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Targeting Treatments

By Colleen Kearney Rich on August 6, 2018


Researchers at Mason’s Center for Applied Proteomics and Molecular Medicine are bringing personalized medicine to more people with metastatic breast cancer in their latest clinical trial. This third phase of breast cancer research, supported the Side-Out Foundation, involves 100 patients from 10 sites around the United States and is the center’s largest clinical trial yet.

Mason researchers are looking for biomarkers to uncover why some patients respond to a specific treatment while others do not. Oncologists can then use that information to find the most effective treatment for their patients.

Mariaelena Pierobon, Research Associate Professor, Applied Proteomics and Molecular Medicine. Photo by Evan Cantwell

“We’re trying to find biomarkers, that will tell us upfront whether a patient will benefit or not so that we can try to provide more targeted treatment for our patients and help physicians recommend the right drug,” says Mariaelena Pierobon, MD, who is leading the work.

The Side-Out sponsored trials are using molecular profiling, involving DNA, RNA, and protein and phosphoprotein information to try to understand what is driving each individual tumor. “So if certain genes or certain proteins were highly deregulated in a patient’s tumor, we look for drugs that block those specific alterations. So every patient receives a different treatment based on their personal biology,” says Pierobon.

The profiling is done using a protein array technology invented at CAPMM. As the tissue that the researchers work with is often tiny, think needle biopsy, they needed to find a way to amplify the proteins they were looking for. “We are actually way beyond a genomics-alone approach to precision medicine—in some ways that has already become ‘old-fashioned.’ The proteome is where the next revolution in precision medicine is—and we had to invent a whole new proteomic technique to meet the need, and that’s what we did,” says Emanuel Petricoin, center’s co-director and the principal investigator of the clinical trials. “Now we have a robust method that allows us to understand what drug targets are actually in use—like a GPS roadmap—for each patient’s tumor, and we’re using that in clinical trials.”

Often by the time patients with metastatic breast cancer come CAPMM’s way, they have exhausted a number of therapies and are running out of time. Although the researchers aren’t promising a cure, the results of the first two trials were better than the researchers had anticipated.

“The oncologists are getting key missing information that we are providing. That’s helping to make treatment decisions, and those treatment decisions appear to be providing clinical benefit,” says Petricoin. “Patients are actually doing better clinically, and that’s is very exciting.”

Breast cancer is not the only disease CAPMM researchers are working on, but the long-term goal is the same: That one day, after a patient receives a diagnosis, their doctor will order a test that will tell them the best way to proceed, eliminating uncertainty and lost time while leading to better outcomes.


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