May 24, 2017
16 min read

Despite successful first year, cancer moonshot’s mission ‘nowhere near’ complete

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Five months after losing his son to glioblastoma, former Vice President Joe Biden promised to lead America’s mission to “end cancer as we know it.”

His pledge prompted the Obama administration to establish a national cancer moonshot initiative. The effort — unveiled in January 2016 — emphasized the need to make more therapies available to more patients, as well as to identify better ways to prevent cancer and detect it earlier.

“The moonshot was about fundamentally doing two things,” Biden told American Association for Cancer Research (AACR) Annual Meeting attendees in April. “[First], to inject the urgency of ‘now’ into this fight and to double the rate of progress, to do in 5 years what would normally take 10. And secondly, to change the culture and come up with a new strategy for this fight.

“I’d like to tell you we solved all the problems we identified,” Biden added. “In the time we had, we were able to get a running start, but we are nowhere near finishing the race.”

The moonshot has sparked renewed interest in cancer research and begun to break down silos that blocked the flow of data between researchers, physicians and patients, Biden said.

In December, Congress overwhelmingly passed the 21st Century Cures Act. The legislation dedicated $1.8 billion for cancer research over 7 years. Although it is no longer a White House initiative, the cancer moonshot is supported by federal funds to the NCI.

President Donald J. Trump proposed cuts to medical research funding and pledged to repeal the Affordable Care Act, prompting fears that the number of uninsured Americans could increase dramatically while prescription drug costs continue to skyrocket.

“It does not matter if there is a moonshot office in the White House,” Greg Simon, former executive director of the cancer moonshot initiative and a cancer survivor, told HemOnc Today. “It only matters if there is a moonshot office at your house.”

Greg Simon
Greg Simon

The not-for-profit Biden Foundation has launched a separate private sector cancer initiative, where Biden intends to continue his focus on support for cancer research in collaboration with the NCI initiative.

“Individuals, universities, companies, foundations, hospitals, doctors — they have all picked this up and ran with it,” Simon said. “The fact there is no coordinating function in the White House is too bad, but it does not mean the program is over.”

HemOnc Today spoke with leaders in the medical and political communities about the progress of the moonshot so far; the hurdles that must be overcome to accelerate cancer research, improve data sharing and expand trial access; and whether the effort will continue to be successful under the new presidential administration.


A ‘renewed commitment’

A 33-member blue ribbon panel — composed of experts in biology, immunology, genomics, diagnostics, bioinformatics, and cancer prevention and treatment — helped launch the moonshot and ensure it remained guided by the best science.

The panel has been charged with providing recommendations for unlocking the power of data sharing and catalyzing scientific breakthroughs to minimize the side effects of cancer treatment and improve patients’ access to care.

The panel released 10 recommendations in September. They included the creation of a national cancer data ecosystem to link the nation’s largest data repositories; the establishment of a network for direct patient involvement in clinical trials; and the intensification of research in cell biology, genomics, proteomics and drug development.

“The thing that impressed me most was how the entire cancer research community was galvanized around this moonshot,” Dinah Singer, PhD, acting deputy director of NCI and co-chair of the blue ribbon panel, told HemOnc Today. “That extended across the entire continuum of the research community, from very basic researchers to people doing transplant research, clinical research, population studies and prevention, to the advocacy community and the private sector.

A blue ribbon panel co-chaired by Dinah Singer, PhD, helped launch the moonshot and ensure it remained guided by the best science.
A blue ribbon panel co-chaired by Dinah Singer, PhD, helped launch the moonshot and ensure it remained guided by the best science. The panel recommended the establishment of a network to direct patient involvement in clinical trials and the intensification of research in cell biology, genomics, proteomics and drug development.

Photo from the NCI, by Bill Branson.

“Everybody came together around this opportunity to see what we could do to accelerate our understanding of cancer for the benefit of patients,” Singer added. “It has brought the community together and renewed a commitment to make a difference.”

That excitement was apparent at the Cancer Moonshot Summit, held June 29, 2016, at Howard University in Washington, D.C. Nearly 300 other satellite summits that day attracted more than 7,000 medical professionals across the United States, Puerto Rico and Guam.

The summit created tremendous energy around Roswell Park Cancer Institute, Congressman Brian Higgins, D-NY, and a member of the House of Representatives Cancer Caucus, told HemOnc Today.

“The vice president, borne out of a family tragedy, used his platform quite admirably to underscore the importance of cancer research toward the goal of developing new therapies that may have helped his son have a better quality of life and increased his survival,” Higgins said in an interview.


“Millions of Americans struggle with these issues every day, and they want one thing from their government — to respond with the kind of funding necessary to bring new, effective therapies with fewer side effects so that they are available for people who are suffering from cancer today and will suffer from it tomorrow,” Higgins added.

Power of data sharing

One of the most challenging undertakings of the moonshot initiative has been its attempt to unleash the power of data sharing.

Biden’s pursuit of increased data sharing stemmed from the inability of his son’s doctors in New York to share MRI scans of his brain tumor with specialists at The University of Texas MD Anderson Cancer Center due to system incompatibility, Brian Bolwell, MD, FACP, chairman of the Taussig Cancer Institute at Cleveland Clinic, told HemOnc Today.

Brian Bolwell, MD, FACP
Brian Bolwell

“Biden had to take pictures of the MRI scans with his iPhone and send them that way,” Bolwell said.

Three large initiatives have been created to promote data sharing.

Last summer, NCI launched Genomic Data Commons, a complex network that allows researchers to access cancer sequencing data. Less than a year after its launch, researchers had accessed the data commons more than 80 million times, Biden said.

The Department of Veterans Affairs and IBM Watson Health unveiled a 2-year, public–private partnership that allows Veterans Affairs oncologists access to Watson’s ability to generate reports that identify cancer-causing mutations and possible treatment options for as many as 30 times more patients than before.

And in October 2016, the Center for Computational Science Research Inc., an Illinois-based not-for-profit corporation, launched Blood Profiling Atlas in Cancer (BloodPAC), a consortium designed to accelerate the development and validation of liquid biopsy assays through data sharing between stakeholders in industry, academia and regulatory agencies.

The goal is to have seamless communication of genetic and clinical data, like imaging scans, made available to physicians to accelerate discovery. This would require the compatibility of electronic medical records (EMRs) between treatment centers.

Data from the CDC indicate 78% of office-based physicians used any type of EMR system in 2013, up from 18% in 2001.

However, CMS reported it has paid more than $30 billion in financial incentives to 468,000 providers for implementing EMRs. An additional $50.6 billion has been spent on data breaches and security threats since 2009, according to estimates from American Action Forum.

“The challenges are patient confidentiality and HIPAA laws, and the cost of the EMR right now is astronomical,” Bolwell said. “EMRs have a hard time talking to each other. As the former vice president would say, this should be something that is solvable, and we all would agree with him. The goals are certainly good, but it is still a work in progress.”


Representatives from several regional sharing networks — including ORIEN, founded by Moffitt Cancer Center and The Ohio State University Comprehensive Cancer Network; AACR’s Project Genie; and ASCO’s CancerLinQ — have begun discussing ways to share data. However, because the cancer community has operated the same way for nearly 50 years, the process required to achieve freely shared data could be lengthy, Simon said.

“We learn things every day that can help cancer patients somewhere, but not every doctor knows what has happened in the last weeks or months,” Simon said. “It is not good enough to wait until you go to the ASCO or AACR annual meetings once a year. It is important for us to have much faster sharing.

“When you think about the financial markets, if you had to wait a year or 6 months to find out what happened on Wall Street, it would be intolerable,” Simon added. We put up with that in health, and we need to ask why.”

Trial accrual

Increasing participation in clinical trials — the cornerstone of cancer research and development — has been a longstanding problem.

Historically, 3% to 6% of eligible patients with cancer enroll.

Three common reasons are the desire for other treatment (34%), distance from a cancer center (13%) and insurance denial (8%), according to a study by Lara and colleagues, published in Journal of Clinical Oncology.

To help address low accrual, the NCI — in partnership with the White House Presidential Innovation Fellows — redesigned how patients and oncologists find information on clinical trials. With the launching of an application programming interface on its central website,, third-party innovators — including Smart Patients, Syapse, Cure Forward and Antidote — have built applications, integrations, search tools and digital platforms tailored to individual communities. The platform offers clinical trial information to more providers, patients and their family members.

“The further you are from a cancer center, the less likely you are to be involved in a clinical trial,” Singer said. “One of the recommendations of the blue ribbon panel is direct patient engagement by setting up a system in which any patient who is diagnosed with cancer can access that network, have their tumor analyzed and make themselves pre-eligible for any trial. There also is a real effort to recruit more minorities into these clinical trials.”

Individuals with cancer now can log onto, enter his or her cancer type, zip code and age, and see a list of clinical trials that may be accepting new patients.


“The people who are even more overjoyed than the patients [about the website] are the drug companies, [which had been] unable to find enough people to participate in their trials,” Biden said at AACR.

Although one key to unlocking access to clinical trials lies in digital technology, another may be broadening enrollment criteria to include patients with comorbidities, according to Wafik El-Deiry, MD, PhD, FACP, deputy cancer center director at Fox Chase Cancer Center and a HemOnc Today Editorial Board member.

“We should have clinical trials that are more inclusive of the type of patient who will be treated when these drugs get approved,” El-Deiry told HemOnc Today. “We all know that pharmaceutical companies in general want to treat healthier patients to avoid interactions with the new drug they are trying to get approved with unexpected toxicities. But, why can’t we have some clinical trials directed at sicker patients or who have other illnesses?

“We would learn more, FDA approval would move faster and some patients might have access to experimental agents that they currently don’t because they don’t meet eligibility criteria,” El-Deiry added.

If higher-risk patients are included in more clinical trials, oncologists would be better able to recommend treatment or encourage patients to enroll in trials specific to their comorbidities, El-Deiry said.

“If a patient’s tumor is matched to a complex genomic profile associated with a therapeutic response, their physician should get a message and they would be referred in real time to participate in a clinical trial,” he said. “We’re just not doing this beyond a few individual genes. If I had an incurable tumor and was living somewhere in the Midwest and there was a relevant clinical trial in New York or Boston or Philadelphia, I would get on a plane and get on that clinical trial because usually the drugs are provided. But to do that, we need access to information and access to the clinical trials.”

‘Rock star researchers’

One of the strategic goals of the moonshot initiative is to catalyze new scientific breakthroughs.

Forty percent of the NCI’s annual budget supports investigative and initiative research and “it’s going to stay that way” in the coming years, Singer said.

During the 2016 fiscal year, NCI awarded 1,230 competing research project grants, which represented an overall success rate of 12% for NIH Research Project Grants (R01) and 14% for NIH Exploratory/Developmental Research Grant awards (R21).

That represents a dramatic decrease compared with 15 years ago, when 25% to 30% of grant requests received funding, Bolwell said.


“At a time when we know so much more about what causes cancer, the genomics behind it and the immunological aspects of it, the percentage of grants that are successfully funded by the NCI has actually eroded significantly,” Bolwell said. “That is a problem because the opportunity for advancement is such a big deal right now.

“The fundamental way to make progress against cancer is to learn more about what causes it, what drives it and what to do about it, and a lot of that is basic science and translational research,” Bolwell added. “That is why having accelerated federal funding for research is so important and why the risk of having that funding diminished is so great. At the end of the day, if the funding to NIH — and, thus, NCI — is significantly cut, the number of cancer discoveries is going to be cut, as well.”

In 2014, Higgins and Congresswoman Rosa DeLauro, D-CT, introduced the Accelerating Biomedical Research Act, which proposed increasing funding to NIH up to $46.2 billion by the end of fiscal year 2021. The legislation — which has not been approved — would give the House Appropriations Committee the authority to increase funding by 10% in each of the first 2 years, and by 5% in each year through 2021.

“We need to do much better when it comes to funding biomedical and cancer research,” Higgins said. “It creates jobs. When there is a reduction in funding, not only do you lose important research, you lose the rock star researchers who decide to leave the field because there is no research being funded at a significant level.”

Singer said she hopes the $300 million NCI earmarked for each of the next 2 fiscal years, along with the $400 million set aside for 2019, helps change that.

“By having a larger upfront allocation — $195 million is appropriated for fiscal years 2020 to 2022 and $216 million for 2023 — we are able to really jumpstart a lot of new initiatives,” she said.

Since January 2016, NCI has announced 14 new open funding opportunities, which can be found on its cancer moonshot website. Funding in 2018 will focus on prevention and symptom management, Singer said.

‘Prohibitively high’ drug costs

The moonshot directive to improve patient access and care has been undermined by the rising cost of prescription medication, experts said.

According to ASCO’s State of Cancer Care in America: 2017 report, the FDA approved 16 new and expanded use cancer therapies in 2016. However, one in three working-age cancer survivors incurred debt as a result of treatment costs and, of those, 55% owed $100,000 or more.


“The cost of newer cancer therapies is prohibitively high and there seems to be no moderation of it,” Bolwell said. “You are talking about hundreds of thousands of dollars per year for a new therapeutic agent for a given cancer. It is fabulous if it leads to clinical results, but the downside is that somebody has to pay that cost, whether it is an employer, an insurance company or a patient. The cost of therapy is enormously important and it continues to rise. It has virtually every leader in the cancer community concerned.”

Cleveland Clinic participates in an alternative payment model that controls costs by decreasing patient hospitalizations and increasing outpatient chemotherapy.

“All of that will not matter very much if the cost of cancer therapy is going to approach a million dollars per patient,” Bolwell said. “Both Hillary Clinton and Donald Trump talked about this issue during the campaign, and both said it had to be addressed by the federal government. We certainly hope it is.”

In June 2015, Higgins and Congressman Leonard Lance, R-N.J., introduced legislation that would require health insurance plans that cover traditional chemotherapy to provide equally favorable coverage for prescribed, orally administered anticancer medications.

Chemotherapy drugs should be treated as a health benefit, not as a prescription benefit, Higgins said.

“Why is that we do not have oral parity when it comes to cancer treatment?” he said. “We paid for the research to get us to this point where we have these drugs available. They should be available to more people who need them. Typically, more people have health insurance than they do prescription benefits. If oral chemotherapy is covered as a prescription, fewer people have insurance that covers that and, thus, don’t get it.”

Efforts should be made to provide patients with cancer drug reimbursement that “rewards innovation” while providing a “safety net” for those who cannot afford treatment, Simon said.

With 70 million Americans on Medicaid, 57 million on Medicare and 28 million with medical insurance through the Veterans Health Administration, the federal government should use that leverage to negotiate the cost of cancer medication and increase the quality of health care, Higgins said.

Meanwhile, the Affordable Care Act — which enabled 20 million Americans to gain health insurance and removed many of the deductibles associated with screening — faces an uncertain future. A CDC survey released in February showed 28.2 million Americans remain uninsured.

“If 20 million more people are going to become uninsured with a new [health care] plan, that directly correlates with whether people get screened appropriately for breast, colon and cervical cancers, the three cancers we know definitively that screening catches at an earlier stage and saves lives,” Bolwell said. “The idea of putting millions of patients at risk for enhanced mortality because they do not have access to appropriate screening is a very, very important issue that needs more national attention.”


‘Draconian’ budget cuts

In his 2018 budget, Trump proposed $5.8 billion in cuts to NIH funding, including nearly 20% in reductions next fiscal year — from $31.7 billion in 2017 to $25.9 billion in 2018.

“The president of the United States is not only not doubling down on our investment, he’s proposing draconian cuts,” Biden said. “This would set the NIH budget and biomedical research back 15 years, and that’s not hyperbole. The chance of getting a grant would almost certainly reach an historic low ... closing labs, ending careers and delaying scientific breakthroughs.”

The passage of the 21st Century Cures Act guarantees $300 million can be used by NCI in 2017, and allocation of funds in future years will require renewed appropriation by Congress each year, Singer said.

Given the bipartisan support for the bill, Singer said she is “very optimistic” the moonshot funds will be appropriated as originally planned. Simon also is hopeful.

“Congress set it up so that if you don’t spend the money on moonshot, you cannot use it for anything else,” Simon said. “So, you would have to be pretty venal not to spend it. To quote Ronald Reagan, ‘Trust, but verify.’ I think the cancer community will stay vigilant that the plan stays the plan.”

Others in the medical community are more concerned about the long-term success of the moonshot initiative under the new administration.

“We’re all worried that, no matter how you slice it, if the amount of cuts to the NIH are that large, federal funding for cancer research in general would be significantly hurt, whether that’s directly related to moonshot or not,” Bolwell said. “That obviously flies in the face of the spirit of moonshot, not to mention the fact it would significantly set us back in our quest to accelerate cancer research breakthroughs.”

El-Deiry said he was “extremely troubled” by Trump’s proposed cuts to the NIH, saying it could jeopardize needed advances and collaborations. Pharmaceutical companies, wealthy individuals and corporations would need to play a more active role in the world of philanthropy, using the “incredible resources” of the private sector to help support cancer research, he said.

“We can talk about pie-in-the-sky goals, but we need to recognize this is about the whole waterfront — including basic science — and we need to preserve that pipeline at a time of dire need,” El-Deiry said.

However, in an unanticipated move, the budget Congress passed — which expires in September — reverses Trump’s proposal, actually adding an additional $2 billion in funding for the NIH.


“This increase reaffirms the United States’ longstanding, bipartisan commitment to scientific research, and will reinvigorate the scientific community as it continues to rebuild after a decade of flat funding,” Clifford A. Hudis, MD, FACP, FASCO, CEO of ASCO, said in a statement. “It also offers hope to millions of Americans and their families who face a cancer diagnosis every year.”

Success vs. failure to launch

During his speech at the AACR Annual Meeting, Biden said the work of the moonshot is “nowhere near done,” and he will continue to lead the fight against cancer through the newly named Biden Foundation.

Experts with whom HemOnc Today spoke agreed there is plenty of room for improvement over the next 7 fiscal years, especially in the areas of prevention and diagnosis.

Whether the moonshot initiative can achieve its goal of 10 years of advancement in 5 remains to be seen. But to those who have been directly involved in the program, it has already been a success.

“I’m not sure what 10 years of progress even looks like,” Bolwell said. “Saying we’re going to accelerate 10 years of progress into 5 years is more of a metaphor than anything.

“I think the moonshot has been successful and we’re all very excited about it and see the enormous opportunity it generates,” he added. “We’re also all concerned that the funding is at risk. Ultimately, the whole goal was to get more public and private funds to try to improve research, screening, access and other initiatives to help prevent, detect and treat people with cancer. If the funding goes away, then it’s all in jeopardy.”

Higgins agreed, saying it is now up to Congress to determine the trajectory of the moonshot mission.

“We need to spend more on cancer and medical research so we can enhance the life quality of people in America,” Higgins said. “The only failure in cancer research is when you’re forced to quit because of lack of funding. Stopping and starting is not helpful. Cancer research needs to be robust and needs to be sustained over a long period of time.”

Even in the short time since the generation of the blue ribbon panel, science has matured to the point where breakthroughs are on the horizon, and the collaboration between the public and private sectors has helped accelerate progress, Singer said.

“Collaboration has always been a critical and integral part of moving science forward,” she said. “The blue ribbon panel really allowed scientists to take that to a new level, and it was very gratifying to see how people responded enthusiastically and wholeheartedly.”


The panel identified worthy and achievable goals, but progress can occur even in the absence of cure, El-Deiry said.

“Everybody wants to see cancer better controlled,” he said. “You have to be careful with the word ‘cure.’ We should aim for the cure, but if we can turn it into a chronic disease to help people live longer and utilize our knowledge to continue to make breakthroughs, our country will continue to be at the forefront of cancer prevention and treatment.” – by Chuck Gormley

Click here to read the POINTCOUNTER, “Are basket trials the most appropriate method to capitalize on increased rates of genomic sequencing?”


ASCO. State of Cancer Care in America: 2017. Available at: Accessed May 2, 2017.

CDC. Health insurance coverage: Early release of estimates from the National Health Interview Survey, January-September 2016. Available at: Accessed on May 3, 2017.

Fleming NS, et al. Health Serv Res. 2014;doi:10.1111/1475-6773.12133/abstract.

Lara PN, et al. J Clin Oncol. 2016;doi:10.1200/JCO.2001.19.6.1728.

NCI. Funding patterns. Available at: patterns/2016. Accessed May 2, 2017.

For more information:

Brian Bolwell, MD, FACP, can be reached at Cleveland Clinic, Taussig Cancer Center, 9500 Euclid Ave., mailcode CA62, Cleveland, Ohio 44195.

Wafik S. El-Deiry, MD, PhD, FACP, can be reached at

Brian Higgins can be reached at 2459 Rayburn House Office Building, Washington, DC 20515.

Greg Simon can be reached at

Dinah Singer, PhD, can be reached at

Disclosure: Bolwell, El-Deiry, Higgins, Simon and Singer report no relevant financial disclosures.