'Incredibly Rapid' Enrollment in Breast Cancer Study. Why?

Nick Mulcahy

June 05, 2016

CHICAGO — In the past 7 months, the Metastatic Breast Cancer (MBC) Project has enrolled more than 2000 patients from 50 states as part of a big-data study that is collecting saliva, tumor tissue, and health records from individuals with advanced disease.

This is "incredibly, incredibly rapid enrollment" said Sumanta Kumar Pal, MD, a medical oncologist at the City of Hope National Medical Center in Duarte, California, who moderated the press conference during which the project was discussed here at the American Society of Clinical Oncology (ASCO) 2016 Annual Meeting.

The enrollment is "remarkable," said Richard Schilsky, MD, chief medical officer of the ASCO, and "far greater" than what investigators anticipated.

Engagement in the project, which began on Metastatic Breast Cancer Day in October 2015, has been strong, despite the fact that participants are offered no great payoff; there is no promise of improved survival or delayed progression. This is a basic-science study that seeks to accelerate genomics research, which in turn could inform clinical decisions.

As part of their promise to patients, investigators are reporting and sharing data as they are gathered.

Currently, median age of the participants is 53 years (range, 24 - 91 years), median time between initial diagnosis of breast cancer and metastatic breast cancer is 2 years, and 36% of the participants were diagnosed with de novo metastatic disease.

Median time since the diagnosis of metastatic breast cancer is 3 years; however, notably, more than 100 patients report having been diagnosed with metastatic disease at least 10 years ago.

Dr Sumanta Pal

More than 600 patients reported being on a therapy for more than 2 years. Remarkably, more than 900 participants — nearly half the current cohort — reported an "extraordinary response" to therapy. In fact, there were 117 prolonged responses to capecitabine, 63 to platinums or PARP inhibitors, and 35 to everolimus.

Genetic analyses of the extraordinary responders and of de novo metastatic breast cancer are underway.

Dr Nikhil Wagle

"Many" of the patients have rare phenotypes, which are "challenging" to identify with traditional approaches, project investigator Nikhil Wagle, MD, a medical oncologist at the Dana-Farber Cancer Institute in Boston and associate member of the Broad Institute in Cambridge, Massachusetts, and his colleagues write in their meeting poster.

To date, the MBC Project cannot claim to have altered the arc of a single case of metastatic breast cancer. So why have metastatic patients flocked to the project?

Answers to this question provide a potential blueprint for the productive engagement of patients in cancer research, according to the oncologists and patients interviewed by Medscape Medical News.

"Most patients have never been asked if they would like to contribute a tissue sample or their medical records," said Dr Wagle. So his team asked them — directly.

They used social media (Twitter and Facebook) and contacts at advocacy organizations in a direct-to-patient initiative. With such methods, clinicians are spared the paperwork and time-consuming effort of enrolling a patient in a study.

"They're cutting out the middle man," said Jenni Grimes, a breast cancer "metser" from Los Angeles; it is a more efficient recruitment and research model.

Grimes explained that most breast cancer tumors are not studied because they are stored in community-based practices, away from the research studies at major centers. Or, even more frustrating, data on patients' tumors are hoarded by academics at those big centers.

The list of advocacy organizations participating in patient outreach for the MBC Project ranges from the well-established Metastatic Breast Cancer Network to fledgling groups such as MET-UP, which vows to agitate for an increase in research funding for metastatic disease. Compared with early-stage disease, and relative to respective incidence, metastatic disease is grossly underfunded, as reported by Medscape Medical News.

In other words, the MBC Project did not reject or avoid any group or person.

The MBC Project staff are "like unicorns, they're magical," said participant Beth Caldwell.

Caldwell, who is from Seattle, is the mother of two small children whose initial diagnosis was metastatic breast cancer. She said that metsers are accustomed to — and fatigued by — breast cancer clinical trials from which they are regularly excluded because of some clinical measure. The MBC Project "takes us where we are," she explained.

Enrollment is simple, and interested patients can sign up on the MBC Project website. Visitors to the site can click the "count me in" button, which will take them to a 16-question survey (completed by 95% of enrollees so far) and an online consent form.

Next, patients are asked to submit a saliva sample using a boxed kit that is mailed to them. The saliva is used to extract germline DNA. The MBC Project takes responsibility for securing medical records and tumor tissue samples from clinicians. Whole-exome and transcriptome sequencing is performed on the tumor and germline.

Beth Calabotta, MBA, a metser from Quincy, Illinois, said she believes the approach will ultimately deliver value. "They are not just throwing drugs at the wall," she said. She acknowledged that she is unlikely to benefit from any insights in her lifetime, but is satisfied to be contributing. "This study is going to help people," she said.

This is "patient-driven research," a term coined for the MBC Project, Dr Wagle explained.

During the press conference, Dr Pal suggested that the concept might catch on widely.

"If this paradigm continues to enjoy the success it has thus far," he noted, the "patient-driven model is something we can implement across [cancer] disease types."

This study received funding from the Broad Institute and the Conquer Cancer Foundation. Dr Pal reports ties to multiple pharmaceutical companies. Dr Schilsky has disclosed no relevant financial relationships. Dr Wagle reports financial ties to Foundation Medicine. Some members of the MBC Project staff report ties to industry, as detailed in the abstract.

American Society of Clinical Oncology (ASCO) 2016 Annual Meeting: Abstract LBA1519. To be presented June 6, 2016.

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