Surprise Drug Discovery Could Extend Life for Cancer Patients

ORION PRODUCTION

A new study from Mass General Brigham is drawing attention after revealing that a widely used pain and anti-seizure medication, gabapentin, may significantly increase survival time for patients battling glioblastoma—the most aggressive and fatal form of brain cancer.

The study, published in Nature Communications, tracked nearly 700 glioblastoma patients, many of whom were already taking gabapentin for nerve pain. Researchers found that those using the drug lived, on average, four months longer—16 months compared to 12 months—than those who did not.

That outcome, which researchers described as “statistically significant,” was enough to spark further inquiry. Lead author Dr. Joshua Bernstock, a neurosurgery fellow at Brigham and Women’s Hospital, said he was both surprised and encouraged by the results.

“It’s always incredible to see a hypothesis come to life,” Bernstock told Fox News Digital. He emphasized that the goal was to explore new ways of targeting the interaction between tumors and the nervous system, a growing area of research called cancer neuroscience.

Bernstock and his team collaborated with researchers at the University of California, San Francisco, to validate the findings in a second cohort of 379 glioblastoma patients. Again, the data revealed that those taking gabapentin lived longer—20.8 months versus 14.7 months.

Across both patient groups, gabapentin use correlated with a measurable drop in TSP-1, a protein found in blood serum that may be linked to tumor progression. Though the exact mechanism isn’t fully understood, researchers believe the drug may interfere with the tumor’s communication pathways, slowing its advance.

That discovery is especially meaningful for a disease that has seen little progress in treatment options over the last two decades. Glioblastoma has a notoriously grim prognosis, with a five-year survival rate of just 6.9% and around 14,500 deaths annually in the United States.

Gabapentin, first approved by the FDA in 1993 for seizures and later for nerve pain, is often prescribed off-label for a wide range of chronic pain conditions. The drug is generally well tolerated, though some patients may experience side effects like dizziness, nausea, or fatigue.

Importantly, the new study was retrospective, meaning it looked back at existing patient data rather than testing gabapentin in a controlled, randomized trial. That means more research is needed before doctors can formally recommend the drug as part of glioblastoma treatment.

“This is not a green light to change clinical practice just yet,” Bernstock cautioned. “But in patients already dealing with nerve pain or seizures, gabapentin could be a particularly smart choice given its potential added benefit.”

The UCSF team is reportedly moving forward with planning controlled clinical trials to confirm the findings.

While early, the results are offering a rare glimmer of hope for patients and families battling one of the most aggressive forms of cancer. In a field that has seen minimal therapeutic breakthroughs, a drug that’s already on pharmacy shelves may become a key weapon in extending lives.

As Bernstock put it, “We need to think more creatively about the biology of these tumors. This is one promising step in that direction.”