In a significant breakthrough in the field of drug repurposing, scientists have discovered that a widely used diabetes medication already taken by millions around the world may offer unexpected protection against a form of blood cancer.
The surprising benefit of metformin in helping to prevent acute myeloid leukemia (AML) among people at high risk of the disease has been uncovered by researchers at the University of Cambridge. The discovery could open new pathways for cancer prevention and offer a life-saving option for those most vulnerable to developing this aggressive blood cancer.
“Blood cancer poses unique challenges compared to solid cancers like breast or prostate, which can be surgically removed if identified early. With blood cancers, we need to identify people at risk and then use medical treatments to stop cancer progression throughout the body,” Professor Vassiliou, from the Cambridge Stem Cell Institute, who co-led the research, explained the relevance of the study.
In the study published in the journal Nature, the researchers investigated the most common genetic mutation linked to AML, which affects a gene called DNMT3A. Changes in this gene are known to initiate 10% to 15% of AML cases.
By targeting these early genetic changes, the team found that metformin could disrupt the disease process by interfering with the way pre-cancerous cells produce energy, effectively stopping them from growing and developing into leukemia. It also helps undo some of the damage caused by a faulty gene called DNMT3A, which is often linked to the disease.
When the researchers evaluated health data from more than 412,000 UK Biobank participants, they found that individuals taking metformin were less likely to show changes in the DNMT3A gene. This link persisted even after researchers accounted for other factors that could have influenced the results, such as diabetes or body mass index (BMI).
Metformin has a long-standing safety record, having been used by millions of people worldwide with minimal side effects. Since it’s already proven to be safe and well-tolerated, it could be repurposed more quickly and easily for leukemia prevention, avoiding the long and costly process of developing a new drug from scratch.
“Repurposing safe, widely available drugs like metformin means we could potentially get new treatments to people faster, without the need for lengthy drug development pipelines,” said Dr Rubina Ahmed, Director of Research at Blood Cancer UK, which funded the study.
