Collaboration in academia designed to increase return on research investments

The statistics are staggering.

More than 1.6 million US residents will be diagnosed with cancer this year, and an estimated 577,190 — about 1,500 per day — are expected to die of malignancy, according to the American Cancer Society.

At the same time, the health care system charged with treating those patients has become overrun with inefficiencies, according to a report the Institute of Medicine (IOM) released in September.

“America’s health care system has become too complex and costly to continue business as usual,” IOM officials said in a press release. “Achieving higher quality care at lower cost will require an across-the-board commitment to transform the US health system into a ‘learning’ system that continuously improves by systematically capturing and broadly disseminating lessons from every care experience and new research discovery. It will necessitate embracing new technologies to collect and tap clinical data at the point of care … and establishing greater teamwork and transparency within health care organizations.”

Two weeks after the IOM released its report, The University of Texas MD Anderson Cancer Center unveiled an initiative intended to do just that.

The Moon Shots Program — in which academia will embark on a comprehensive, action-oriented, milestone-driven effort — is designed to accelerate the pace with which scientific discoveries are translated into clinically meaningful advances, health policies and public education, according to Ronald DePinho, MD, president of The University of Texas MD Anderson Cancer Center.

“A defining and distinguishing aspect of this effort is its emphasis on execution,” DePinho told HemOnc Today. “Through the development of first-of-its-kind professional platforms capable of driving discoveries toward clinical endpoints, the cancer Moon Shots program will harness today’s game-changing technologies and knowledge already in hand to accelerate declines in cancer mortality. Like the moon shot of the 1960s, this program is goal-oriented.”

Moon Shots will begin with a core team of researchers and staff in the Houston area, harnessing the critical mass and singular focus of MD Anderson Cancer Center’s 19,000-plus employees and 7,000 trainees. The program is expected to expand rapidly through collaborations across schools, legislatures, academia and industries.

“We will be intensely collaborative,” DePinho said. “We will embrace the talent and technology of our colleagues around the Houston area, Texas, the nation and the world.”

As the health care industry renews its focus on health economics and cost containment, several academic institutions likely will undertake similar unique ventures to enhance cancer research and improve patients’ chances for survival.

“We are definitely heading into an era of dramatically increased collaboration throughout academia,” Chris Coburn, executive director of Cleveland Clinic Innovations — the corporate venturing arm of Cleveland Clinic — said in an interview. “As institutions seek to get more return on their research investments, we would expect them to work together at an even greater degree.”

Moon Shots

The Moon Shots Program name hearkens to a speech President John F. Kennedy delivered in 1962 at Rice University, in which he declared the United States would land on the moon by the end of that decade.

“Generations later, the Moon Shots Program signals our confidence that the path to curing cancer is in clearer sight than at any other time in history,” DePinho said. “I believe that we have many of the tools we need to pick the fight of the 21st century. Let’s focus our energies on approaching cancer comprehensively and systematically, with the precision of an engineer, always asking, ‘What can we do to directly impact patients?’ The program activities must span the entire cancer care continuum from prevention to early detection to prognostication to treatment and finally to survivorship.”

Moon Shots will empower six multidisciplinary teams and 10 professional platforms to convert knowledge into tests, devices, drugs and policies that can benefit patients with eight malignancies. The teams are:

• Acute myeloid leukemia and myelodysplastic syndrome;
• Chronic lymphocytic leukemia;
• Melanoma;
• Lung cancer;
• Prostate cancer; and
• Triple-negative breast cancer and high-grade serous ovarian cancer.

The program is expected to cost an additional $3 billion to $4 billion during the next 10 years, building upon the current annual research budget of approximately $700 million. About one-third of the money will come from philanthropy, one-third will come from government and foundation grants, and one-third will come from commercial revenues, DePinho said.

With regard to commercial revenues, he predicted additional revenue would come from developing and licensing more mature assets in drugs and diagnostics with the enablement of the professional platforms. Those revenues will be infused back into the program to make it evergreen. The first licensing deal, valued at a potential $335 million, has been struck between GlaxoSmithKline and the moon shot drug development platform, the Institute for Applied Cancer Science.

Each team will receive money and other resources to allow them to work on projects that will directly affect patients, including basic and translational research, biomarker-driven clinical trials, public policy initiatives and behavioral interventions.

“For example, it is not enough to say early childhood sun exposure causes melanoma in your 30s and 40s,” DePinho said. “That kind of knowledge should be converted into laws preventing kids from using tanning salons. It also requires K-12 educational initiatives, teaching children to protect themselves from the sun.”

Frank McCormick, PhD, FRS, director of the University of California, San Francisco, Helen Diller Family Comprehensive Cancer Center and leader of a review panel that selected the initial priority focus areas for the Moon Shots Program, said the initiative will yield “a collective vision on moving cancer research forward.”

“Nothing on the magnitude of the Moon Shots Program has been attempted by a single academic medical institution,” McCormick, who also is president of the American Association for Cancer Research, said in a press release. “The process of bringing this amount of horsepower together in such a focused manner is not normally seen in academic medicine and is valuable in and of itself.”

Quest for ‘the knockout blow’

Program leaders chose the eight targeted malignancies based on the current state of scientific knowledge, as well as whether MD Anderson Cancer Center had resources in place that could make an immediate impact when Moon Shots officially begins in February.

For example, researchers and clinicians are ready to deliver “the knockout blow” to CLL, DePinho said.

“A novel Bruton’s tyrosine kinase inhibitor and advances in transplantation put us in a position where we can think in terms of complete control of the disease,” he said. “We are trying to accomplish what we have done with childhood leukemia, which is helping drive that disease to one that is not completely cured but largely controlled and eradicated for those individuals. CLL is in a similar position and will receive support to bring it across the goal line.”

Other target areas likely will prove more difficult.

“There are an incredible number of challenges at every level,” Gordon B. Mills, MD, PhD, chair of the department of systems biology at MD Anderson Cancer Center, told HemOnc Today. “How do you get the patient involved? How do you perform the studies? How do you make this an inclusive program that covers everything from risk assessment to survivorship? How do you do that in a way that requires maximal and optimal amounts of high-quality data from every patient in the program?”

The key is to ensure “cross-fertilization,” Mills said.

When one Moon Shot team has a problem, members of other teams will have the opportunity to offer solutions.

Although the program initially will target eight malignancies, resources will be allocated to other areas of opportunity. They include pancreatic cancer, glioblastoma, and bladder and colon cancers. In the coming year, strategic planning efforts will be launched to develop moon shot projects for myeloma, lymphoma, sarcoma and thyroid cancer.

“While we have yet to achieve major breakthroughs that produce durable responses for patients with pancreatic cancer, glioblastoma and other diseases, the framework developed here may set the stage for such advances,” DePinho said. “Melanoma was in a similar situation a few years ago and now we are achieving apparent cure in one in four cases. For these lethal cancers, we will focus on a laboratory and clinical framework to get us closer to the goal of cures in a significant fraction of patients.”

Inside a Moon Shot

One particular area is the investigation into triple-negative breast and ovarian cancers, which are linked at the molecular level.

“We know the two most aggressive forms of ovarian cancer and triple-negative breast cancer are diseases with defects in BRCA1, BRCA2 and P53 function,” Mills said.

Prior research has shown that the prevalence of BRCA1 and BRCA2 abnormalities in germline DNA from unselected patients with ovarian cancer is approximately 11% to 15.3%, according to study results published by Hennessy and colleagues in the Journal of Clinical Oncology.

MD Anderson Cancer Center researchers plan to identify families who have the germline abnormalities in BRCA1 and BRCA2, as individuals with those abnormalities are at higher risk for developing breast, ovarian or additional cancers.

“This will be an automatic for every patient who comes in with these diseases,” Mills said. “We want an active cancer control program that reaches out to family members to help them with genetic counseling. Tests are needed to determine if they are carrying the abnormality. For those family members who are carrying the abnormality, we will offer them active early detection and cancer prevention programs. We have needed to do this, but we have never had the resources, drive and will to do it.”

Every patient with breast cancer admitted to MD Anderson Cancer Center will be characterized extensively at the molecular level, including genetic and transcriptional profiling. The research data, most of which will come from genomic sequencing, will be linked to clinical trial data. All of the data will be captured, stored and processed in a sharable platform, to which all Moon Shots researchers will have access.

“We have never been able to do this at this scope and scale,” Mills said. “Every patient will be offered entry into a program that will allow us to characterize every step and stage of their disease.”

The effort likely will begin in the Houston area, allowing researchers to identify and overcome obstacles on a smaller scale.

“If we can get to the point where we are extending this across Texas, the program can be rapidly adapted across the country,” Mills said. “It is an obvious and immediate opportunity.”

A ‘win-win’ in Ohio

Collaborative research is just one area in which advances in cancer care can be translated from the bench to the bedside.

Public–private partnerships — such as one formed between Cleveland Clinic and The Ohio State University — also can help achieve that goal.

“Public–private partnerships are not that new, but they are going to be the key to medical innovation in the future,” Brian Cummings, vice president for technology commercialization at The Ohio State University, told HemOnc Today.

Ohio is home to approximately 225 hospitals. Eleven are ranked among the best in the country by U.S. News and World Report, and another 32 met the criteria for strong performance. The majority of these high-ranking hospitals are located in two of the most densely populated metro areas in the state: Cleveland and Columbus.

“These two areas are separated by about 140 miles just up the I-71 corridor, and we wanted to find a way to get plugged into their region,” Cummings said. “If you travel that interstate, you find some of the best hospitals in the country. We felt that if we could somehow connect the two sides, it would be a win-win for everyone.”

In July, Cleveland Clinic Innovations joined with The Ohio State University’s office of technology commercialization and knowledge transfer to accelerate the commercialization of novel medical devices, patient care processes, software and consumer products.

The partnership leverages the access and capabilities of the two largest medical-related research developers in the state. In fact, the partnership created the largest medical commercialization network in Ohio, and it will better position the state to attract innovative companies and jobs, Cummings said.

“It is key to our strategic plan,” he said. “We actively look for it because it is a win-win for both sides.”

Both organizations will evaluate their portfolios and prioritize the first 20 projects on which to collaborate.

The partnership will initially focus on neuromodulation, which employs implantable devices to alter the nervous system through the use of electrical stimulation, and then will expand into other areas.

“We expect we would be tapping into some of the special competency of the university,” Coburn said.

Cancer will be one such area. The number of inventions produced by the Ohio State Wexner Medical Center and the Comprehensive Cancer Center — Arthur G. James Cancer Hospital and Richard J. Solove Research Institute has increased by 130% in recent years. Late in 2012, the university was on pace to have its best year in terms of new revenue, licenses, inventions received, and the numbers of students and faculty members engaged in new programs.

“This partnership holds enormous potential for Ohio to reshape the future of medicine,” E. Gordon Gee, president of The Ohio State University, said in a press release. “For many years, the research conducted at places such as Cleveland Clinic and Ohio State has provided the foundation for major advances in medical technology and patient care. Working together, we will be able to fully realize the potential of this innovation and research — moving more quickly to take research out of the lab and into hospitals around the world.”

Quality vs. costs

Based on current economic parameters, the estimated total cost of cancer care in the United States in 2020 will reach nearly $158 billion if disease incidence and survival rates remain the same, according to study results published online in the Journal of the National Cancer Institute.

If the cost of care increases by 2% in the initial phase and final year of care, that total cost would increase to $173 billion, a 39% increase from 2010, according to the study.

That’s why Fred Hutchinson Cancer Research Center in Seattle intends to launch an institute dedicated to improving the health economics of cancer outcomes research. The initiative, expected to begin operation this spring, is the first of its kind among cancer centers, according to its developers.

The Institute for Cancer Outcomes Research and Evaluation (ICORE) will be designed to identify and implement new business models that maintain quality of care while reducing the financial burdens of cancer for patients, Judy Nelson, project director at Fred Hutchinson Cancer Research Center, told HemOnc Today.

“What we are doing now is marshaling our resources and expanding our scope,” Nelson said. “We will be recruiting new faculty and engaging with regional stakeholders in health care and business.”

The Fred Hutchinson Cancer Research Center will provide initial funding for the program. ICORE eventually should be self-sustaining through support from research grants, philanthropy and industry contracts, Nelson said.

Efforts to prevent and cure cancer will remain at the forefront, but the institute also will focus on other areas — including cost-effectiveness of prevention, early detection and treatment clinical trials, as well as comparative effectiveness and pragmatic trial design and implementation — to ensure therapies are well selected clinically and economically.

“We are not setting up a program to simply lower costs,” Nelson said. “We are thinking about how to make the best use of the resources available so that every patient is treated with the highest of care regardless of their circumstances.”

One of the cornerstones of the project will be research databases and clinical informatics, Nelson said. Fred Hutchinson Cancer Research Center will provide foundational investments for data processing and the creation of a regional oncology databank in collaboration with NCI to identify real patterns of care among cancer patients.

“The issue of outcomes and cost-effectiveness is relevant throughout the spectrum, whether you are talking about prevention, diagnosis, treatment or long-term care,” Nelson said. “The idea is to improve the efficiency and effectiveness of cancer care for all 
patients.”

ICORE will identify and implement new models that will minimize the financial burden on patients while maintaining the quality of care, according to Scott Ramsey, MD, PhD, who has been appointed as its first director.

“The institute will serve as a new model for partnerships with all stakeholders in the cancer research and care delivery enterprise,” Ramsey said. “Success in this effort will result in broader use of evidence-based care, more efficient and effective models of health care delivery, and reducing the overall burden of cancer.” – by Anthony Calabro

References:

American Cancer Society. Cancer Facts & Figures 2012. Atlanta, GA: American Cancer Society; 2012.

Best hospitals in Ohio. U.S. News and World Report. Available at: http://health.usnews.com/best-hospitals/area/oh. Accessed on Dec. 19, 2012.

Hennessy BTJ. J Clin Oncol. 2010;28:3570-3576.

Institute of Medicine of the National Academies. Best Care at Lower Cost: The Path to Continuously Learning Health Care in America. Washington, D.C.: National Academy of Sciences; 2012.

Mariotto AB. J Natl Cancer Inst. 2011;103;117-128.

For more information:

Chris Coburn can be reached at Innovations at Cleveland Clinic, 9500 Euclid Ave., GCIC10, Cleveland, OH 44195; email: coburnc@ccf.org.

Brian Cummings can be reached at Technology Commercialization and Knowledge Transfer Office, Ohio State University, 1524 N. High St., Columbus, OH 43201; email: cummings.244@osu.edu.

Ronald DePinho, MD, can be reached at The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Houston, TX 77030.

Frank McCormick, PhD, FRS, can be reached at University of California, San Francisco, Helen Diller Family Comprehensive Cancer Center, Box 0128, UCSF, San Francisco, CA 94143-0128; email: director@cc.ucsf.edu.

Gordon B. Mills, MD, PhD, can be reached at the Department of Systems Biology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Houston, TX 77030.

Judy Nelson can be reached at Fred Hutchinson Cancer Research Center, Department of Public Health Sciences, 1100 Fairview Ave. North, P.O. Box 1902, Seattle, WA 98109-1024; email: jnelson@fhcrc.org.

Scott Ramsey, MD, PhD, can be reached at Fred Hutchinson Cancer Research Center, 1100 Fairview Ave. North, M3-B232, P.O. Box 19024, Seattle, WA 98109-1024.

Disclosure: Coburn, Cummings, DePinho, McCormick, Mills, Nelson and Ramsey report no relevant financial disclosures.

Will innovative partnerships benefit from an infusion of research funding?

Private-public partnerships are a very good option.

There are certain realities that exist in 2013. The first is that overall reimbursement in health care is declining and will continue to do so. Everyone in health care will be paid less for what they do. Second, given the current state of the budget deficit, federal funding for grants will not increase and may very likely decrease.

For any major academic medical center, covering research costs is going to be an increasing challenge. Academic centers will need to be creative at finding alternative revenue streams to try to maintain their academic missions. One of those ways may be philanthropy. Some academic centers do that very well. But depending on how the economy fluctuates, philanthropic support is a variable that is difficult to predict.

Because the cost of clinical research continues to rise, in part due to the regulatory scrutiny that exists, it is very difficult for many private oncology groups to offer clinical research to their patients. They simply do not have a practice model to cover the costs associated with clinical research and, therefore, are frequently anxious to partner with an academic cancer center to be able to offer these services to their patients. These private oncology groups are willing to pay for that service.

From an academic medical center perspective, there are several possible benefits. The first is the more that we increase our opportunities and capacities to accrue patients to clinical research trials, the better. That’s a very important part of our academic mission. If we can use these partnerships to maintain our academic mission, then it is likely worthwhile.

Second, these partnerships do create a revenue stream to the academic center to help defray some costs associated with research. Frequently, these partnerships have agreements that include a variety of other components besides research, so the revenue back to the academic center covers additional organizational parts, but some revenue does support research.

We are all going to have to be creative in finding additional ways to conduct both basic translational and clinical research. I think these partnerships represent a very real and viable option to assist research costs and maintain our academic mission.

Brian J. Bolwell, MD, FACP, is chairman of the Taussig Cancer Institute at Cleveland Clinic. He can be reached at Cleveland Clinic Main Campus, Mail Code R32, 9500 Euclid Ave., Cleveland, OH 44195; email: bolwelb@ccf.org. Disclosure: Bolwell reports no relevant financial disclosures.

These partnerships are not so much financially driven as they are driven by the nature of the field.

The world has changed. The science and technologies we need to develop a comprehensive picture of cancer is often outside the capacity of any one institution. Collaborations are becoming more common because they are science-driven.

I see innovative partnerships as an opportunity, but also as an obligation. The public, the NCI and the NIH expect these essential collaborations, which enable us to bring cancer discoveries into the clinic. When they are driven only by financial factors, you have a much higher chance of failure because the entities involved may have different goals and different cultures, and you are trying to blend different interests. If these collaborations are strictly financially driven, you also encounter problems managing conflict of interest on the key issues.

Clearly, there are many different types of collaborations. Some of them, like partnerships between pharmaceutical companies and academic institutions, are often both scientifically and financially driven. Academic institutions are looking to pharma for components of drug development that we can’t accomplish and are not really the forte of universities. At the same time, pharma is looking to us for the clinical relevance, for identifying targets, validation and — of course — clinical trials.

In those cases, there are financial ramifications because academia cannot conduct these studies without financial support from pharmaceutical companies. This is where managing conflict of interest becomes a high priority. Most institutions have done a very credible job of developing firewalls and creating strong guidelines that prevent those individuals in a position of authority from unduly influencing the conduct of research, including clinical trials.

Michelle M. Le Beau, PhD, is director of the Cancer Cytogenetics Laboratory at University of Chicago. She can be reached at The University of Chicago, AMB H212Q (MC 1140), 5841 S. Maryland Ave., Chicago, IL 60637; email: mlebeau@medicine.bsd.uchicago.edu. Disclosure: Le Beau reports no relevant financial disclosures.