Priscilla Brastianos, MD, is leading a research effort to understand the molecular and genomic drivers of metastatic brain tumors and develop more effective treatments.

One of the most common, and most devastating, complications of cancer is brain metastasis, which is when the primary cancer spreads to the brain. Many types of cancers result in brain metastases – among the most common are lung, breast, skin, colon and kidney cancer. Approximately 25% of all cancer patients will develop a metastatic brain tumor; of those, 50% will die.

As Dr. Brastianos puts it: “This is an area of extraordinary clinical need.”

Now Priscilla Brastianos, MD, director of the Central Nervous System Metastasis program at the Massachusetts General Hospital Cancer Center, is leading an effort to change those grim statistics. Her lab is working to understand the molecular and genomic drivers of brain metastases, so they can better identify new therapeutic targets to treat the disease.

Current treatments for metastatic brain tumors are not particularly effective, as the statistics indicate. As Dr. Brastianos puts it: “This is an area of extraordinary clinical need.”

Creating a Tissue Bank

When she began her study of this condition, Dr. Brastianos was confronted with a serious challenge – virtually no tissue was available for research. “The largest study ever performed had been on one patient,” she recalls. Her idea was to compare the genomics of the two tumors – the original with the metastasis – and identify the differences as therapeutic targets. But to do this on a useful scale, she needed to study many samples. And there were none to study.

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Read more: Dr. Brastianos Named 2016 Honoree for the one hundred

Undaunted, Dr. Brastianos took the extraordinary step of creating a tissue bank. She teamed with collaborators around the world to recruit patients to donate samples, including facilities in Spain, Korea and across the United States. “We spent more than 3 years getting patients to provide samples for our study. So far, more than 400 patients from all over the world have generously donated their tissue samples,” she says.

New Brain Tumor Targets

With samples in hand, Dr. Brastianos worked with computational biologists to identify the genetic variations and identify treatment targets. They found that more than 50% of brain metastases had clinically actionable mutations (meaning mutations for which there are drug targets) not present in the original primary tumor. Based on these data, Dr. Brastianos and her team are starting a genomically guided brain metastasis trial at Massachusetts General Hospital, the first of its kind in the US.

Moreover, Dr. Brastianos has applied this methodology in several other types of brain tumors. For example, in patients with a specific type of tumor called a craniopharyngioma, Dr. Brastianos identified a genetic defect similar to one found in melanoma. Melanoma patients are often treated effectively with a BRAF inhibitor. When Dr. Brastianos treated a craniopharyngioma patient with a BRAF inhibitor, the results were dramatic: “80% of the patient’s tumor had shrunk within 30 days,” she says.

It’s a remarkable outcome, and her lab is now working on translating other genomic defects into clinical trials. This novel approach offers hope to cancer patients experiencing metastatic brain tumors.

Working as a Team

The MGH Fund is an important source of funding for such groundbreaking biomedical research and a crucial part of our mission at Mass General.

Her lab is a truly collaborative enterprise. In addition to the computational biologists, Dr. Brastianos works with pathologists, oncologists and other specialists to administer the treatment and run the trials. And she herself sees patients one day per week.

Dr. Brastianos is just getting started. Even though she is still in her mid-thirties, she is already a renowned figure in her field. The work underway at the Brastianos Laboratory is an example of the pioneering biomedical research conducted at Mass General. “This is the only program of its kind in the United States,” says Dr. Brastianos. “With the BRAF inhibitor, we went from the lab to a clinical setting inside of 2 years. That kind of rapid development saves lives.”

The MGH Fund is an important source of funding for such groundbreaking biomedical research and a crucial part of our mission at Mass General. For our 2016 MGH Fund Annual Drive, we hope to raise $2 million to increase our support for biomedical research, patient care initiatives and community outreach. As a donor, you are helping our physicians and scientists conduct pioneering biomedical science that leads to lifesaving treatment.

Please give generously to the 2016 MGH Fund Annual Drive, so we can fund more research, help more people and save more lives.