TGen study offers path for late-stage cancer patients to leverage their own health data to guide their care
Rick Stanton thinks it might be the little things that count.
At a June virtual meeting of the cancer hackathon he’s participating in, he’s asking about a very little thing called an organoid. A lab-grown ball of his own prostate tumor cells, about as wide across as a strand or two of hair.
At the meeting, Payel Satterjee, Ph.D., explains how she and her team craft organoids from tumor cells, pitting them against a carefully curated list of drugs to find promising “hits” against a patient’s unique cancer.
Maybe, Stanton thinks, an organoid could start bearing the brunt of what he’s had to endure for four years and counting. He’s an advanced prostate cancer patient, yes, but he’s also a surfer and a guitar player. His latest bout of chemotherapy, however, has left his fingers tingling too badly to play.
And now one of his doctors is talking about the possibilities of yet another new drug.
“But I’d rather have you test it,” he says, his lively face taking up center square in the Zoom discussion, “then, you know, then have me test it—then you go through the whole thing, yes, I understand that,” Satterjee, chief scientist at SEngine Precision Medicine, finishes Stanton’s sentence from her own square.
They laugh together. “Yeah,” Stanton says. “So it’s not just, ‘oh wow, it didn’t work. Sorry, bud.’”
In many ways, Stanton is like a lot of cancer patients. The endless blood draws, biopsies, scans, genetic testing, clinical trials, all of it creating a tidal wave of data that describes his unique cancer journey. He is nearing the end of the national guidelines for prostate cancer care, without a lasting response for his stage IV cancer.
But in another way Stanton is unique: he is the sole patient participant in TGen’s PEOPLES Protocol, a new study that shows how patients can direct their own tidal waves of data to guide their future treatment.
The protocol (PEOPLES stands for Patient Engagements, Operational Practices, and Laboratory Environment Standardization) allows Stanton to access his cancer data and request new analyses with the help of TGen researchers.
Stanton meets weekly to talk through how best to organize and present his data that have been collected so far. The group, led by TGen’s Nicholas Schork, Ph.D., also discusses new tests and analyses that could expand the range of treatment possibilities for Stanton.
The PEOPLES protocol was inspired in part by a growing “right to try” movement among patients, who advocate for access to experimental medicines or procedures for conditions not treatable by currently available drugs. Under the rubric of “compassionate care,” for instance, the U.S. Food and Drug Administration now offers an Expanded Access Program for patients and their doctors to apply for some of these treatments.
If these programs exist for drugs, thought Schork, why not for data?
“For many people who are in the later stages of their cancer, the treatment guidelines no longer apply,” he says. “If we’re OK with people trying drugs at end of life, we should at the very least be OK with them trying to explore their data for information that may help lead to a drug.”
Schork, along with his TGen colleagues Laura Goetz, M.D. and Edward Kalpas, M.D., hope the protocol offers a way for Stanton and future patients to use their data safely and perhaps even creatively. The prostate cancer hackathon that Stanton participates in, organized by CancerHacker Lab founder Brad Power, is one of the pathways that the TGen researchers had in mind when they began the protocol.
“What Rick and the hackathons have been opening up for advanced cancer patients in general is more testing to guide personalized treatment decisions,” Power says.
“Our motivation for this study was thinking about what data we could bring to the table to help the patients and interested physicians make decisions about the next steps,” says Schork, “to make it less guesswork and more scientifically backed.”
Stanton’s motivation for participating? “In a nutshell, it was the hope of a best chance to extend my life.”
There’s another way in which Stanton isn’t a typical cancer patient, or a typical healthcare data consumer. He’s an engineer, specifically one with 20-plus years of experience in bioinformatics and biological signaling. A family-run company, Stanton Biosciences, performs some of the same kinds of tumor testing that he’s using to search for his next treatment.
So his discussions with Schork and colleagues about how to handle and sort through his data can take on a top-level technical tone. But even he is looking for new ways to understand what his data mean.
The engineer in him likes to visualize problems, he says, so he is creating a “battle map” of sorts. The battlefield consists of some of the complex concepts in cancer treatment—immunotherapy, the cell environment around the tumor, and the specific molecular signaling pathways that researchers know are related to certain cancers.
A patient’s data are sort of like the battalions that have been mustered so far, placed on the battlefield to show “where and how it makes the most sense to intervene,” he says.
Stanton thinks visualizations like the battle map can help people like his companion Brian McCloskey in the ongoing hackathon. McCloskey feels comfortable with data from his work as an e-commerce and tech marketing executive. But at this point in his advanced prostate cancer treatment, the father of three is facing a bewildering array of 17 possible treatment options.
“He was saying that I’m trying to understand my cancer and fight it, and it seems like I’m supposed to learn Ph.D. material in a few weeks just to know what this stuff means,” Stanton says of McCloskey.
There’s no real precedent for how best to share research data with a patient, which is another reason why the PEOPLES protocol is so important, says Schork.
Genetic sequencing of tumors, for example, has become more frequent for patients. “The companies that do the sequencing tend to focus on the presence of mutations that have been shown already to match the effects of certain drugs,” Schork explains. “There is a lot information that is generated from sequencing that might all be filtered down to one or two recommendations consistent with the guidelines. But then the question is what about all that other stuff?”
Finding ways to share the “research-only” data that are generated during these tests, in a format that is intuitive and understandable to a patient, “is something we’ve been grappling with,” says Schork.
Under the protocol, the TGen team has provided Stanton with new analyses that could expand his treatment possibilities. Stanton received neoantigen profiling, for instance, which looks for tumor proteins that activate the immune system. Stanton and his doctors can now consider new cell-based therapies based on these data.
Neoantigens, organoids, whole genome sequencing—Stanton is eager to have all these data to move forward. “Each is a stone unturned, and possibly an important one,” he says. “But is the key under there? You won’t know until you look.”
Schork is quick to point out that the idea that patients should have access to and control over their health data is nothing new. The accompanying concerns that these patients will seek out inappropriate, dangerous or costly treatments have also been around for a long time.
The PEOPLES protocol, he says, shows that it’s possible for a patient to gain access to their own data in a safe and ethical way.
For Stanton, the protocol has given him a path forward just as he reaches the end of standard care.
“What the PEOPLES protocol and TGen have enabled is a suite of analyses and multiple shots on goal that could save my life,” he says.
Not all patients will want to take Stanton’s route of sharing his data in hackathons or drawing up battle maps. But the TGen team hopes that their protocol will help empower patients as they discuss their options with their healthcare team.
“Physicians may feel threatened when patients want to explore experimental testing strategies, because they [the physicians] often don’t know how to order, let alone interpret, such tests. Dr Kalpas and I want to encourage our colleagues to embrace such efforts and see the PEOPLES protocol as a first step in normalizing this process for medical professionals,” Goetz said.
“We don’t doubt that there are patients out there who are perfectly willing to put this decision-making into their physicians’ hands and trust them completely,” Schork says “But in an era of big data and the right to try, why not make an effort and say it’s OK to cater to the interests of patients that would like to go a little bit deeper?”
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