Precision Cancer Medicine by Functional Biomarkers

Anthony Letai M.D., Ph.D., Dana-Farber Cancer Institute

David Barbie M.D., Dana-Farber Cancer Institute
Anna Barker Ph.D., Arizona State University
Jeffrey Engelman M.D., Ph.D., Novartis Institutes for BioMedical Research, Inc.
Mark Frattini M.D., Ph.D., Columbia University
Carla Grandori M.D., Ph.D., Cure First
Oliver Jonas Ph.D., Massachusetts Institute of Technology
Christopher Kemp Ph.D., Fred Hutchinson Cancer Research Center
Richard Klinghoffer Ph.D., Presage Biosciences
Keith Ligon M.D., Ph.D., Dana-Farber Cancer Institute
Scott Manalis Ph.D., Massachusetts Institute of Technology
David Parkinson M.D., ESSA Pharmaceuticals Corporation
David Tuveson M.D., Ph.D., Cold Spring Harbor Laboratory
Jeffrey Tyner Ph.D., Oregon Health & Sciences Institute
Jennifer Wargo M.D., MD Anderson Cancer Center
Krister Wennerberg, Institute for Molecular Medicine Finland (FIMM)


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.