Question ID: WS-21
Submitted by: C. Anthony Blau
January 31, 2011
How can we convert the experiences of individual cancer patients into scientifically valid experiments? Background: We know that every cancer is unique, yet our clinical trials assume that patients can be clustered into groups that are more or less similar. Feasibility: It would be feasible to intensively study a small number of cancer patients who are beyond "standard of care." These patients would be highly selected based on their interest and the clinical and scientific appropriateness for their participation in a "Manhattan Project" in which the patient and their tumor would be the subject of investigation. In a "no-holds-barred" effort, any scientifically defensible technology that might provide insight into the patient and/or their tumor should be considered. The goal of this effort would be to generate hypotheses regarding tumor drivers and potential susceptibilities, and then to directly test these hypotheses by treating the patient with the corresponding drug (or drugs) that are predicted to be effective. Repeated biopsies of the tumor following drug treatment would allow one to monitor the molecular responses to the treatment and to correlate these with clinical responses. Additional biopsies after disease relapse might uncover tumor strategies for escape, and the treatment could be modified accordingly. While this approach may be premature for some types of advanced cancer, it seems especially well suited for cancers where a lot is already known, for example GIST. An obvious difficulty of this approach rests in a lack of controls, however innovative strategies could be envisioned, for example comparisons of stable versus progressive disease within an individual patient. Implications of success: Efforts to deeply understand a few cancer patients may uncover important findings for cancer patients generally.
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