A common pain medication may add months to the lives of brain cancer patients, offering new hope in a disease with few treatment advances in decades.
Key Takeaways
- Gabapentin, a widely used medication for pain and seizures, was associated with a four-month survival increase in glioblastoma patients
- Newly diagnosed patients taking gabapentin survived 20.8 months compared to 14.7 months for non-users, a significant 41% improvement
- The drug appears to work by reducing levels of thrombospondin-1, a protein linked to tumor progression
- As an already FDA-approved medication with a known safety profile, gabapentin could potentially be rapidly implemented in treatment plans if confirmed by clinical trials
- Glioblastoma remains one of the deadliest cancers with a five-year survival rate of just 6.9%, making any survival improvement significant
Common Medication Shows Remarkable Promise Against Deadly Brain Cancer
Researchers at Mass General Brigham have discovered that gabapentin, a medication commonly prescribed for nerve pain and seizures, may significantly extend the lives of patients with glioblastoma, one of the most aggressive forms of brain cancer. The retrospective study analyzed 693 glioblastoma patients and found that those who took gabapentin survived an average of 16 months compared to just 12 months for those who didn’t take the drug. This four-month improvement represents a meaningful advancement in a disease that has seen few treatment breakthroughs in decades.
Even more promising results emerged when researchers examined a subset of 379 newly diagnosed patients. Those taking gabapentin survived an average of 20.8 months compared to 14.7 months for non-users – a 41% improvement. Across the entire study population of over 1,000 patients, the survival benefit remained statistically significant, suggesting that the effect is robust and not merely a statistical anomaly. The findings were recently published in the prestigious journal Nature Communications.
Understanding the Science Behind Gabapentin’s Effect
The mechanism behind gabapentin’s apparent anti-cancer properties involves the reduction of a protein called thrombospondin-1 (TSP-1), which is known to play a role in tumor progression. Patients taking gabapentin showed lower serum levels of TSP-1, suggesting this could serve as a potential biomarker for treatment response. This discovery didn’t come out of nowhere – it was built on previous laboratory studies showing gabapentin’s potential to target tumors in mice, demonstrating the value of translational research that bridges laboratory findings with clinical observations.
“This study is an exciting step forward,” said Joshua Bernstock, MD, PhD, neurosurgery resident at Mass General Brigham and the study’s lead author.
Gabapentin was initially approved by the FDA in 1993 for seizures and later expanded for use in treating nerve pain following shingles. Its well-established safety profile and widespread availability make it particularly attractive as a potential cancer therapy. Common side effects include fatigue, headache, dizziness, and nausea – concerns that are relatively manageable compared to many dedicated cancer treatments. This existing safety data could potentially accelerate its implementation if clinical trials confirm these early findings.
A Ray of Hope for a Devastating Diagnosis
Glioblastoma represents one of the deadliest challenges in oncology. It is characterized by rapid growth, resistance to treatment, and nearly universal fatality. With a five-year survival rate of just 6.9%, even modest improvements in survival time are considered significant victories. Many patients in the study were already taking gabapentin for nerve pain – a common symptom among brain cancer patients – which allowed researchers to observe its potential secondary effects on cancer progression.
“[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,” said Joshua Bernstock, MD, PhD.
The researchers emphasize that while these findings are promising, their retrospective nature represents a significant limitation. Unlike a controlled clinical trial, patients were not randomly assigned to receive gabapentin, meaning other factors could potentially influence the results. Nevertheless, the consistency of the survival benefit across different patient subgroups strongly suggests that the effect is real and worthy of further investigation in more rigorous clinical trials.
The Path Forward
Medical researchers are cautiously optimistic but appropriately restrained in their enthusiasm. Before gabapentin can be recommended as a standard treatment for glioblastoma, controlled clinical trials are necessary to definitively establish its efficacy and optimal dosing. The study was partially funded by the DFCI/Kiki Leptomeningeal Disease Grant, highlighting the importance of continued research support in this challenging field. If confirmed, the findings could represent a significant victory in the fight against a disease that has long defied medical advances.
“While the findings are promising, the study is retrospective — patients were not given gabapentin in a controlled, randomized manner to directly assess its effects,” cautioned Joshua Bernstock, MD, PhD.
Perhaps most encouragingly, this discovery represents a potential breakthrough using an existing medication rather than an entirely new compound. Drug repurposing – finding new uses for approved medications – can dramatically reduce the time and cost of bringing treatments to patients. With gabapentin already widely available and its safety profile well-established, implementation could be relatively swift if clinical trials validate these initial findings, offering new hope to patients facing one of medicine’s most formidable challenges.
