The American Society of Clinical Oncology has released a sweeping report setting forth a new vision for cancer research incorporating the latest findings from molecular biology.
The report, “Accelerating Progress Against Cancer: ASCO's Blueprint for Transforming Clinical and Translational Research,” was introduced during a news telebriefing timed to come out 40 years after President Nixon signed the National Cancer Act in December 1971.
“Cancer science is now in a period of revolutionary change,” said ASCO President Michael P. Link, MD, the Lydia J. Lee Professor of Pediatric Cancer at Stanford University School of Medicine. He added that right now, the nation's cancer research system is not equipped to take full advantage of science in the molecular era, which has revealed multiple pathways with mutations that potentially could yield to ever more targeted therapies.
Dr. Link added that while there has been remarkable progress in the last 40 years—including curing nearly 80% of all children with cancer—”we still have a long way to go.”
He noted that progress against cancer has not been uniform, and that while major advances have been made in the treatment of breast, prostate, and colorectal cancers, treatment of pancreatic, lung, and many advanced cancers has been far less effective.
Dr. Link said ASCO advocates a new approach to cancer research based on an increased understanding of the biology of cancer, the potential of targeted therapies, and the full use of health information technology (HIT), which he described to date as “limited, uncoordinated, and inconsistent.”
The key question for cancer researchers to ask themselves in the molecular era, he said, is, “Where do we go from here?”…Our aim here is to lay out the first initial steps and then see them through.” And, he said, this approach could be important not only for the survival of cancer patients but also for the United States economically: “Smarter therapy is ultimately going to be cheaper therapy.”
3 Key Areas
ASCO's blueprint vision for transforming cancer research doesn't attempt to predict a clear path toward future progress against cancer, but can be broken down into three key areas, noted Neal J. Meropol, MD, one of the report's three oncologist executive editors, along with Mark G. Kris, MD, and Eric P. Winer, MD.
1.Establishing a new approach to therapeutic development driven by a firmer understanding of cancer biology. This new approach will identify and rank by priority molecular targets that have the greatest promise to improve survival in cancer patients, said Dr. Meropol. Such an approach will provide incentives for collaboration between industry and academia to discover therapies and diagnostics used in combination.
“It's the combination treatments that are gong to be increasingly essential,” said Dr. Meropol, Chief of Hematology and Oncology at University Hospitals Case Medical Center and Case Western Reserve University. The new therapeutic approach will ensure aggressive and timely development of key biomarkers in tissue, blood, and other bodily fluids, he said.
2.Designing smarter, faster clinical trials to provide results targeted to specific patient groups in a timely manner. Dr. Meropol said cancer researchers need to shift away from the old paradigm of “trials that promise only marginal improvements in care” toward those that address “clear unmet needs….Today's trials are often me-too trials.”
He said ASCO envisions flexible trial designs that allow researchers to demonstrate efficacy with smaller and smaller populations defined by their specific molecular biology. Selecting study subjects based on molecular characteristics, he said, will ensure that only those who are likely to benefit from a given therapy are included in a trial, and that patients are not excluded from a trial due to irrelevant health conditions.
A new concept is emerging, noted Dr. Meropol: the idea that “all cancers are becoming rare cancers,” due to the identification of more and more subsets of a specific cancer. This concept will lead to “smaller studies with fewer patients in them,” he said.
A major aspect of this part of ASCO's transformative vision pertains to the ongoing revitalization of the Clinical Trials Cooperative Group Program of the National Cancer Institute. He said ASCO strongly supports the recommendations by the Institute of Medicine in 2010 (OT, 5/10/10 issue) aimed at updating and reinvigorating NCI's cooperative groups, and that the ongoing reorganization and consolidation of cooperative groups has “really improved the coordination of our national portfolio” and “stimulated an opportunity to integrate science into the cooperative groups.”
3.Harnessing advances in health information technology to integrate clinical research with patient care. Dr. Meropol said ASCO's vision includes using electronic health records (EHRs) and rapid learning systems to use real-world scientific findings for better cancer treatment in the clinic. He noted that harnessing the potential of HIT will involve standardizing EHRs so that information is not scattered in unconnected computer systems, developing industry standards for storing and capturing important information from biospecimens, and ensuring patient privacy.
“The patient does not want to repeat their medical history every time they go to a new doctor,” said Dr. Meropol. “We need an agreed-on platform so [HIT] crosstalk can occur.” He said ASCO will be working over the next three years to use HIT to connect to a central knowledge base.
“This is not a new topic; what's important is that this initiative is coming from doctors,” said John Mendelsohn, MD, Chair of the IOM's National Cancer Policy Forum and Co-Director of the Khalifa Institute for Personalized Cancer Therapy at the University of Texas MD Anderson Cancer Center, who also participated in the telebriefing.
Dr. Mendelsohn, who chaired the IOM's Committee on Cancer Clinical Trials and the NCI Cooperative Group Program, said he strongly supports the report's emphasis on treatment based on an understanding of cancer genes and its emphasis on clinical trials based on each patient's tumor characteristics. “I see more clinical trials that involve an adaptive design,” he predicted.
This new approach could help to encourage young scientists to become clinical investigators, a career choice that is becoming more and more difficult. Currently, “the regulatory sphere has become very risk-averse,” said Dr. Mendelsohn, who added that it is important to let cancer patients decide what level of risk they want to take. He said he agrees with the ASCO report that HIT “needs standardization and interoperability.”
Research collaboration—as advocated in the new ASCO blueprint vision—is considered controversial in a field that has been dominated by fierce competition. But, said Dr. Mendelsohn, “it's finally beginning to happen. What's driving it is economics; there aren't any blockbusters.”
He said pharmaceutical companies are now starting to realize how collaborative research can be a good investment for them. Asked by OT if there are enough incentives for pharmaceutical companies to develop cancer drugs for ever-dwindling subgroup populations, Dr. Mendelsohn cited the success of imatinib for chronic myelogenous leukemia, a targeted drug that proved highly profitable though directly at a small-population indication even before the drug was found to be effective and approved for the other indication of gastrointestinal stromal tumors (GIST).
Cancer survivor Ruth Rechis, PhD, Director of Research & Evaluation for LIVESTRONG and a clinical trial participant—who also took part in the telebriefing—said LIVESTRONG applauds the new ASCO blueprint for transforming cancer research. In addition to focusing on the value of better-designed clinical trials that yield results faster, she said, she is happy the report stresses the potential of HIT to improve cancer patients' lives.
Dr. Rechis, who was diagnosed with Hodgkin's lymphoma as a teenager, said, “I have spent countless hours in doctors' offices repeating my health history,” a history primarily contained in stacks of paper records.