Mass General Study Drops Brain Cancer Bombshell

Woman undergoing brain scan with doctor monitoring results
Woman Wearing Brainwave Scanning Headset Sits in a Chair while Scientist Adjusts the Device, Uses Tablet Computer. In the Modern Brain Study Laboratory Monitors Show EEG Reading and Brain Model.

Glioblastoma patients taking a common pain and seizure medication lived up to 6 months longer than those who didn’t, offering new hope against one of medicine’s most challenging cancers.

Key Takeaways

  • Gabapentin, a readily available medication used for seizures and nerve pain, has been linked to significantly extended survival in glioblastoma patients according to a new Mass General Brigham study.
  • Newly diagnosed glioblastoma patients taking gabapentin survived 20.8 months compared to just 14.7 months for those not taking the drug – a 6-month increase.
  • Across all 1,072 patients in the study, gabapentin use consistently showed a statistically significant improvement in survival rates.
  • The medication appears to reduce levels of thrombospondin-1, a protein that may contribute to tumor growth, suggesting a potential mechanism for its effectiveness.
  • While promising, researchers emphasize the need for larger controlled clinical trials before changing standard treatment protocols.

A Breakthrough Discovery for Deadly Brain Cancer

In what could be a significant advancement for treatment of one of the deadliest forms of cancer, researchers have discovered that gabapentin – a common medication used for seizures and nerve pain – may substantially extend survival for glioblastoma patients. The retrospective study from Mass General Brigham analyzed nearly 700 glioblastoma patients, finding that those taking gabapentin lived approximately four months longer on average than patients not using the drug. This discovery is particularly noteworthy given glioblastoma’s notoriously poor prognosis and the limited progress in treatment options over the past two decades.

The findings published in Nature Communications revealed that across the entire study population of 693 patients, those taking gabapentin survived a median of 16 months compared to just 12 months for non-users. Even more impressive, when researchers specifically examined 379 newly diagnosed patients, gabapentin users survived for 20.8 months versus 14.7 months for those not taking the medication – a remarkable 6-month survival advantage. These results have generated cautious optimism among oncologists and neurosurgeons who have long struggled with the limitations of current glioblastoma treatments.

Understanding Gabapentin’s Mechanism Against Cancer

Scientists believe they may have identified how gabapentin works against glioblastoma tumors. The study revealed that patients taking the medication had lower levels of serum thrombospondin-1, a protein that appears to play a role in tumor growth. This reduction suggests gabapentin may be disrupting crucial pathways that allow these aggressive brain tumors to proliferate. The research was inspired by previous mouse studies that initially demonstrated gabapentin’s potential in targeting tumors, providing a scientific foundation for the clinical observations in human patients.

“This study is an exciting step forward,” said Joshua Bernstock, MD, PhD. “Glioblastoma is a relentlessly progressive and nearly universally fatal disease. The discovery that an already approved drug with a favorable safety profile can extend overall survival represents a meaningful and potentially practice-changing advance.” ASCO Post

Gabapentin was initially approved by the FDA in 1993 for treating seizures and later for managing nerve pain following shingles. Its safety profile is well-established, with common side effects including fatigue, headache, dizziness, and nausea. This existing safety data and wide availability could accelerate its potential adoption for glioblastoma treatment, assuming further studies confirm these preliminary findings. The drug’s mechanism against glioblastoma represents an innovative example of what researchers call “cancer neuroscience” – exploring the connections between neural pathways and tumor progression.

Promise Tempered with Caution

Despite the encouraging results, researchers emphasize that significant work remains before gabapentin can be recommended as a standard treatment for glioblastoma. The study’s retrospective nature means patients were not given the drug in a controlled, randomized manner to directly assess its effects. This limitation, while not invalidating the findings, necessitates additional research through properly designed clinical trials. Glioblastoma’s devastating 6.9% five-year survival rate makes even modest improvements in treatment outcomes potentially life-changing for patients.

“There have been very few advances in survival among patients with glioblastoma since the early 2000s,” explained Dr. Bernstock. “Ultimately, our goal was to highlight the emerging role of cancer neuroscience in GBM progression and emphasize the importance of exploring creative strategies to therapeutically target this evolving neural-tumor axis.”

The study was funded in part by the DFCI/Kiki Leptomeningeal Disease Grant and has drawn significant attention from the medical community. If confirmed through further research, gabapentin could represent a rare victory against glioblastoma, , widely available medication offering meaningful survival benefits without the extensive development timeline of entirely new drugs. For patients and families affected by this devastating diagnosis, even an additional four to six months of survival could provide invaluable time for treatment, memory-making, and improved quality of life.