As is known to all, metformin HCL has been helping patients with Type 2 diabetes control their blood sugar for more than two decades. Most 2A-grade and 3A-grade hospitals in China use metformin as the double or triple combinations in relevant prescriptions, of which the frequency of use and dose are no less than the insulin.

Researchers led by McGill University are investigating a new use for metformin: to treat some forms of autism caused by fragile X syndrome (mental retardation).

Note: Martin and Bell found 11 male patients and 2 mild mental retardation females in two generations of one family in 1943 and considered the mental retardation of the family to be X linked, therefore X mental retardation is also called Martin-Bell syndrome.

The incidence of this disease in males is second only to Down syndrome, mainly manifesting as moderate to severe mental retardation; some patients have hyperactivity, aggressive behavior or autism, moderate to severe mental retardation, and language and behavior disorders.

In the laboratory, researchers selected some mice which lost mouse socialization (having communication barriers with own kind in behavior) and showed typical fragile X syndrome (mental retardation). But within 10 days of being injected with metformin, their brain connections and behaviors were normal, therefore, researchers delightedly published the findings in the journal Nature Communications.

According to the article: Fragile X is an inherited genetic disorder that causes excess protein production in the brain and improper connections between neurons. The disease impairs speech, behavior and social interactions and sometimes coexists with anxiety and seizures.

However, the researchers observed that metformin restored molecular pathways that became disrupted by the defective gene, called the fragile X mental retardation 1 gene (FMR1). They’re still trying to figure out exactly how the drug acts on those pathways, but their animal experiment data suggest that metformin work in autism.

This result made the Albert Einstein College of Medicine in New York (one of the top diabetes and cancer hospitals in the U.S.) determined in using metformin to study and treat patients suffering from many diseases, including cognitive impairment and heart disease, to see if the drug can forestall comorbidities of the diseases.

The McGill researchers understand that simply observing behavioral changes in mice of fragile X doesn’t guarantee that metformin is a wonder drug they want. But the safety of the drug is well-established, and they need to do more work to establish a clinical trial system that can fast track mental retardation autism, to understand how exactly the drug is operating in the brain.

Note: The good news is that it is not the first time that metformin has led researchers to go "off course" in drug research. Previous observational study results have suggested that the cancer risk of diabetes patients who take this drug is reduced, which caused several scientific teams to shift from the original mission of studying diabetes treatment to finding out whether metformin really has anti-tumor effects.

In 2016, among those scientists, scientists at the University of California in San Diego published research suggesting that metformin fortifies epithelial cells, creating a barrier for the cells against cancer-causing substances. By tightening up the junctions between those cells, the drug helps create a barrier against inflammation, toxins and other cancer-causing substances. They succeeded in raising USD 15 million investment to develop compounds similar to metformin to deprive cancer cells of grow inducement.

By: Chen Bin