Metformin: risk-benefit profile with a focus on cancer

Expert Opin Drug Saf. 2015 Oct;14(10):1573-85. doi: 10.1517/14740338.2015.1084289. Epub 2015 Sep 11.


Introduction: Epidemiological evidence suggests an increased incidence of cancer in obese, prediabetic, and diabetic patients and a reduced risk of cancer incidence and mortality in diabetic patients on metformin compared with other antidiabetic drugs. In vitro studies support the efficacy of metformin in cancer therapy and prevention. Although metformin seems to be promising as a cancer chemopreventive or therapeutic drug, the principal consideration is whether metformin will be effective in cancer clinical trials for nondiabetic subjects or only in diabetics or subjects with insulin resistance. Safety of metformin is even more important in treating nondiabetic patients.

Areas covered: The present review focuses on epidemiological data and clinical trials testing the efficacy of metformin on cancer, the safety in nondiabetic patients and the future development of this promising drug.

Expert opinion: Meta-analyses of epidemiological in which metformin treatment has been used for diabetic patients show a positive trend for benefit; nevertheless, clinical data outcomes are preliminary and the results of ongoing trials are awaited. The different types of cancer, heterogeneity of populations and presence of comorbidity make it difficult to determine the benefits of metformin in cancer prevention and treatment.

Keywords: clinical trials; metformin; neoplasm; safety.

Publication types

  • Review

MeSH terms

  • Diabetes Mellitus / drug therapy
  • Humans
  • Hypoglycemic Agents / pharmacology*
  • Incidence
  • Insulin Resistance
  • Metformin / pharmacology*
  • Neoplasms / epidemiology
  • Neoplasms / etiology
  • Neoplasms / prevention & control*
  • Obesity / complications
  • Prediabetic State / complications
  • Risk Factors


  • Hypoglycemic Agents
  • Metformin