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Cancer Moonshot has diagnostic thrust

October 2016—Vice president Joe Biden’s Cancer Moonshot now has a flight plan, drafted by a blue-ribbon panel and published in September. Coming as it does in the final year of president Obama’s term in office, there are doubts about whether the ambitious $1 billion program—aimed at achieving 10 years’ progress in cancer research and treatment in a five-year period—will ever get off the launching pad.

Nonetheless, two pathologists involved with the initiative say it has already spurred creative thinking about how to break down silos within the cancer community and reinforced the central role diagnostics will play in detecting, preventing, and better understanding cancer.

Dr. Downing

James R. Downing, MD, a pathologist and president and CEO of St. Jude Children’s Research Hospital, was invited in April to serve as one of the 28 members of the blue-ribbon panel. He has long investigated the genetic basis of cancer and was the corresponding author of a New England Journal of Medicine study that uncovered new information about the role that genes associated with cancer predisposition play in childhood cancer (Zhang J, et al. 2015;373[24]:2336–2346).

Dr. Downing says the broader cancer community submitted more than 1,600 ideas for the panel to consider. Over the course of many face-to-face and virtual meetings, the panel—with the help of seven working groups composed of more than 120 physicians, scientists, patient advocates, and other experts—narrowed that mammoth catalog of ideas to a relatively targeted list of 10 high-impact recommendations. The report, available in full at bit.ly/moonshotreport, recommends the federal government fund efforts to:

• Establish a network for direct patient involvement.
• Create a clinical trials network devoted exclusively to immuno-therapy.
• Develop ways to overcome resistance to therapy.
• Build a national cancer data ecosystem.
• Intensify research on the major drivers of childhood cancers.
• Minimize cancer treatment’s debilitating side effects.
• Expand use of proven prevention and early detection strategies.
• Mine past patient data to predict future patient outcomes.
• Develop a three-dimensional cancer atlas.
• Develop new cancer technologies.

“Pathologists, and other people in the field of laboratory medicine and diagnostics, need to be participants across many of the 10 recommendations,” Dr. Downing says. “In cancer immunotherapy, a tremendous amount needs to be learned about why some patients respond and some don’t. What is the mutational burden? What is the composition of the immune system? And some of that work will be tissue-based analysis. And then there is monitoring response to cancer immunotherapy.”

Pathologists also would be key to the 3-D atlas of cancer, which requires profiling tumors and understanding how they vary over time. And, as long-time experts in informatics, pathologists could prove invaluable to “this idea of developing an ecosystem for sharing and analyzing data,” Dr. Downing says.

“Pathologists have a unique skill set and experience and a viewpoint that can play a role in many of these investigational studies,” he adds. “Within precision medicine—how do we treat more specifically for individual tumors—is buried a lot of genomic analysis, immunophenotypic analysis, tissue environmental analysis, and understanding those dynamics with those tumors. Out of this work will come new diagnostics and treatments.”

Dr. Downing lauds the report’s call to fund more work on the fusion oncoproteins that drive many childhood cancers and turn them from “undruggable” to targets for new drugs. Such research could yield big insights that “would spread into adult cancer,” he says.

“The blue-ribbon panel was instructed not to think about dollars and cents at all,” Dr. Downing says. “We were asked to think about what are the biggest opportunities, irrespective of what they would cost. So we did the exercise without looking at the potential total dollars invested in this, or the reality or likelihood of money being invested in it.”

Of the $1 billion in funding outlined, $195 million was redirected from the National Institutes of Health as part of its 2016 budget. Obama proposed another $755 million of the total as mandatory funding for the NIH and the FDA as part of his 2017 budget, but Capitol Hill watchers say the likeliest scenario—even after the elections in November—is that Congress will pass a continuing resolution that will forgo any new spending.

Dr. Downing says he does not see the moonshot program’s blue-ribbon panel “as an exercise in futility” because it has brought together “a serious group of individuals, representing a broad swath of research, advocacy, and others involved in cancer to think about approaches that, if pursued, could really accelerate progress.”

“My experience over the last 30 years,” he adds, “is that not all funding can come from the federal government. With these kinds of exercises, you can look at what needs to be accomplished and not just what we can accomplish. There is a benefit to having thought leaders focus on these kinds of efforts.”

Aside from the central question of whether Cancer Moonshot will get the funding envisioned, another uncertainty is whether the plan’s reach exceeds its grasp, says Keri Donaldson, MD. He directs the CLIA laboratory at the Penn State Institute for Personalized Medicine and was invited to participate in one of the moonshot task force’s public meetings, held June 29 at Fox Chase Cancer Center in Philadelphia.

“If you look at the primary goals of the moonshot, some of them are pretty lofty,” says Dr. Donaldson, who also directs the clinical processing specimen laboratory at Milton Hershey Medical Center and is an assistant professor of biochemistry and molecular biology at Penn State. “Whether they are attainable or not is a reasonable question.”