publication date: Sep. 9, 2016

NCI’s Moonshot Advisory Panel Identifies Ten Opportunities in Cancer Research

By Matthew Bin Han Ong and Tessa Vellek

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 panel proposes creating tumor atlases and national networks for patient engagement, immunotherapy clinical trials, and data sharing. Recommendations also include 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 write.

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 accepted the report, forwarding it to NCI Acting Director Douglas Lowy. The vote was unanimous, with one abstention. 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.

“Thanks to the coalescence of new scientific insights and technological innovations, cancer research is poised to make unprecedented advances,” said NIH Director Francis Collins in a statement Sept. 7. “The approaches identified by the Blue Ribbon Panel offer exceptional promise in tipping the odds in favor of cancer patients.”

Implementation of the panel’s recommendations over the next two years—and by extension, the moonshot effort—will depend on Congressional support and an increase in appropriations, said NCI Acting Director Douglas Lowy, who will share the report with the task force.

“The way in which the recommendations are implemented will depend on how much funding we receive for the implementation,” Lowy said in an interview with The Cancer Letter. “If there is no funding, clearly, we would only be able to start on a small part, with perhaps some pilot projects.

“If there is substantial funding, then we could think about implementing the majority of the recommendations during FY17 and the others during FY18.”

A conversation with Lowy appears on page 1.

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

These recommendations will be publicly available when the task force releases its report in mid to late October, said Greg Simon, executive director of the White House Cancer Moonshot Task Force.

“The White House Cancer Task Force is involved in internal government deliberations on a variety of issues, and the Blue Ribbon Panel’s policy recommendations in several different areas are issues in which many are already being discussed and some were new from the Blue Ribbon Panel,” Simon said in a Sept. 7 press call. “The task force report, when it is released, will address not just policy issues, but a lot of programmatic initiatives at the agency level—some of which also involve private sector.”

“The policy recommendations from all 20 agencies will be included in the report. Every agency has policy issues in their sphere, so we have not tried to release in piecemeal but to deal with them all at once, which you will see in the task force report.”

 

NCAB Members: What About Health Disparities?

At its meeting Sept. 7, NCAB praised the panel’s hard work, but several members questioned its method for selecting and prioritizing its recommendations.

“I brought up numbers, which I strongly recommend be inserted as much as possible in this recommendation, because they’re really not,” said Electra Paskett, Marion N. Rowley professor of cancer research and director of the Division of Cancer Prevention and Control at The Ohio State University. “We owe it to the public to tell them how many cases we potentially could impact by doing this. We have to put a personal face on this report and not make it about tissue and data and drugs.

Paskett urged the co-chairs to amend the document.

“Could you please possibly insert a few words for my population science colleagues so they are not left out?” Paskett said. “The other important factor… I don’t think health disparities has been done enough justice in this report, and it would be very easy, important, and we owe it to our population to add a concern about health disparities.”

Mack Roach, professor of radiation oncology and urology and director of the Particle Therapy Research Program at the University of California—San Francisco, abstained from NCAB’s vote to accept the report.

The report—which mentions health disparities eight times over 72 pages—does not explicitly list the issue as one of the 10 recommendations.

“I think disparities should be sitting top, front, center,” Roach said. “I was there when Biden got up and talked about the urgency to get 10 years of work in five years. And why was that? Because people are dying who don’t need to be dying. The low-hanging fruit are the people who are dying from reasons we already know about.

“I think that when we look at the data on health disparity—we have known for more than 20 years that if you are African American, you have more than a 50 percent likelihood of dying of cancer. And I see this report and the summary, and I don’t see disparity listed Número Uno on the report. And articulated in a clear-cut way with deliverables that will impact those disparities that are real. I could go on, but I’m not.”

Other members noted that health disparities are a complex problem.

“I don’t think, Mack, that it’s actually the job entirely of the NCI to solve the disparities problem,” said Judy Garber, director of the Center for Cancer Genetics and Prevention at Harvard Medical School. “I think this is a Congressional issue—we aren’t without responsibility, but we are unable on our own to solve this problem entirely.

“And I think disparities absolutely belong front and center in this document, but no amount of money going to the moonshot is going to fix all of the disparities problem. Yet disparities have to drive progress.”

Health disparities will be addressed at the task force level, not through the NCI or the Blue Ribbon Panel, Simon said.

“We don’t look to the NCI scientific recommendations to deal with disparities, so much as we do the issues of access and distribution of therapies through community centers and a gap between cancer centers and community centers,” Simon said. “In terms of people’s access to clinical trials, to novel therapies, to extraordinary uses of unapproved therapies—all of which relate to access and disparities—but there has been a focus in the task force on the issue, which was written up just the other day, of place as a surrogate for survivorship, even more than race, and the fact that we have such geographic disparities in survivorship and opportunities for standard of care. That is definitely a focus of the task force.”

Other NCAB members had questions about the report’s recommendations on research methods—in particular, retrospective analysis of biospecimens of patients receiving standard-of-care treatments.

“I think resources will be diverted from an area where they could be more useful,” said Max Wicha, deputy director of the Taubman Institute, distinguished professor of oncology, and professor of internal medicine at the University of Michigan. “I think [retrospective analysis] is not that likely to produce much in the way of fruitful information compared to a detailed analysis of patients on standardized therapies in clinical trials in which one can make useful comparisons of the biology.”

 

Simon: A “Breakthrough Research Panel”

Simon said that the report could not—and should not—include all cancer-related goals.

“This panel went from being a Blue Ribbon Panel, which has a certain knee-jerk reaction in a lot of people’s minds of ‘I don’t want anything to do with it,’ to what I call a Breakthrough Research Panel,” Simon said. “The speed and quality with which they worked in an already full workday environment, working with very busy people to get them to spend hours a week doing something quickly and well is really commendable.

“We try not to confuse moonshot projects as being the total sum of all projects undergone in the cancer enterprise at large in the country,” Simon said. “We are trying to reach in and pull out the things that are truly moonshot leaps that have not had the attention they should have had, maybe haven’t had the funding they should have had, or the collaboration they should have had.”

The panel should be cautious when classifying issues into moonshot-worthy and not-moonshot-worthy projects, Garber said.

“I’m concerned about taking the Blue Ribbon Panel recommendations, the work here for the Moonshot, and dividing life into Moonshot/non-Moonshot,” Garber said. “That’s very good for some practical aspects of this, but it does not begin to solve the cancer problem in the way the vice president talked about it.

“My concern is that in the efforts to focus on bringing the moonshot to reality, that we not compromise on all of the other work that goes on in the NCI. This was supposed to be extra money. If it turns out not to be extra money, it will be a challenge to figure out how to use this to make sure that we are not neglecting all that important population science, basic work, clinical work, and still chase what here are great opportunities that have been laid out by very thoughtful people.”

Federal agencies are likely to face a short-term continuing resolution at the end of the fiscal year, and funding can be uncertain in a lame duck year for both Congress and The White House.

After accepting the report, members approved a motion for the NCAB to urge increased and sustained appropriations to ensure the success of the moonshot and Blue Ribbon Panel’s recommendations.

“There are no appropriations right now and we don’t know where appropriations will go,” said Deborah Watkins Bruner, the Robert W. Woodruff chair of nursing at Nell Hodgson Woodruff School of Nursing and associate director for outcomes research at Winship Cancer Institute at Emory University. “So should there be no appropriations? What could be salvaged from this fantastic intensive effort?

“I heard one comment about pharma being interested, but what were the thoughts about if we get no more money, what can we do with this report?”

Jacks, Lowy and Simon responded:

Jacks: Dollars from the Congress are critically important in my view. I don’t really know where the dollars will shake out, but I’m going to assume that we will in fact successfully engage with industry through various means, but including through funding, that will enhance our opportunities in these areas, so I hope that’s true.

Lowy: On some level, we have just received the Blue Ribbon Panel recommendations today, so we don’t have a fully fleshed out plan depending on how much the appropriations—whether they remain the same for FY17, go up, and by how much … My view is that this is a compelling set of recommendations and I share Tyler’s cautious optimism that this will translate into an increase in the appropriation for FY17. If we didn’t get one, it’s not that we wouldn’t be able to start anything, it’s just that the size, scope, and speed would be dramatically different.

Simon: Let me quote one of my favorite literary characters, who says, ‘With enough ifs, you can put Paris in a bottle.’ Think about that for a second … The power of the ideas that are represented here will not rise or fall based on any one-year appropriation. And we are in one of those bizarre moments where you have the end of one administration and the beginning of another, and whatever is true in November and December will not be true in January and February. It is 52-card pickup with the budget.

Copyright (c) 2017 The Cancer Letter Inc.