The "aim" is to match patients to the molecular characteristics of tumors most likely to respond. The "goal" is to mount smaller studies by enrolling only patients with genetic profiles found to respond to a drug. Results among patients who enroll early in the trial will up the odds that subsequent patients will be assigned to a drug that matches their tumor's genetic profile. The "hope" is that winners and losers emerge more quickly than under conventional studies, where it's not known until the end of a trial whether a drug is working.
The "intent" is to apply "window therapy" (i.e. using the window of time before definitive intervention to introduce and test new therapies) to facilitate drug introduction. However, despite the enthusiasm, the design and application of this type of trial is demonstrably less than meets the eye. At its core, this is just a randomized selection of candidates. While it may enable the investigators to interrogate the tissue biopsies to answer scientific questions of interest, it does so with no immediate benefit to the patients who participate.
Indeed, patients who gain benefit (after being randomized to the investigational arm and then receiving a new therapy that actually works) receive said benefit by what could best be described as blind luck. The suggestion that this is incorporating "molecular triaging" into their phase I clinical trial unit, falls flat when one realizes that a good outcome is nothing more than a chance event.
Truly personalized care represents the application of validated predictive models to select candidates for specific therapies. Good outcomes can then be ascribed to the intelligent selection and application of effective treatments. The cancer research community’s single-minded focus on genomic platforms, to the exclusion of functional platforms, forces patients to continue to participate in randomized trials to test hypotheses of interest to the investigators, largely at the expense of the patients in need.
What these genomic investigators are expecting their patients to say to them is “You may not be able to treat me any better, but I like the way you think.” What informed patients should be saying instead is,“I don’t care how you think. I want you to treat me better!”