Glioblastoma patients taking common pain drug gabapentin lived up to six months longer, potentially revolutionizing treatment for this deadly brain cancer.
Key Takeaways
- Gabapentin, a drug commonly prescribed for seizures and nerve pain, may extend survival in glioblastoma patients by approximately four months
- A study of 693 glioblastoma patients showed those taking gabapentin survived 16 months versus 12 months for non-users
- In newly diagnosed patients, the survival advantage was even more pronounced: 20.8 months for gabapentin users versus 14.7 months for non-users
- Researchers discovered lower levels of thrombospondin-1 protein in gabapentin users, potentially explaining its anti-cancer effect
- While promising, these findings require confirmation through larger, randomized clinical trials before changing standard treatment protocols
Common Pain Medication Shows Unexpected Cancer-Fighting Properties
A groundbreaking study from Mass General Brigham researchers has revealed that gabapentin, a medication commonly prescribed for seizures and nerve pain, may significantly extend survival in patients battling glioblastoma, one of the deadliest forms of brain cancer. The retrospective analysis examined nearly 700 glioblastoma patients, many already taking gabapentin for nerve pain, and discovered those receiving the drug lived approximately four months longer than patients not on the medication. This represents a potentially major advancement in treatment options for a cancer with dismal survival rates.
The research, published in Nature Communications, analyzed 693 glioblastoma patients and found that those receiving gabapentin treatment survived approximately 16 months compared to just 12 months for those not taking the drug. Even more encouraging, a separate analysis of 379 newly diagnosed patients showed an even more pronounced benefit – gabapentin users survived 20.8 months versus 14.7 months for non-users. These findings offer hope for patients facing a disease that currently has a five-year survival rate of only 6.9% and has seen few treatment advances in over two decades.
The Science Behind Gabapentin’s Anti-Cancer Effects
Researchers discovered an intriguing biological mechanism that may explain gabapentin’s effect on glioblastoma. Patients taking the drug showed lower levels of serum thrombospondin-1 (TSP-1), a protein associated with cancer progression. This reduction may help decrease tumor aggression and improve survival outcomes. The study was inspired by previous mouse studies that demonstrated gabapentin’s potential in targeting tumors, suggesting the medication may disrupt the neural-tumor communication pathways that help cancer cells thrive and spread.
“This study is an exciting step forward,” said Joshua Bernstock, MD, PhD. “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 research is particularly promising because gabapentin is already FDA-approved, with a well-established safety profile and relatively mild side effects. Common side effects include fatigue, headache, dizziness, and nausea. First approved by the FDA in 1993 for seizures and later for nerve pain following shingles, the drug is widely available and could potentially be repurposed quickly if further studies confirm its effectiveness against glioblastoma.
Cautious Optimism and Next Steps
Despite the encouraging results, researchers emphasize that the retrospective nature of the study has limitations. Since patients were not given gabapentin in a controlled, randomized manner specifically to assess its effects on cancer, further research is necessary before changing clinical practice. Larger, prospective clinical trials will be needed to definitively establish gabapentin’s safety and effectiveness as a treatment specifically for glioblastoma patients.
“[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,” stated Joshua Bernstock, MD, PhD. “Across both cohorts (1,072 patients total), gabapentin use was consistently associated with a statistically significant improvement in survival.”
For patients and families affected by this devastating diagnosis, these findings provide a glimmer of hope in what has been a relatively stagnant treatment landscape. If confirmed through rigorous clinical trials, gabapentin could represent the first significant advancement in glioblastoma treatment in decades. The study, partially funded by the DFCI/Kiki Leptomeningeal Disease Grant, highlights the potential benefits of repurposing existing medications – a strategy that could accelerate the development of new cancer treatments while avoiding the lengthy and expensive process of creating entirely new drugs.
