September 26, 2017
Investigators gave enrollees a frank assessment of potential benefits, but human spirit seemed to overpower that message
Beginning four years ago, 15 women with incurable (metastatic triple-negative) breast cancer were enrolled in a unique clinical trial. It explored one concept of “precision medicine” – the suggestion that the brain power of multiple medical experts and the collection of an immense volume of data about each woman’s condition would yield the most personalized, customized care possible.
The women were advised during enrollment that they would shoulder a burden far greater than that involved in a more typical test of an unproven chemotherapy. Each would have to submit to multiple tumor biopsies and other lab tests to create an unprecedented database of genomic information.
And they were told that this trial, for all its personalized attention and requisite burden, gave them no better chance of survival than any other medical path; rather, the benefits and wisdom of this trial would likely be experienced by future patients.
“These patients are very highly motivated to have their experience with cancer transcend their own lifetimes. The idea that their experience could help others in the future compelled them,” said Dr. Tony Blau, the trial’s lead investigator and a UW Medicine hematologist.
He discussed his recently published survey of participants’ impressions about their trial experiences.
“We go through a long informed-consent process to emphasize that we don’t expect a better outcome for them in this trial then they would get in any other trial. Despite trying hard to make that point, we found that most patients do believe that they are more likely to benefit personally from their participation than we have intended to convey. Hopeful optimism – the human spirit –may be at the root of that.”
Blau himself was diagnosed with myeloma two years ago, so “I totally understand this concept that, despite apparent long odds, you hope for the very best.”
He described the huge challenge of trying to apply so much genomic test information to patient care. “Conventionally you do a single validated test and examine the characteristics of the results. What we’re talking about here is using 100,000 tests, all at once, and none of them are validated. There’s tremendous uncertainty in trying to synthesize these incredibly large data sets into an accurate understanding of what’s going on in the patient. The patients in our trial understand that, yet they want us to try.”
The trial recently enrolled its 27th patient. Fourteen of the women have died. A a few have survived longer than expected, although this is typically the case with conventional treatment as well, Blau said.
Contact Brian Donohue if you’d like to interview Dr. Tony Blau.