A common diabetes medication could help prevent one of the most aggressive types of blood cancer, according to research led by a Cambridge University team and published in the journal Nature on Thursday.
The study found that metformin, a safe and affordable diabetes drug, may prevent the development of acute myeloid leukemia (AML) in high-risk individuals by inhibiting the growth of blood cells with specific mutations.
Professor George Vasiliou, who led the international research team at the Cambridge Stem Cell Institute, said the findings could transform prevention approaches for blood cancers.
“Blood cancer doesn’t give us the luxury of surgical removal, as happens with other forms of cancer. Our only option is to stop it before it begins. That’s our goal: to identify those at risk early and prevent AML from developing using safe and simple means,” Vasiliou said.
The research, funded by Blood Cancer UK, Cancer Research UK, the Leukemia & Lymphoma Society (USA) and Wellcome Trust, focused on how clonal hematopoiesis—a condition where blood-forming cells acquire mutations in genes such as DNMT3A—can progress to leukemia.
Researchers demonstrated that metformin inhibits the growth of these mutated cells in both animal models and human samples, potentially preventing disease progression.
The significance of the study was further supported by analysis of data from 412,000 UK Biobank volunteers, which showed individuals taking metformin had significantly lower chances of developing DNMT3A mutations.
Dr. Pavlos Kosteas, Executive Director of the Karaiskakio Foundation where Vasiliou serves as Scientific Director, called the study “a milestone in the global effort to prevent leukemia”.
The research paves the way for clinical trials aimed at using metformin as preventive therapy for individuals with increased genetic risk. Using an already approved and widely used medication significantly reduces the time and cost of establishing a new prevention strategy.
Vasiliou, who is Professor of Haematological Medicine at Cambridge University and a Haematology Consultant at Cambridge University Hospital, also holds positions at the Cyprus Cancer Research Institute and chairs the Board of Directors at the Centre for the Study of Haematological Malignancies.
