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Study: More Metastatic Cancer Patients Should Be Included in Clinical Trials

woman getting brain MRI

Sebastian Condrea / Getty Images

Key Takeaways

  • Researchers have historically excluded cancer patients whose disease has spread to the brain from clinical trials due to their poor prognosis.
  • A new tool called the Graded Prognostic Assessment estimates survival based on multiple factors, including the initial cancer type.
  • Cancer patients with brain metastases may still be suitable candidates for research, according to new study findings.

Limited treatment options are available to cancer patients whose disease has spread to the brain. Usually, these patients are not eligible for clinical trials. But new research shows that some of these individuals could still benefit from inclusion in trials.

A report published in the Journal of Clinical Oncology last month studied nearly 7,000 cancer patients with brain metastases. Along with his team, lead study author Paul W. Sperduto, MD, a board-certified radiation oncologist with Minneapolis Radiation Oncology, developed a diagnosis-specific tool called the Graded Prognostic Assessment (GPA) to determine the expected length of life for patients with cancer that has metastasized to the brain.

When they considered all factors assessed by the tool, researchers noted a wide variability in the life expectancy of patients with brain metastases. As a result, Sperduto and his co-authors recommend that researchers consider metastatic cancer patients with a 50% or greater probability of one additional year of survival to be candidates in clinical trials.

What Is the GPA?

The GPA is a free online tool that predicts survival in lung, melanoma, breast, renal cell, and gastrointestinal cancer patients with metastases to the brain. Patients and clinicians can access it free of charge. The tool assesses several factors to generate a score of 0 to 4, with 0 being the worst possible prognosis and 4 being the best possible prognosis. The factors include:

  • The primary type of cancer
  • Tumor subtype
  • Age
  • Functional ability
  • The extent of brain metastases
  • Molecular markers (alterations in the DNA of tumors)

How Does the GPA Differ From Earlier Tools?

The RTOG RPA classification system, which has been the standard for estimating prognosis in cancer patients with brain metastases, did not consider the initial cancer diagnosis.

“That tool was too subjective,” Sperduto tells Verywell. “It asked the clinician to determine if the primary tumor and systemic disease were under control. Based on that, I wanted to create a more objective prognostic index.”

“Previously, providers surmised that it didn’t matter where your metastases came from,” Minesh P. Mehta, MD, a board-certified radiation oncologist, Deputy Director and Chief of Radiation Oncology at Miami Cancer Institute, and senior author of the study, tells Verywell. “We’ve discovered that the primary tumor did drive the prognosis of these patients.”

Researchers plan to refine the GPA as more data become available. For example, it will eventually evaluate prognosis for other types of tumors once more statistical data for other cancer types and brain metastases become available.

“We will have to update it as practice changes,” Sperduto says. “The next thing we add will be immunotherapy receptor site status.”

GPA Score Can Guide Decision-Making

“This data is useful for the doctor, patient, and family,” Sperduto says. “If the patient’s expected survival time is four months, their treatment choices are different than if it’s four years. This information helps them navigate their options.”

Still, the study authors acknowledge that not everyone wants to know their prognosis.

"We don’t want to impose that knowledge on them—but others very much want to know, and we want to design goals around that,” Mehta says. “Every cancer patient is unique, and we need solid information to make the right decisions, whether they are treatment-related decisions or life-related decisions.

Why Research Should Include Patients With Advanced Cancer

“Historically, the perception was that the median survival for patients with brain metastases was six months. We would not enroll them in clinical trials because they may not live long enough to see the effect of the treatment,” Mehta says. "[But] if you associate certain patients with poor outcomes and you don’t treat them as aggressively, then they don’t have as high a rate of survival. It's a self-fulfilling prophecy.”

The GPA helped show that some patients with brain metastases are projected to have more time. And their participation in clinical trials matters.

If researchers follow Sperduto's recommendation to consider patients with a 50% or greater probability of one additional year of survival to be candidates in clinical trials, they broaden the pool of possible subjects for cancer research.

“Researchers can use this tool to stratify patients in future trials, so they are studying comparable groups,” Sperduto says. “[While] patients achieve most of the endpoints of metastatic disease in less than one year, those with an estimated survival of greater than one year should be allowed in clinical trials.”

“If there are two pathways on a clinical trial, and all the better-prognosis patients receive one treatment, it will make the other treatment look less effective,” Mehta says.

The GPA also helps cancer patients find research trials for which they may be eligible.

“Trials become very important for people who have failed standard treatments because they don’t have a lot of options," Emily Jordan, PhD, a neuroscientist, the Chief Operations Officer for Intrepida and Ancora, tells Verywell. "Many patients are passing away because they are ineligible for trials simply because they have brain metastasis on their medical records, even if it has been treated and is stable.”

What This Means For You

If you are facing advanced cancer and would like to be considered for experimental therapies, speak with your treatment team about your prognosis and options.

“Getting into a trial often comes down to how much you push,” Jordan says, adding the principal investigators of a study may have flexibility in who they allow in. “Treatments are getting better, and patients are living longer, giving cancer more time to metastasize, so we will likely be seeing more brain metastasis patients in the future.”

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  1. Sperduto PW, Mesko S, Li J, Cagney D, Aizer A, Lin Nu. Survival in patients with brain metastases: summary report on the updated diagnosis-specific graded prognostic assessment and definition of the eligibility quotient. Journal of Clinical Oncology. September 15, 2020. doi:10.1200/JCO.20.01255

Additional Reading
  • Survival in Patients With Brain Metastases: Summary Report on the Updated Diagnosis-Specific Graded Prognostic Assessment and Definition of the Eligibility Quotient | Journal of Clinical Oncology. Ascopubs.org. https://ascopubs.org/doi/10.1200/JCO.20.01255. Published 2020.