Medication Offers Hope for Glioblastoma Patients
A commonly prescribed medication might provide new prospects for individuals fighting glioblastoma, a highly aggressive tumor that makes up the majority of malignant brain cancers in adults and generally has one of the worst prognoses in oncology.
According to a recent study published in a peer-reviewed journal, patients taking gabapentin—a drug often used for nerve pain and seizures—experienced several additional months of life compared to those who didn’t use it.
This insight comes from a retrospective analysis conducted by researchers at Mass General Brigham and the University of California, San Francisco. Each year, glioblastoma affects approximately 12,000 Americans, yet survival rates have shown little improvement, as most patients live only 12 to 14 months after diagnosis.
The notion for this research was sparked by Dr. Joshua Bernstock, the lead author, who described his growing interest in cancer neuroscience—a developing field focused on understanding how tumors manipulate the nervous system for their growth and survival.
In a 2023 study, West Coast researchers discovered that gliomas, which include glioblastoma, integrate into neural circuits, utilizing a protein called thrombospondin-1 (TSP-1) to promote their growth. Bernstock recalled that gabapentin had been found in previous studies to interfere with TSP-1 activity, leading him to consider the implications.
“I reviewed it several times the day it was published and thought, ‘Oh my God,’” Bernstock, a clinical fellow in neurosurgery, stated. “This wasn’t some new experimental treatment. It was already accessible and being prescribed.”
His team analyzed the medical records of 693 glioblastoma patients from Mass General Brigham, many of whom had received gabapentin for chronic nerve pain. The data indicated that these patients had a median survival of 16 months—four months longer than those who weren’t on the medication.
To verify the trend, they collaborated with researchers from UC San Francisco, who examined data from an additional 379 patients. Their findings revealed an even larger difference: patients on gabapentin lived, on average, 20.8 months compared to 14.7 months for those not on the drug.
While Bernstock highlighted the encouraging nature of these findings, he noted that only a randomized clinical trial could definitively confirm gabapentin’s effectiveness. However, the biological mechanisms backing this hypothesis seem promising. Patients on gabapentin exhibited reduced TSP-1 levels in their blood, and the team controlled for numerous confounding factors.
“We know it’s biologically plausible,” he remarked. “The drop in serum TSP-1 adds weight to our hypothesis.”
He expressed hope that this data would lead to prospective trials, some of which are already starting to recruit participants.
Glioblastoma’s resistance to traditional treatments like chemotherapy and radiation, combined with its heterogeneous nature—meaning that no two tumors look the same—even in one patient, has hindered advancements for years, making breakthroughs infrequent.
“This is an incredibly challenging disease,” Bernstock noted. “Even small steps forward can be significant.”
A few extra months can profoundly affect patients and their loved ones. “You haven’t seen these families in the clinic,” he said. “You don’t know the father who wishes to see his daughter graduate or the mother wanting to settle family matters before passing.”
These personal stories inspire him to seek out new solutions in the lab. “It’s really painful to sit across from someone and feel powerless given what’s conventionally available,” he expressed. “That’s what drives me to keep striving for progress.”
