BY PEGGY EASTMAN

WASHINGTON, DC -- Even though intense funding pressure threatens continued major scientific advances against cancer, potential opportunities in targeted diagnosis and treatment could turn the disease into a manageable, chronic condition within the next decade, according to a new white paper released at a national cancer conference here.

Based on interviews with more than 30 recognized leaders in the cancer field, the white paper, “Sustaining Progress Against Cancer in an Era of Cost Containment,” was developed by Feinstein Kean Healthcare and distributed to attendees at the “Turning the Tide Against Cancer Through Sustained Medical Innovation” conference, a meeting convened by the American Association for Cancer Research,  the Personalized Medicine Coalition and Feinstein Kean Healthcare.

The document sets forth specific pathways to achieve the goal of turning cancer into a chronic disease in the era of increased understanding of cancer’s molecular underpinnings. These pathways include:

• Greater use of molecular biomarkers in the clinic.
• Use of molecularly targeted therapies in combination to form “personalized treatment cocktails” based on each patient’s genetic makeup and cancer biology.
• Evidence-based physician/patient decision making.
• Development of regulatory and reimbursement policies to advance and enhance targeted therapies.

 Most of the 30 leaders interviewed said that they are hopeful about making continued progress against cancer based on rapid increases in scientific knowledge.

© Max Taylor

Similarly the conference speakers: “The field is robust,” said John J. Castellani, President and CEO of the Pharmaceutical Research and Manufacturers of America, speaking of cancer drug research and development. Right now there are nearly 1,000 compounds in various stages of development for cancer, he noted, and what is needed is a business model to attract capital.

Today, the goal is to match cancer patients to a targeted therapy based on their genes, ideally in a clinical trial, said white paper contributor

 © Max Taylor

John Mendelsohn, MD, now Director of the Khalifa Institute for Personalized Cancer Therapy at the University of Texas MD Anderson Cancer Center, Chairman of the National Cancer Policy Forum of the Institute of Medicine, and a member of the conference’s advisory committee.

Citing the development of imatinib, olaparib, gefitinib, and crizotinib, he said he realized the potential for targeted therapies back in 1980 when he started working on the EGF receptor, known to be over-expressed in a number of cancers.

And while he is optimistic about the development of new targeted cancer therapies, he noted that a number of complex challenges must be faced to keep the stream of tailored-therapy discovery flowing strongly.

These include: the heterogeneity of cancer; the plasticity of cancer cells; the need to distinguish driver from “passenger” genetic abnormalities; the need to identify molecular and genetic influences in the cancer and in the patient that can modify responses to targeted therapies; and regulatory obstacles, including what he termed a risk-averse US Food and Drug Administration. Currently, “We do not know how to kill a cancer cell; it’s not just apoptosis,” Mendelsohn said.

© Max Taylor

Luncheon speaker Siddartha Mukherjee, MD, author of the Pulitzer Prize winning book The Emperor of All Maladies: A Biography of Cancer, and Assistant Professor of Medicine at Columbia University, agreed that the complexity of cancer remains a major hurdle in scientific discovery of new medicines. “Not only are there many genes, but there are many combinations of genes,” he said, using the term “combinatorial diversity” to describe this complexity.

He cited one of his patients, who had two cancers in the same breast – each genetically different, and each requiring a different treatment.

Collaborate and Share Data

Mendelsohn struck a theme that was stressed repeatedly throughout the day-long conference: the need for cancer researchers to collaborate and share data. “The clinical trial process is very complicated; this is a team sport,” he said. “We all hold onto our data and we don’t want to share it.…We have to share data and the methodology used to get that data.”

Continuous, Rapid-Learning

In addition to the need for greater collaboration and data sharing to move cancer diagnosis and treatment forward, a second key theme stressed in the white paper and at the conference was the necessity of a continuous, rapid-learning health care system. Such a system, advocated by the Institute of Medicine and by the American Society of Clinical Oncology, “links research and care into a seamless process,” in the words of the white paper, a process that fosters the rapid use of new evidence-based knowledge in the clinic.

A rapid-learning health care system relies on advances in information technology to compile evidence from a number of sources to deliver the best, targeted therapy to each cancer patient.

“You need to learn in the course of care; each patient you treat informs the care of the next patient,” said white paper contributor

© Max Taylor

Laura Esserman, MD, MBA, Professor of Surgery and Radiology at the University of California, San Francisco, Director of the Carol Franc Buck Breast Care Center, and Co-Leader of the Breast Oncology Program at the UCSF Helen Diller Family Comprehensive Cancer Center.

“This is the kind of thing patients want us to do, and we need to get to it,” added Esserman, a member of President Obama’s council of advisors on science and technology Working Group on Advancing Innovation in Drug Development and Evaluation and Principal Investigator of the I-SPY Trial program. I-SPY, a multi-site neoadjuvant clinical trial, has evolved into a model for translational research and adaptive trial design.

Patient-Centered

A third major theme emphasized in the white paper and during the conference is that cancer care must always be patient-centered -- that is, patients’ needs and values must be placed at the center of care, informing clinical decision-making. This emphasis is especially important in this time of fiscal restraint, said conference speakers.

One option when money is tight is to say to a prostate cancer patient, for example, “Here is $100,000; you and your doctor can decide how to spend it,” suggested

© Max Taylor

Lee Newcomer, MD, MHA, Senior Vice President for Oncology at UnitedHealthcare.

Asked by OT to comment on this approach,

 © Max Taylor

J. Leonard Lichtenfeld, MD, another white paper contributor and a member of the conference advisory committee and Deputy Chief Medical Officer of the American Cancer Society, said, “Dr. Newcomer is saying this to be thought provoking. We’re going to get to a point where we have to address the business case for cancer care….You hear about personal values; what do people want? We’re sort of now in middle land. If we want to move toward a more rational system, we’re going to have to make some choices.”