Editor's note: This is Part 1 of a 2-part series on new approaches to translational medicine.
This past September, on the 40th anniversary of John F. Kennedy's proclamation of America's intent to reach the moon by the end of the decade, The University of Texas MD Anderson Cancer Center (MDACC) in Houston formally announced a new institution-wide program of attacking cancer that has been in the works for nearly a year. Called the Moon Shots Program, it is billed by the center as “an unprecedented effort to dramatically accelerate the pace of converting scientific discoveries into clinical advances that reduce cancer deaths.”
Moon Shots essentially seeks to bring together professional teams who will work, using ideas from academia and industry, to convert discoveries into tests, drugs, devices, and policies that can benefit patients as quickly as possible. The program has selected 6 initial multidisciplinary teams of researchers and clinicians that will target 8 cancers: acute myeloid leukemia/myelodysplastic syndrome; chronic lymphoblastic leukemia; melanoma; lung cancer; prostate cancer; and triplenegative breast and ovarian cancers.
Current “Ecosystem” Unsuccessful
The program will be implemented in February 2013 and is expected be fully launched by the middle of next year. Specific goals and timelines are still being developed, but one example is to measure ultimate success in lung cancer by achieving a 20% reduction in disease mortality, says Ronald DePinho, MD, president of MDACC.
Dr. DePinho, who assumed his post in September 2011, launched the program with the idea that “the path to curing cancer is in clearer sight than at any other time in history.” Furthermore, he says, “We are at a tipping point that we've never been at in the history of this field. There has been a confluence of changing technologies over the last 2 to 3 years and advances on many fronts that led us to ask if we can exploit these technologies to advance human health.”
These developments combined with the extensive resources of MDACC led Dr. DePinho to decide that the time and place was right to introduce the Moon Shots concept. He and his colleagues began by acknowledging that the current “ecosystem” of academia, biotechnology, and industry has not been very successful. Despite the many discoveries and advances that have occurred in recent years, that system still produces a 95% failure rate in cancer drug development and has been unable to develop drugs in a cost-efficient manner. At the same time, “precious few” of the thousands of prognostic biomarkers identified to date have actually been used in the clinic, and those that have—such as prostate-specific antigen—are often flawed, he says.
With that in mind, he asked the center's faculty if there were major unmet clinical needs they could identify that could be addressed using current technology and knowledge, and how they would go about dramatically reducing cancer mortality in the near term and set the stage for control of the disease within the next decade, without regard to resources or process. “That's a very different exercise than what we normally would do, which is to look at the balance of activities we have in an academic institution and make sure we have all the right things in place,” Dr. DePinho says. “To really have an impact on the disease, we had to reach across the entire beachfront, including cancer control, early detection, prognostication, treatment, and survivorship.”
A Collaborative Approach
Such a different approach required the development of new platforms that will be designed and provided with resources and leadership to ensure that teams have the necessary breadth and depth of expertise to respond to rapid changes in science and technology, according to an MDACC-issued news release. Among the platforms are:
Adaptive Learning in Genomic Medicine: Workflow that enables clinicians and researchers to integrate realtime patient clinical information and research genomic data.
Big Data: The capture, storage, and processing of huge amounts of information, much of it from next-generation sequencing machines.
Cancer Control and Prevention: Community-based efforts in cancer prevention, screening, early detection, and survivorship to educate and achieve a measurable reduction in the cancer burden.
Clinical Genomics: Infrastructure designed to bank and process tumor specimens for clinical tests that can guide medical decisions.
Institute for Applied Cancer Science: Infrastructure for development of effective targeted cancer drugs.
Institute for Personalized Cancer Therapy: Extensive infrastructure that analyzes genomic abnormalities in tumors to direct patients to the best treatments and clinical trials.
Massive Data Analytics: Computer model that develops or uses computational algorithms to analyze large-scale patient and public data.
Translational Research Continuum: Framework to facilitate the efficient transition of a candidate drug from preclinical studies to the early stages of human clinical trial testing.
What sets the Moon Shots program apart is the comprehensive and integrated aspect of the program as well as the execution element, Dr. DePinho says. “These platforms, which include industry and seasoned staff in government affairs, education, and communication, allow us to take the ball down the playing field,” he says.
“Although we initiated the program at MD Anderson, our intent is to collaborate with many institutions,” he says. “As big as we are, we can't do everything and we don't know how to do everything.” Working with colleagues at Rice University in nanotechnology or at NASA in imaging or other cancer centers on cancer control are just a few of the examples of the collaboration Dr. DePinho cites.
In the first 10 years, the cost of the Moon Shots program could reach $3 billion, which will be supported by institutional earnings, philanthropy, competitive research grants, and commercialization of new discoveries. The program will not disrupt any of the center's existing supported research, according to a news release issued by MDACC.
Part 2 of this story, which will be published in the January 1 issue, will look at additional programs combining expertise and resources to tackle specific problems in cancer.
Cancer Leaders React to MD Anderson's Ambitious Effort
• Otis Brawley, MD, chief medical and scientific officer of the American Cancer Society, is largely complimentary of MDACC's effort to translate knowledge into measurable successes in combating cancer. “We've had 10 years of flat budgets at the [National Institutes of Health] NIH, and it is becoming increasingly difficult to get funding for research,” he says. “Young and mid-level scientists are especially frustrated. I applaud any effort to actually improve morale among scientists and attract more attention and funds toward scientific research.”
• Chi Van Dang, MD, PhD, director of the Abramson Cancer Center of the University of Pennsylvania in Philadelphia, says it was to be expected that under the new leadership of Dr. DePinho, MDACC was committed to a heavy research-based leader with a strong translational bent who could “make something big happen.” Dr. Dang points out that the idea of setting ambitious goals to cure cancer is not new, but he applauds MDACC for its efforts. “I think it's important to set some goals,” he says. “At the same time, we understand we can't predict everything. Cancer biology is very complex, and we don't know where it's going to take us.”
• Eric Small, MD, deputy director of the University of California at San Francisco Helen Diller Family Comprehensive Cancer Center, says that although many comprehensive cancer centers nationwide are taking on these translational efforts, MDACC should be credited for “putting it on paper and going after this in a concrete way.” He also says he loves that they called it “Moon Shot. “ He adds, “It's not just rhetoric. It points to the fact that you have to have a goal in a very intentional fashion as opposed to just enabling good science to happen.” He agrees with Dr. Dang that there are many different potential outcomes, but he believes the goals of the Moon Shots program are reasonable and attainable. “It's aggressive—there's no question about it,” he says. “But that's good.” In addition, the publicity around Moon Shots provides “a good shot in the arm” to other centers, Dr. Small says. “It will get everyone thinking—this is how they're going to approach it. How are we going to do it?”