BOSTON STUDY FINDS COMMON DRUG MAY EXTEND LIFE FOR BRAIN CANCER PATIENTS
A recent study from Mass General Brigham has uncovered a promising lead in the fight against glioblastoma, the deadliest and most common brain cancer in adults. Researchers found that patients who were already taking the drug gabapentin — commonly used for seizures and nerve pain — lived longer than those who were not on the medication.
Lead researcher Joshua Bernstock, a clinical fellow in neurosurgery at Brigham and Women’s Hospital, described the results as a major step forward. “GBM (glioblastoma) is a relentlessly progressive and nearly universally fatal disease,” he said. “The discovery that an already approved medication with a favorable safety profile can extend overall survival represents a meaningful and potentially practice-changing advance.”
Glioblastoma affects around 12,000 new patients in the U.S. each year and accounts for the majority of primary brain tumors in adults. Survival rates have remained grim for decades — with most patients living only about a year after diagnosis.
The idea to look into gabapentin came from previous studies in cancer neuroscience that showed positive effects in mice. Bernstock and his team then analyzed medical records of 693 glioblastoma patients treated at Mass General Brigham. They found that patients taking gabapentin lived an average of 16 months — four months longer than those who weren’t.
To confirm their findings, the team collaborated with researchers at the University of California, San Francisco. In a separate group of 379 patients, those taking gabapentin lived an average of 20.8 months, compared to 14.7 months for those not on the drug.
In total, the combined data from 1,072 patients showed a consistent and statistically significant increase in survival time linked to gabapentin use.
Despite the encouraging results, Bernstock cautioned that the study was retrospective — meaning the researchers analyzed past patient data rather than conducting a controlled trial. As such, more rigorous clinical testing is necessary to confirm whether gabapentin directly contributes to improved survival.
“There have been very few advances in survival for GBM patients since the early 2000s,” said Bernstock. “We need to think more creatively about the emerging biology in these tumors and how to target them.”
COMMENTARY:
The findings from this new study on gabapentin’s effect on glioblastoma are nothing short of remarkable. For decades, glioblastoma has stood as one of the most stubborn and lethal forms of cancer, with little to no progress in treatment outcomes. The fact that an existing, FDA-approved medication like gabapentin — primarily used for seizures and nerve pain — could potentially extend the life expectancy of glioblastoma patients is both exciting and hopeful. This breakthrough brings new energy to a field that has long struggled to find effective solutions.
One of the most encouraging aspects of this study is that gabapentin is already widely used, meaning its safety profile is well understood. This gives it a major advantage over experimental therapies that must go through years of safety testing before being deemed suitable for human trials. If further studies confirm its effectiveness, gabapentin could be integrated into glioblastoma treatment protocols relatively quickly, offering patients an immediate option with very low risk. This kind of low-cost, high-reward opportunity is rare in oncology.
What also makes the results stand out is the consistency across two separate patient groups. Both the Mass General Brigham and UCSF data showed that patients on gabapentin lived noticeably longer — an average of 16 to 20.8 months versus the typical 12 to 14. This wasn’t just a statistical fluke; it was a reproducible outcome, which gives researchers and clinicians greater confidence that gabapentin might be doing more than just treating nerve pain. It may actually be interfering with the cancer’s ability to grow or spread.
In a disease where every additional month of life is a victory, a four- to six-month increase in survival time is meaningful. But perhaps more importantly, this discovery opens the door to further research on how gabapentin might be working at the cellular level. Does it block neural pathways that tumors use to feed off the brain’s communication systems? Does it disrupt the microenvironment that allows these tumors to thrive? The biological mechanisms deserve urgent exploration.
This also raises a provocative and potentially transformative question: could gabapentin’s effectiveness be enhanced through alternative methods of delivery? If patients receiving oral gabapentin are seeing these kinds of results, what might happen if a higher dose or concentrated form of the drug were injected directly into the tumor site? Direct delivery could allow for higher local concentrations of the drug without increasing systemic side effects — a strategy that has worked for other brain-targeted therapies.
The idea of localized treatment is especially appealing for glioblastoma, given its invasive nature and resistance to traditional chemotherapy. Direct injection could potentially bypass the blood-brain barrier, which is a major obstacle for many cancer drugs. Gabapentin is known to cross the blood-brain barrier to some extent, but a localized approach could deliver a much stronger, targeted punch to the tumor itself. This line of inquiry seems not only logical but urgently needed.
Moreover, if direct injection is feasible, it opens up the possibility of developing a new formulation of gabapentin — perhaps a slow-release version or a gel-based delivery system — tailored specifically for brain tumor treatment. This could maximize the therapeutic impact while minimizing unwanted side effects. Researchers might even look into combining gabapentin with other drugs to create a synergistic effect, enhancing its potential further.
Of course, these ideas would need to be tested in clinical trials, beginning with animal models and then progressing to human studies. But the existing data provides a compelling reason to pursue this direction. If we’ve learned anything from the history of medicine, it’s that some of the most groundbreaking treatments come from thinking outside the box — and sometimes, from repurposing drugs that have been right in front of us all along.
The emotional weight of this breakthrough cannot be overstated. Glioblastoma is a diagnosis that often leaves families feeling hopeless. For doctors and patients alike, knowing that a familiar drug may offer more time — more holidays, more birthdays, more memories — is incredibly powerful. It may not be a cure, but it’s a step toward one, and that step matters.
This study reminds us that sometimes, the answers to our most difficult medical challenges don’t always lie in the newest, most high-tech solutions. Sometimes, they’re hidden in plain sight, waiting to be discovered in medications we’ve been using for years. Let’s hope this sparks not only more research, but more optimism — and leads to real, life-changing outcomes for those battling glioblastoma.
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