Vice President Joe Biden talking to scientists inside a Duke University School of Medicine Lab.

Vice President Biden discusses his cancer moonshot at Duke University School of Medicine earlier this year.

Ben McKeown/Associated Press

Blue ribbon report urges U.S. cancer moonshot to invest in 10 promising areas

Vice President Joe Biden’s proposed moonshot to conquer cancer should invest in large collaborations, data sharing projects, and the promising cancer treatment known as immunotherapy, among 10 areas described in an advisory group’s draft report released this morning. The report was accepted (with one abstention) today by the National Cancer Institute’s (NCI's) advisory board, which is expected to pass it on to the NCI director and then to a federal task force. Now, Congress just needs to come up with the money to pay for the moonshot, research advocates say.

“We are hopeful that these recommendations are going to be so exciting that Congress will be looking to identify paths forward to fund the cancer moonshot initiative,” says Jon Retzlaff, managing director of science policy and government affairs for the American Association for Cancer Research (AACR) in Washington, D.C., one of several cancer research and patient organizations that are praising the report.

Biden first proposed a moonshot to cure cancer last year after his son Beau died of brain cancer. President Barack Obama embraced the idea in his January State of the Union address, with Biden vowing to make a decade’s worth of advances in 5 years. To help guide the effort, a 28-member blue ribbon panel of NCI’s National Cancer Advisory Board (NCAB) then consulted with more than 150 experts and reviewed more than 1600 suggestions from researchers and the public.

The panel’s 10 recommendations, from studies of tumor biology to research on patient care (see table, below), don’t contain any big surprises. That’s because the panel was asked to look for existing research areas that were ready to be accelerated and could “produce results in the near term,” says co-chair and cancer biologist Tyler Jacks of the Massachusetts Institute of Technology in Cambridge. “However, the way these efforts are organized, resourced, and coordinated is new.”

Ten targets for the cancer moonshot

Blue ribbon panel’s recommendations for Vice President Joe Biden’s cancer-fighting plan range from tumor databases to immunotherapy trials.
Project Description
Patient network Compile patients’ tumor profiling data and preregister them for clinical trials.
Immunotherapy clinical trials network Test novel immune-based treatments for adult and pediatric cancers.
Drug resistance research Uncover mechanisms that allow cancer cells to evade previously effective treatments.
Cancer data ecosystem Link and share large data sets to facilitate discoveries that will improve patient outcomes.
Fusion oncoproteins in pediatric cancer Study abnormal proteins resulting from chromosomal translocations that drive many childhood cancers.
Symptom management research Support guidelines for routine monitoring and management of patient symptoms.
Evidence-based prevention Study ways to speed adoption of proven strategies, including screening, to reduce cancer risk.
Biopsy analysis Analyze biopsies from thousands of patients given standard treatments to learn which tumor features predict outcome.
Human tumor atlas Catalog genetic lesions and immune cell and other interactions in the tumor microenvironment for cancers of all types.
Technology Develop promising new tools that will speed testing of therapies and characterization of tumors.

Source: NCI

One thrust is a better understanding of tumors—how their cellular and genetic makeup shapes a patient’s response to drugs and how tumors evolve to develop resistance. Some researchers are already compiling this kind of information in various databases. The panel recommends creating a “data ecosystem” that would link them together. "We want something where NCI may be acting in the role of coordinating all of these databases so we can benefit from huge amount of data available but now are fragmented,” says report co-chair Dinah Singer, NCI acting deputy director in Bethesda, Maryland. 

Another recommendation is a new patient network that would allow cancer patients to submit their tumors for genomic testing and share the results and their clinical outcomes with researchers. Participants would also be able to “preregister” themselves in a database to indicate that they’re interested in enrolling in clinical trials. Right now, patients in trials tend to be those being treated at major cancer centers, and only 5% of all patients are enrolled in trials.

To make tumor profiling available to all, however, the federal Medicare program will need to revise its policies to allow reimbursement for this testing, Singer says. The blue ribbon panel passes this and several other policy issues on to Biden’s federal moonshot task force.

The panel also wants to build a completely new human tumor database that would probe not only mutations that trigger cancer in a person, but the 3D complex of immune and other cells surrounding a tumor that help sustain its growth and allow it to spread. It would be like an older NCI-funded database, the Tumor Cancer Genome Atlas, but “on steroids,” Jacks says. 

And the moonshot should consider funding a clinical trials network that would test in adult and pediatric cancer patients novel immunotherapies, which harness the immune system to fight tumors, the report says. For this, NCI expects to collaborate with the Parker Institute for Cancer Immunotherapy led by Jeffrey Bluestone of the University of California, San Francisco, who was a member of the blue ribbon panel, Singer says. That is one of many public-private partnerships that the panel envisions. “We see NCI playing a critical role in coordinating these activities but there's no way NCI could undertake the funding of all this,” Singer says.

As part of new prevention efforts, another project would test colon cancer patients for mutations in DNA repair genes, which underlie about 3% of colon cancer cases as well as some endometrial cancers. If a patient carries these mutations, family members could then be tested to learn whether they’re at high risk for these cancers. As many as a million Americans carry these mutations, but most don’t know it, Singer says. Jacks, Singer, and co-chair Elizabeth Jaffee, a cancer researcher at Johns Hopkins University in Baltimore, Maryland, discuss the report in a Policy Forum published today in Science.

One looming question discussed by NCAB today is whether Congress will fund the moonshot. President Obama requested $755 million for the moonshot in the 2017 fiscal year that begins 1 October, including $680 million for the National Institutes of Health. But lawmakers have so far not included that money in draft spending bills. Another option is that the moonshot funding would become part of other bills that aim to accelerate medical innovation, but their prospects too are uncertain.

Jacks urged NCAB members to “implore” Congress to fund the moonshot. And AACR’s Retzlaff says his group now thinks lawmakers will be ready to move forward with some type of moonshot funding, whether through a standalone bill or by adding the funding to the spending bills.

Singer said that even without new funding, NCI could begin funding some projects in the report on a small scale. And Greg Simon, executive director of the moonshot federal task force and CEO of health care investing company Poliwogg in Washington, D.C., assured NCAB members today that the report will remain relevant into the next presidential administration. “These ideas are going to survive,” he said. 

*Update, 7 September, 1:12 p.m.: This story has been updated with comments from report co-chairs and NCAB discussion.

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