Off label uses of metformin
Pro Research Analysisby 
Searched over 200M research papers
Off-Label Uses of Metformin
Metformin in Type 1 Diabetes
Reducing Insulin Requirements and Cardiovascular Risks
Metformin is frequently used off-label in type 1 diabetes to reduce the required insulin dose. It has been shown to improve glucose control in overweight and obese individuals, although evidence is limited. The REMOVAL trial suggests that metformin can reduce atherosclerosis progression, weight, and LDL-cholesterol levels, potentially lowering long-term cardiovascular disease risk.
Metformin for Polycystic Ovary Syndrome (PCOS)
Hormonal and Metabolic Benefits
Metformin is a widely recognized off-label treatment for PCOS, helping to manage insulin resistance, hirsutism, and acne. It is also used to improve ovulation, either alone or in combination with clomiphene citrate. However, its anti-androgenic effects are inconsistent. Metformin is effective in reducing the risk of ovarian hyperstimulation syndrome and increasing live birth rates in IVF/ICSI cycles .
Comparison with Myo-Inositol
A meta-analysis comparing metformin with myo-inositol (MI) in PCOS patients found no significant differences in fasting insulin, HOMA index, testosterone, SHBG levels, or BMI. However, metformin was associated with a higher risk of adverse events, making MI a more tolerable option for some women.
Metformin in Obesity and Insulin Resistance
Adolescents and Long-Term Effects
Metformin is used off-label to treat obesity and insulin resistance in adolescents. Studies show that while metformin can initially decrease BMI and HOMA-IR, its long-term effects are less clear, with some studies indicating no sustained benefit after prolonged use . Compliance and dosage issues may contribute to these mixed results.
Weight Management in Autism Spectrum Disorder
In children and adolescents with autism spectrum disorder who are on atypical antipsychotics, metformin has been shown to stabilize weight gain. Participants in a study maintained prior decreases in BMI z-scores, although no additional weight loss was observed during an open-label extension.
Metformin in Cardiovascular Health
Cardioprotective Effects
Metformin has been found to offer cardiovascular benefits beyond glucose reduction. It reduces the risk of reinfarction and all-cause mortality in patients with coronary artery disease and congestive heart failure (CHF). These effects are thought to be mediated through increased AMPK phosphorylation and enhanced nitric oxide production in cardiomyocytes .
Metformin in Cancer Prevention and Treatment
Antitumor Activity
Population studies suggest that metformin reduces the incidence and mortality rates of cancer in diabetic patients. It is being explored as a potential adjunct to chemotherapy or targeted therapy due to its antitumor mechanisms, which include effects on the PI3K/AKT/mTOR pathway .
Conclusion
Metformin's off-label uses extend beyond its primary role in managing type 2 diabetes. It shows promise in treating type 1 diabetes, PCOS, obesity, cardiovascular diseases, and even certain cancers. While its benefits are well-documented in some areas, further research is needed to fully understand its long-term efficacy and safety in these diverse applications.
Sources and full results
Most relevant research papers on this topic