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Precision Cancer Medicine by Functional Biomarkers

May 19–20, 2017

Chaired By

 Anthony Letai MD, PhD of Dana-Farber Cancer Institute

Meeting Description

With the ever-growing number of targeted cancer therapies comes the growing need for predictive biomarkers to assign these therapies to the patients who will most benefit from them. This is the task of precision medicine. While the precision medicine in cancer is often equated with cancer genomics, there are important and increasingly appreciated limits on how well genomic information can serve as a precision medicine tool in cancer. An emerging alternative strategy that is very important to my laboratory and others is to put drugs of interest into contact with the patients’ actual tumor cells and measure the effect. Exactly what is measured and how it is measured differs depending on the approach.

Meeting Summary

It would be ideal if we knew that the drug treatment used for an individual’s cancer would work and kill the malignant cells. To achieve this, we need markers that are associated with a disease that also give you information as to how a tumor responds to therapy. To date, the overwhelming majority of effort has focused upon identifying genetic markers that are thought to help in predicting the outcome. However, most patients’ tumors lack these types of genetic markers so that an effective therapy can be assigned. An alternative method is to apply drugs directly to a biopsy of the patient’s cancer cells and make relevant measurements that can predict response to therapy. This meeting gathered 18 individuals who shared an interest in making such technologies work. Several Forbeck Foundation SAB members also attended as observers.

The meeting opened with dinner at the Endicott House on the evening of the 18th with a lively informal scientific discussion. The following day was packed with presentations. The first two sessions focused on comparing the different technologies that can be used to try and predict the response of an individual’s cancer cells to different drugs. The tumors under consideration included both solid tumors and leukemia. A detailed discussion of the different approaches to culturing patient’s cells in the presence of different types of drugs ensued. This was followed by a debate about the best way to readout how the tumor cells respond to drugs.

In addition to technical discussions, further sessions revolved around the barriers to uptake to these strategies. Identified barriers included matching the best culture conditions to tumor type, the problems of obtaining tumor tissue for this type of analysis, and regulatory concerns. Moreover, this type of therapeutic optimization is vastly different to how conventional drugs are tested and therefore there is a real need to identify methods to validate these approaches to allow them to be adopted for clinical trials and ultimately introduced into practice.

Several points were agreed upon following the meeting. The group will author a white paper defining functional precision cancer medicine, and explaining its use in meeting the unmet need for predicting an individual’s response to drug therapy. It was proposed to form groups to explain our strategies to the FDA and to engage them as partners. In addition, we will meet with thought leaders within the NCI, to present a vision of precision medicine that goes beyond just a genomic analysis. This latter will be important for gaining the resources needed. Several of the attendees volunteered to travel to Washington /Bethesda in person for these meetings. Finally, there was general agreement that a society should be formed, for purposes of awareness, sharing of ideas, and organizing subsequent meetings of a larger community. Attendees greatly enjoyed an interactive and exciting meeting which will hopefully catalyze much useful improvement in the state of cancer precision medicine.

Forum Participants

Anna Baker, PhD
Arizona State University

David Barbie, MD
Dana-Farber Cancer Institute

Jeffrey Engelman, MD, PhD
Novartis Institues for BioMedical Research Inc.

Mark Frattini, MD, PhD
Columbia University

Carla Grandori, MD, PhD
Cure First

Oliver Jonas, PhD
Massachusetts Institute of Technology

Christopher Kemp, PhD
Fred Hutchinson Cancer Research Center

Richard Klinghoffer, PhD
Presage Biosciences

Anthony Letai, MD, PhD
Dana-Farber Cancer Institute

Keith Ligon, MD, Phd
Dana-Farber Cancer Institute

Scott Manalis, PhD
Massachusetts Institute of Technology

David Parkinson, MD
ESSA Pharmaceuticals Corporation

David Tuveson, MD, PhD
Cold Spring Harbor Laboratory

Jeffrey Tyner, PhD
Oregon Health & Sciences Institute

Jennifer Wargo, MD
MD Anderson Cancer Center

Krister Wennerberg
Institute for Molecular Medicine Finland (FIMM)