. The pilots also aim to expand the GDC to other data types, such as proteomics or imaging data. The cloud pilots are an important part of exploring new mechanisms for data access, computation, and analysis for cancer data. The pilots are a partnership of The Broad Institute, The Institute for Systems Biology, and Seven Bridges Genomics. They went public in the spring of 2016 and are under active evaluation through September 2017. As part of the Cancer Moonshot, NCI jointly announced a new public–private partnership designed to build a sustainable model for maintaining cancer genomic data in the cloud for use by cancer researchers through the GDC and the Genomics Cloud Pilots.
National Institutes of Health Public-Private Partnership for Accelerating Cancer Therapies
NIH is collaborating with 12 biopharmaceutical companies, multiple research foundations, philanthropies, and the Foundation for the NIH to develop a new program under the Cancer Moonshot, the Partnership for Accelerating Cancer Therapies (PACT). PACT will fund pre-competitive cancer research and share broadly all data generated for further research, ultimately bringing more new therapies to patients in less time. Potential initial focus areas include understanding responses to cancer therapies, clinical trial platforms for combination therapies, predictive modeling approaches, and therapies for rare cancers.
Forging New Partnerships to Catalyze New Drug Discovery and Development
The Department of Energy, NCI, and GlaxoSmithKline are forming a new public–private partnership designed to harness high-performance computing and diverse biological data to accelerate the drug discovery process and bring new cancer therapies from target to first in human trials in less than a year. This partnership will bring together scientists from multiple disciplines to advance our understanding of cancer by finding patterns in vast and complex datasets to accelerate the development of new cancer therapies.
The Department of Defense (DoD), the Department of Veterans Affairs (VA), and NCI have formed a new collaboration using state-of-the-art research methods in proteogenomics to more rapidly identify unique targets and pathways of cancer for detection and intervention. These methods are looking at a patient’s genes that may lead to cancer and the expression of these genes in the form of proteins, with potential impact on understanding disease formation and treatment for cancer patients. The full set of medical images, including CT and MRI scans, obtained for each patient before and during treatment will also be acquired by and curated in The Cancer Imaging Archive. Each set of noninvasive images can be connected to the patient’s clinical, genomic, and proteomic data, and big data science techniques can be applied to understand the relationships among these data. Using all of the data available (analytical, invasive, noninvasive, and clinical) will enable researchers to develop predictive and prognostic models to improve patient care.
Initial collaborative efforts are focusing on a cohort of 8,000 patients with lung cancer from the nation's two largest health care systems—the VA and DoD—and will make data broadly available to the research community. Ultimately, the effort will be expanded to additional cancer types to reach more cancer patients within the VA and DoD, providing knowledge scalable for physicians across the country treating the more than 1.6 million new patients diagnosed with cancer each year.
The United States has signed Memoranda of Understanding (MOUs) with eight countries since July 2016 to facilitate collaborations for basic and clinical proteogenomic studies and their translation to care. The signed MOUs are with Australia, Canada, China, Germany, Japan, the Republic of Korea, Switzerland, and the American Institute of Taiwan/Taipei Economic and Cultural Representative Office.
Proposed Activities with NCI Involvement
Create a high-quality performance status tracking system for cancer patients during therapy and long-term follow-up
A joint effort between NCI and DoD is aimed at improving the lives of cancer patients undergoing treatment, as well as members of the military attempting to complete a mission. Both cancer patients and military personnel suffer similarly from physical, physiological, and environmental stressors that affect their ability to perform as they each face potentially life-threatening challenges. An accurate, quantitative assessment could prevent doctors from sending patients for treatment they are not healthy enough to endure—and could help commanding officers avoid sending military personnel on missions they are not healthy enough to complete. The Analytical Tools to Objectively Measure Human Performance (ATOM-HP) project will create a high-quality performance status tracking system for cancer patients during therapy and long-term follow up. The goal is to be able to assess, in real time, a cancer patient’s experiences with physical, psychological, and environmental factors, among others. This is expected to advance the ways by which doctors can monitor core dynamics in cancer patients on a regular basis.
Rapidly analyze the molecular profile of thousands of tumors
Advances in molecular profiling of patients are difficult to test and rapidly deploy in the current translational pipeline. The APOLLO network will strengthen and develop research cooperation in using state-of-the-art methods in proteogenomics to characterize and compare tumors, develop a deeper understanding of cancer biology, and identify potential targets and pathways of cancer prevention, detection, and intervention. Developing these methods will lead to better diagnostic tools and effective treatments once disease pathways are discovered. Collaborations between the APOLLO consortium and the new DoD and VA Infrastructure for Clinical Intelligence (DAVINCI) project will synergize the data-sharing infrastructure. Protocols for data sharing will be reviewed by appropriate institutional review boards and pilot projects will validate the feasibility of sharing existing samples. Ongoing prospective tissue collection will occur into the next 3 years as will mergers with APOLLO genomic and proteomic workflows.
Develop predictive computer algorithms to rapidly develop, test, and validate predictive preclinical models
Combined efforts across NCI and DOE will provide a practical, scalable approach to preclinical screening aimed at opening up new therapeutic options for cancer patients. These models will seek to support the treatment choices of physicians and patients with the goal of achieving the best possible clinical outcome. To lay the groundwork for this effort, a memorandum of understanding between DOE and NCI was established in July 2016. It outlines the agencies’ intentions to collaborate in developing a shared technology ecosystem and targeted applications to bring advanced computing capabilities to biological research, thereby transforming drug and treatment development and improving patient care and outcomes. NCI and DOE will begin by identifying key data gaps; developing initial frameworks and reference implementations; establishing standards of use for data to support model development; and extending involvement to other agencies, academia, and industry. The partnership then aims to develop a set of models that can predict drug responses across a range of cell lines and intends to develop an approach for integrating mechanistic models and mechanism-based constraints into the machine learning framework.
Build collaborative relationships with the private sector and academia
Academic partners not only bring scientific and medical expertise, but they also can provide a richness of data tied to their patient care needs. The technology sector can help co-develop next-generation instruments and technologies at many scales that can be driven by the priority questions being tackled. Drawn together, these can be important resources for unleashing the
power of data in cancer treatment. Currently, the University of California, Intel, IBM, and General Electric have engaged DOE and NCI to build on their existing resources, technologies, tools, data, collaborations, and governance expertise to focus on the intersection of technology, cancer, and data. With this collective potential to collect, aggregate, integrate, share, and analyze vast and diverse datasets, these partnerships could enable discoveries to transform cancer prevention, detection and diagnosis, accelerate therapeutic development, and advance a patient-centered learning healthcare system. The overarching vision includes a virtual data ecosystem (drawing from the UCHealth Data Warehouse, which brings together approximately 15 million patient records), The Parker Institute for Cancer Immunotherapy, Athena Breast Health Network, and the University of California’s five NCI-Designated Comprehensive Cancer Centers, and potentially a physical co-location at the University of California, San Francisco, that unites researchers, physicians, engineers and computer scientists from academia, national laboratories, health centers and the private sector. This collaborative, synergistic framework will be a key driver for achieving precision medicine—with cancer at the leading edge. Such a regional center will serve as a powerful model, and hub, for national-scale activity.
Create a knowledgeable, sustainable, and agile biomedical data science workforce
A multi-pronged approach is needed to address the skills and workforce gaps in biomedical data science, from early education exposure to data science of those being trained in the biomedical sciences, to educating established biomedical and clinical investigators about applying computation to biomedical research questions. It is important to note that successful efforts across the federal government to achieve these goals are underway and will continue to be a priority. However, to truly support biomedical data science, the federal government needs to send a strong signal to the academic community that the development of the tools and algorithms for data analysis is a valued and “legitimate” scientific discipline. This signal should come in the form of dedicated set-aside funding for biomedical data science undergraduate and graduate education by departments and agencies such as DOE, NIH, NSF, and NCI. Federal departments and agencies involved in the Cancer Moonshot must also have the capability to recruit top talent and provide training for existing staff for developing, deploying, and disseminating novel technologies and best practices for big data analytics.
Comprehensively identify cancer survivorship issues and develop solutions to improve health outcomes for cancer survivors
The medical, psychosocial, and economic challenges facing cancer survivors are an increasingly important and complex set of issues, especially as more Americans survive cancer diagnosis and treatment. NCI estimates that there are currently 14.5 million cancer survivors in the United States. Although a body of research exists on this topic, more work remains to be done to comprehensively identify survivorship problems and solutions, and to translate those solutions into effective policies. Under the Task Force’s leadership, federal agencies, including CMS, VA, DoD, CDC, and NIH, will engage in a series of efforts related to addressing survivorship challenges. Initial priorities include hosting a summit on the topic of cancer survivorship, with follow-up activities focused on developing a framework for addressing the medical, psychosocial, and economic challenges encountered by cancer survivors and integrating relevant elements of this framework into EHR standards.