What is it about?

The study investigated the effects of metformin on non-muscle-invasive bladder cancer (NMIBC) marker lesions. In a phase II, open-label trial, 11 patients with low-grade Ta-T1 NMIBC were treated with metformin for 3 months. A complete response was observed in one patient, a partial response in another, while the majority showed no significant change, and some developed new lesions. The most common adverse event was mild diarrhoea, and no severe treatment-related adverse events occurred. Metformin concentrations were higher in urine than blood, but immunohistochemical analysis showed no changes in relevant markers. The study found no conclusive evidence for metformin's antitumor effect in bladder cancer.

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Why is it important?

This research is important because it evaluates the potential use of metformin, a commonly prescribed diabetes medication, as a treatment for non-muscle-invasive bladder cancer (NMIBC). The study explores an alternative therapeutic approach that could offer a more affordable and less invasive option compared to the existing treatments, which can significantly impact patients' quality of life and place a heavy burden on healthcare resources. Although the study did not find conclusive evidence of metformin's antitumor effects, it contributes valuable data to the ongoing search for effective NMIBC treatments and underscores the importance of exploring repurposed medications in oncology. Key Takeaways: 1. Limited Efficacy: The study found that metformin treatment led to a complete response in only one of 11 patients, with a partial response in one other, highlighting the limited effectiveness of metformin as a monotherapy for NMIBC in this trial. 2. Safety Profile: Metformin was generally well-tolerated by patients, with no serious adverse events reported, indicating its safety as an adjunctive treatment in the explored dosage and duration, though gastrointestinal issues like diarrhea were common. 3. Research Implications: Despite the lack of antitumor activity observed, the study provides a starting point for further investigations into the potential mechanisms by which metformin might affect cancer cells, encouraging continued research into drug repurposing for cancer therapy.

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This page is a summary of: A study of oral metformin for the intravesical treatment of non‐muscle‐invasive bladder cancer, BJU International, May 2025, Wiley,
DOI: 10.1111/bju.16767.
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