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Taking Flight – NCI Embraces Scientific Road Map to Meet Cancer Moonshot Goals

The National Cancer Institute (NCI) recently accepted the recommendations of a blue-ribbon panel on 10 scientific approaches most likely to make a decade’s worth of progress against cancer in five years under the Cancer Moonshot plan. The report was presented by the panel to the National Cancer Advisory Board (NCAB), and was subsequently considered and accepted by the NCAB with revisions that reflect NCAB’s discussion. An overview of the report was published in the journal Science.

“The bold but feasible cross-cutting initiatives in this report will improve outcomes for patients with cancer, prevent cancer, and increase our understanding of cancer,” said Dr. Douglas Lowy, NCI acting director. “NCI stands ready to accelerate cancer research in the critical areas identified by the blue-ribbon panel.”

In January 2016, during his State of the Union address, President Barack Obama announced the Cancer Moonshot “for the loved ones we’ve all lost, for the families we can still save.” The NCI’s scientific road map outlined by the panel is one component of the Cancer Moonshot’s broader effort focused on accelerating progress on the prevention, diagnosis, and treatment of cancer. Under Vice President Joe Biden’s leadership, a full set of recommendations for leveraging federal investments, private-sector efforts, patient initiatives, and more under the mission will be announced later this fall.

The 10 transformative approaches poised for acceleration aim to:

• Engage patients to contribute their comprehensive tumor profile data to expand knowledge about what therapies work, in whom, and in which types of cancer;

• Establish a cancer immunotherapy clinical-trials network devoted exclusively to discovering and evaluating immunotherapy approaches;

• Identify therapeutic targets to overcome drug resistance through studies that determine the mechanisms that lead cancer cells to become resistant to previously effective treatments;

• Create a national ecosystem for sharing and analyzing cancer data so that researchers, clinicians, and patients will be able to contribute data, which will facilitate efficient data analysis;

• Improve understanding of fusion oncoproteins in pediatric cancer and use new pre-clinical models to develop inhibitors that target them;

• Accelerate the development of guidelines for routine monitoring and management of patient-reported symptoms to minimize debilitating side effects of cancer and its treatment;

• Reduce cancer risk and cancer health disparities through approaches in development, testing, and broad adoption of proven prevention strategies;

• Predict response to standard treatments through retrospective analysis of patient specimens;

• Create dynamic 3-D maps of human tumor evolution to document the genetic lesions and cellular interactions of each tumor as it evolves from a pre-cancerous lesion to advanced cancer; and

• Develop new, enabling cancer technologies to characterize tumors and test therapies.

“Thanks to the coalescence of new scientific insights and technological innovations, cancer research is poised to make unprecedented advances,” said National Institutes of Health Director Dr. Francis Collins. “The approaches identified by the blue-ribbon panel offer exceptional promise in tipping the odds in favor of cancer patients.”

Specific Goals

In addition to the 10 scientific approaches, the road map has specific, special projects. These include a demonstration project to test for Lynch syndrome, a heritable genetic condition that increases risk of several types of cancer, to improve early detection and prevention; the establishment of a nationwide pediatric immunotherapy clinical-trials network to enhance the speed with which new immunotherapies can be tested in children; exploring patient-derived organoids; and ‘microdosing’ devices to test drug responses in living tumors.

The Cancer Moonshot scientific road map creates a vision for the future of cancer research and treatment in which:

• Patients contribute their data, obtain genomic profiling information about their tumor, learn about what treatments might work best given their tumor’s genomic profile, and find other relevant information, including clinical trials that may be appropriate;

• Researchers can identify possible targets for the development of new treatments and preventive interventions, including immunotherapies, as well as learn more about how to avoid or counter drug resistance; and

• Doctors have access to information that better predicts treatment outcomes and helps control patients’ symptoms and side effects.

The blue-ribbon panel comprises scientific experts, cancer leaders, and patient advocates convened to inform the scientific direction and goals of the Cancer Moonshot. The panel is a working group of the National Cancer Advisory Board. The Cancer Moonshot Task Force, chaired by Biden, is focused on making the most of federal investments, targeted incentives, private-sector efforts from industry and philanthropy, patient-engagement initiatives, and other mechanisms to support cancer research and enable progress in treatment and care. Lowy will share the report with the task force.

“NCI greatly appreciates Vice President Biden’s leadership of and passion for the Cancer Moonshot,” Lowy said. “The vice president has galvanized the community to move forward so we can greatly improve our ability to prevent, diagnose, and treat cancer. The efforts of the BRP and working-group members have been extraordinary, and I thank them for their time, energy, and ideas. I am confident that the cancer community will build on this effort and seize this unprecedented opportunity to accelerate progress.”