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Cancer Moonshot Recommendations Highlight Precision Medicine, Data Sharing, Genetic Screening

Joe Biden addresses attendees of the ASCO annual meeting in 2016.

NEW YORK (GenomeWeb) – A National Cancer Institute advisory board today discussed a number of recommendations for speeding the prevention, diagnosis, and treatment of cancer within a large-scale White House project.

Earlier this year, Vice President Joe Biden championed the Cancer Moonshot, a national effort to move the disease toward a cure in the next five years. Within this program, a group of researchers, oncologists, and patient advocates, the so-called Blue Ribbon Panel, have identified research opportunities that they believe could quickly bolster our understanding cancer, and improve our ability to detect it early and treat it more precisely.

After several months of discussion and considering more than 1,600 ideas from the larger cancer community, the Blue Ribbon Panel today presented 10 recommendations for improving the science and infrastructure supporting cancer research to the NCI's National Cancer Advisory Board. Several recommendations involve using molecular information to investigate the development and progression of cancer, and identify which treatments work best in patients. One demonstration project put forth by the panel seeks to enhance access to genetic testing and counseling for colorectal and endometrial cancer patients.

For example, the panel called for the creation of a federated network through which patients can get their tumors molecularly profiled, share that data, and be contacted if they qualify for clinical trials. Data in this knowledgebase could also help improve understanding of personalized treatment strategies. The panel also put forth the idea of creating a National Cancer Data Ecosystem to collect and share large datasets that doctors, researchers, and patients can contribute to the Cancer Moonshot.

"We live in an information age and we're experiencing an explosion of cancer-relevant information. This is impacting how we do our work every day and impacting how patients are cared for," Tyler Jacks, co-chair of the Blue Ribbon Panel, said at a webcast meeting today where he and others panelists presented their recommendations in a report.

"But we still need to do better in how we enable the community to input data, access data, use data, and share data," added Jacks, who is also director of the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology.

Another key data sharing tool within the Cancer Moonshot will be the Genomic Data Commons, a cloud-based platform that the NCI launched in June to make genomic and clinical data from large-scale projects available to researchers. The platform currently has data from 14,500 cancer cases from The Cancer Genome Atlas and the Therapeutically Applicable Research to Generate Effective Treatments.

However, Warren Kibbe, director of the NCI Center for Biomedical Informatics and Information Technology, said at the same meeting that recent commitments from companies like Foundation Medicine, and drawing data form other repositories and ongoing studies such as NCI-MATCH, make it likely that GDC will have information on approximately 56,000 patient cases in one to three years. Eventually, submissions from extramural research projects would likely double the amount of data in the platform. "It won't be necessarily easy to get to 100,000 [cases]," Kibbe said, "but we already see it on the horizon."

Additionally, the Blue Ribbon Panel's report called for the creation of a cancer immunotherapy trials network for adult and pediatric cancers; mapping of genetic, molecular, and cellular mechanisms that result in cancer resistance; preclinical modeling to investigate fusion oncoproteins in pediatric cancer; retrospectively studying samples from patients who received the standard of care to identify factors of response and resistance; and building three-dimensional maps of pediatric and adult tumors that document all the genetic and cellular events that lead to cancer.

The panel also felt that the Cancer Moonshot should improve cancer screening, early detection, and prevention in areas such as human papillomavirus and cervical cancer, colorectal cancer, smoking cessation, and among those with genetic predispositions for cancer. According to the panel's report, better uptake of prevention strategies could reduce deaths due to cervical cancer by 90 percent, colorectal cancer by 70 percent, and lung cancer by 95 percent. 

Toward this end, the panel has proposed a demonstration project to screen all colorectal and endometrial cancer patients for Lynch syndrome. This project would utilize NCI's existing research network and cancer centers to improve genetic testing, counseling, and access to clinical trials for patients and families.

Of the 135,000 new colorectal cancer cases in the US each year, 7,000 are due to Lynch syndrome, which is an inherited condition that increases a person's risk of cancers of the digestive and gynecologic tract and other organs. Guidelines recommend all newly diagnosed colorectal cancer patients get genetically tested for Lynch syndrome, yet fewer than 5 percent of colorectal cancer patients receive testing.

Under the demonstration project, colorectal and endometrial cancer patients would first be screened for mismatch repair deficiency, and individuals found to be potential carriers for Lynch syndrome would receive targeted sequencing to identify specific genetic mutations. First-degree relatives of these patients will also have a chance to get screened and receive counseling.

In a vote, NCI's National Cancer Advisory Board accepted the panel's draft recommendations, but the report will ultimately be reviewed by the VP's Cancer Moonshot Task Force, which in turn will issue a final set of recommendations in the fall for how federal dollars will be spent within the project. Despite their support for the panel's overall report, board members expressed reservations about specific recommendations, and questioned the ability of the government to implement the panel's ideas in an uncertain funding environment.

Advisory board member Mack Roach from the University of California, San Francisco, called the panel's report "academic," and criticized the group for failing to address known health disparities in cancer. "When we look at the data on health disparities, we've known for more than 20 years that if you're African American, you have a greater than 50 percent likelihood of dying of cancer," Roach said at the meeting. "I see this report, and I don't see disparities listed numero uno and articulated in a clear-cut way with deliverables that will impact those disparities that are real."

In issuing the recommendations, as well as the suggested demonstration projects, members of the Blue Ribbon Panel said they backed ideas that could be funded and achieved in the short term. The Cancer Moonshot began with $195 million in FY2016, and the White House has asked for another $755 million for FY2017 that hasn't yet come through.  

Given funding uncertainties in an election year, several board members backed a motion from Peter Adamson from the Children's Hospital of Philadelphia to urge Congress to provide additional funding for the Cancer Moonshot. "I don't think this board should be timid in recommending the need for appropriations," Adamson said, noting that sustained investment is critical for achieving the Cancer Moonshot goals.

"The extent and rate of implementation of these recommendations will clearly depend on congressional appropriations," admitted Dinah Singer, Blue Ribbon Panel co-chair and acting deputy director at NCI. "Although, we will continue to implement [the recommendations] to the extent that we can."

While there may be opportunities to fund proposals focused on immunotherapy or molecular testing through public/private partnerships, some of the other research projects will be more difficult to get off the ground if appropriations don't come through, panelists and board members acknowledged.

According to Singer, with the available funding, NCI will be able to start working on some of the panel's recommendations. Toward that end NCI has already identified proposals that are most feasible to implement in FY2017, she said, as well as areas where ongoing NCI initiatives might be leveraged.

The Blue Ribbon Panel also forwarded to the VP's Cancer Moonshot Task Force several policy concerns that could hinder implementation of its proposals, such as privacy and consent issues related to patient data, fragmented community care, data sharing barriers, and coverage and reimbursement challenges.

In a statement, Personalized Medicine Coalition President Edward Abrahams lauded the panel for recognizing in its report the progress in cancer care due to improving knowledge of genomics and molecular markers. However, Abrahams also cautioned that regulatory and reimbursement policies, left unaddressed, could hinder investment in personalized medicine. "As we know, unless we align public policies around promoting personalized medicine, notably in the regulatory and reimbursement arenas, progress in fighting cancer will be much slower than we would like," he said in a statement.

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