Study Provides Glimmer of Hope for Glioblastoma Patients

Introduction to Glioblastoma

Glioblastoma, the most common and deadly form of brain cancer in adults, has seen little improvement in overall survival rates over the last several decades. With about 12,000 new cases diagnosed each year in the U.S., it accounts for most primary brain tumors in adults. The prognosis is grim, with most patients living only 12 to 14 months after diagnosis, and 5.5 months after recurrence.

Breakthrough Study

A new study by Mass General Brigham researchers offers a glimmer of hope for patients with glioblastoma. The researchers found that glioblastoma patients who received a common anti-seizure and pain drug — gabapentin — ended up living longer.
The study, led by Joshua Bernstock, a clinical fellow in the Department of Neurosurgery at Brigham and Women’s Hospital, is an exciting step forward in the fight against this relentlessly progressive and nearly universally fatal disease.

Key Findings

The discovery that an already approved medication with a favorable safety profile can extend overall survival represents a meaningful and potentially practice-changing advance. 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.

Validation of Results

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.

Future Directions

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. There have been very few advances in survival for GBM patients since the early 2000s, and the need for creative thinking about the emerging biology in these tumors and how to target them is evident.

Conclusion

The study provides a glimmer of hope for patients with glioblastoma, a disease that has seen little improvement in overall survival rates over the last several decades. The findings suggest that gabapentin, a common anti-seizure and pain drug, may extend overall survival in glioblastoma patients. However, larger, randomized clinical trials are needed to confirm the results and fully understand the potential of gabapentin in the treatment of glioblastoma.

FAQs

• Q: What is glioblastoma?
  A: Glioblastoma is the most common and deadly form of brain cancer in adults.
• Q: What is the current prognosis for glioblastoma patients?
  A: Most patients live only 12 to 14 months after diagnosis, and 5.5 months after recurrence.
• Q: What did the study find?
  A: The study found that glioblastoma patients who received gabapentin, a common anti-seizure and pain drug, ended up living longer.
• Q: What are the next steps?
  A: Larger, randomized clinical trials are needed to confirm the results and fully understand the potential of gabapentin in the treatment of glioblastoma.
• Q: When was the study originally published?
  A: Originally Published: May 19, 2025 at 11:40 AM PDT