A new study by Mass General Brigham researchers provides a glimmer of hope for patients with glioblastoma, the most common and deadly form of brain cancer in adults.
The researchers found that glioblastoma patients who received a common anti-seizure and pain drug — gabapentin — ended up living longer.
“This study is an exciting step forward,” said lead author Joshua Bernstock, a clinical fellow in the Department of Neurosurgery at Brigham and Women’s Hospital.
“GBM (glioblastoma) is a relentlessly progressive and nearly universally fatal disease,” Bernstock added. “The discovery that an already approved medication with a favorable safety profile can extend overall survival represents a meaningful and potentially practice-changing advance.”
With about 12,000 new glioblastoma cases diagnosed each year in the U.S., the cancer accounts for most primary brain tumors in adults.
Overall survival has barely improved over the last several decades, with most patients living only 12 to 14 months after diagnosis, and 5.5 months after recurrence.
The new study was sparked by insight from the field of cancer neuroscience, which showed the success of using gabapentin in mouse models.
Intrigued by this research, Bernstock and colleagues looked at the outcomes of 693 glioblastoma patients at Mass General Brigham.
Many of the patients were already taking gabapentin for reasons that typically centered on nerve pain. Those who were taking the drug survived an average of 16 months, compared to 12 months for those who weren’t. This four-month survival benefit was statistically significant.
Because the results seemed “almost too good to be true,” according to Bernstock, he connected with Shawn Hervey-Jumper’s team at University of California, San Francisco to add to the dataset — and discovered they were able to replicate what was found at Mass General Brigham.
Of the 379 patients with newly diagnosed glioblastoma included in the UCSF cohort, patients on gabapentin survived an average of 20.8 months — while those not taking gabapentin survived an average of 14.7 months. Together, the data included 1,072 patients and found a significant survival benefit.
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“There have been very few advances in survival for GBM patients since the early 2000s,” Bernstock said. “We need to think more creatively about the emerging biology in these tumors and how to target them.”
While the findings are promising, the study is retrospective — the researchers didn’t give patients gabapentin in a controlled way to test its effects.
Bernstock stressed that larger, randomized clinical trials are needed to confirm the results.
