Repurposing metformin for cancer treatment: Current clinical studies

Young Kwang Chae*, Ayush Arya, Mary Kate Malecek, Daniel Sanghoon Shin, Benedito Carneiro, Sunandana Chandra, Jason Kaplan, Aparna Kalyan, Jessica K. Altman, Leonidas Platanias, Francis Giles

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

224 Scopus citations


In recent years, several studies have presented evidence suggesting a potential role for metformin in anti-cancer therapy. Preclinical studies have demonstrated several anticancer molecular mechanisms of metformin including mTOR inhibition, cytotoxic effects, and immunomodulation. Epidemiologic data have demonstrated decreased cancer incidence and mortality in patients taking metformin. Several clinical trials, focused on evaluation of metformin as an anti-cancer agent are presently underway. Data published from a small number of completed trials has put forth intriguing results. Clinical trials in pre-surgical endometrial cancer patients exhibited a significant decrease in Ki67 with metformin monotherapy. Another interesting observation was made in patients with breast cancer, wherein a trend towards improvement in cancer proliferation markers was noted in patients without insulin resistance. Data on survival outcomes with the use of metformin as an anti-cancer agent is awaited. This manuscript will critically review the role of metformin as a potential cancer treatment.

Original languageEnglish (US)
Pages (from-to)40767-40780
Number of pages14
Issue number26
StatePublished - 2016


  • Cancer
  • Clinical trials
  • Metformin

ASJC Scopus subject areas

  • Oncology


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