publication date: Sep. 7, 2016
NCI’s Moonshot Advisory Panel Identifies Ten Opportunities in Cancer Research
By Matthew Bin Han Ong
The Blue Ribbon Panel—a group of experts selected to identify scientific opportunities for the National Cancer Moonshot Initiative—has submitted 10 recommendations to the National Cancer Advisory Board.
The recommendations include creating tumor atlases and national networks for patient engagement, immunotherapy clinical trials, and data sharing, and supporting research on drug resistance, fusion oncoproteins, symptom management, and development of cancer technologies.
“The Blue Ribbon Panel recommendations outline a set of opportunities that, if implemented, will transform our understanding of cancer and result in new opportunities to more effectively prevent and treat the disease,” the authors wrote.
The 28-member panel, announced in April, consists of leaders in oncology, scientific experts, and patient advocates (The Cancer Letter, April 8).
The panel presented its report at the NCAB meeting Sept. 7. The board will, in turn, deliver recommendations to NCI Acting Director Douglas Lowy. A final report by the White House Cancer Moonshot Task Force, chaired by Vice President Joe Biden, will be produced and delivered to President Barack Obama by Dec. 31.
The panel’s report can be downloaded here.
The Blue Ribbon Panel’s three co-chairs are:
- Tyler Jacks, chair of the National Cancer Advisory Board, and director of the Koch Institute for Integrative Cancer Research at MIT.
- Elizabeth Jaffee, professor and deputy director for translational research at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University.
- Dinah Singer, acting deputy director of NCI and director of the Division of Cancer Biology.
“This is an enormous, once-in-a-lifetime opportunity for the cancer community and our nation to come together around a single disease that touches everyone,” the co-chairs wrote in the report. “The recommendations in this report represent the merger of science, technology, advocacy, social science, and big data coming together to solve cancer’s greatest challenges.
“This report reflects a combined effort of government, private industry, researchers, oncologists, patients, advocates and philanthropic organizations to identify a finite set of programs that are poised for acceleration and that could unleash new cancer breakthroughs if implemented.”
The panel’s working groups concentrated on opportunities in seven areas: clinical trials, enhanced data sharing, cancer immunology, implementation science, pediatric cancer, precision prevention and early detection, and tumor evolution and progression.
The panel considered over 1,600 ideas and initially proposed 13 recommendations, which were narrowed to 10 in the report. The 13 original recommendations can be viewed on www.cancer.gov/brp.
In addition to the scientific recommendations, several working groups identified policy issues that were beyond the scope of the Blue Ribbon Panel, but that “will need to be addressed for many of the recommendations to move forward.”
The policy issues have been forwarded to the Vice President’s Task Force for consideration. These include:
- Coverage and reimbursement
- Privacy and consent with regard to patient data
- Fragmentation of the delivery of patient care in the community
- The need to improve the clinical trials system
- Incentives to encourage pediatric drug development
- New federal research funding models
- Barriers to data sharing
A summary of the Blue Ribbon Panel’s recommendations follows:
Network for direct patient engagement. Enlist direct patient engagement through a federated network where patients will be offered comprehensive tumor profiling. Many patients are eager to provide their data, and gathering this information in a linked network of databases would enable more precise knowledge about what works, in whom, and in which types of cancer. Providing their data would also “pre-register” patients for clinical trials, enabling them or their physician to be contacted if their tumor’s molecular characteristics made them eligible for new clinical trials.
Cancer immunotherapy clinical trials network. Organize a cancer immunotherapy clinical trials network, for both adult and pediatric cancers, that would develop and implement a national strategy to discover and evaluate novel immune-based approaches, with the goal of increasing the cure rate in cancer patients and eventually develop vaccines to prevent cancers of all types.
Therapeutic target identification to overcome drug resistance. Launch interdisciplinary studies to delineate the range of genetic, molecular, cellular, and physiologic mechanisms that lead cancer cells to become resistant to previously effective treatments, with the goal of informing the development and clinical testing of new therapies to prevent or overcome drug resistance and thereby reduce deaths from recurrent disease.
A national cancer data ecosystem for sharing and analysis. Create a National Cancer Data Ecosystem to collect, share, and interconnect a broad array of large datasets so that researchers, clinicians, and patients will be able both to contribute and analyze data, facilitating discovery that will ultimately improve patient care and outcomes.
Fusion oncoproteins in pediatric cancer. Improve our understanding of the abnormal fusion proteins that result from chromosomal translocations and have been found to drive many pediatric cancers. Integral to this is a coordinated research effort that will lead to the creation of new preclinical models of these pediatric cancers, the identification of their key dependencies, and the application of this knowledge to develop novel therapeutic approaches that target their mechanisms of action.
Symptom management research. Support research necessary to accelerate the development of guidelines for routine monitoring and management of patient-reported symptoms in all care settings, throughout the cancer continuum (from diagnosis throughout survivorship and at end-of-life) and tailored to differing patient and survivor needs. Systematically gathered patient-reported outcomes data and evidence-based symptom management are needed to improve patients’ quality of life and the likelihood that they will adhere to effective treatments that are effective rather than abandoning them because of intolerable side effects.
Prevention and early detection: implementation of evidence-based approaches. Conduct implementation science research to accelerate development, testing, and broader adoption of proven cancer strategies to significantly reduce cancer risk and health care disparities. Research should focus on identifying effective, sustainable strategies that involve individuals, families, and caregivers; health care providers and systems; and the greater community. High-priority areas for which much is known about effective prevention and screening modalities are human papillomavirus vaccination, colorectal cancer screening, tobacco control, and identification of individuals with genetic predisposition to cancer, both in the general population and among medically underserved groups.
Retrospective analysis of biospecimens from patients treated with standard of care. Analyze acquired tumor samples from thousands of patients who have received standard treatments to develop hypotheses about which tumor features predict clinical benefit, treatment resistance, and other clinical outcomes. Once these categorizations are validated in clinical trials, they can be used to develop better risk stratification of cancers and allow tailored treatments to be developed for patients who are at high risk of relapse or who likely would not benefit from standard of care alone.
Generation of human tumor atlases. Create a dynamic three-dimensional map of the evolution of human tumors of all types, pediatric and adult, by documenting the genetic lesions and cellular interactions that guide the development of each tumor as it evolves from a precancerous lesion to advanced cancer while interacting with its microenvironment (including suppressing immune system recognition) to promote tumor growth, metastasis, and development of resistance to treatment. This comprehensive picture of the events and interactions that determine cancer cell behavior will help reveal the processes that underlie cancer, make it possible to predict how cancers will develop and respond to treatment, and enable the identification of new therapies for cancer treatment and new strategies for cancer prevention.
Development of new enabling cancer technologies. Support the development of promising new technologies that will accelerate testing of therapies and characterization of tumors. These include implantable micro-dosing devices for testing drug effectiveness directly in tumors; new tumor models such as organoids and other patient-derived tissue models that preserve the architecture and range of cell types seen in actual tumors; advanced imaging technologies such as single-cell mass cytometry and multidimensional fluorescence microscopy that allow proteins to be visualized within cells; new patient imaging approaches such as radiologic imaging, nuclear medicine imaging methods using new metabolic probes, and PET imaging using labeled antibodies, as well as methods that allow the results of different imaging technologies to be combined; and computational platforms that allow integration of data derived from these studies.