Researchers from the University of Oxford will undertake the world’s first cancer prevention trial in adults with Li Fraumeni Syndrome (LFS). The “Metformin in Li Fraumeni” (MILI) trial is a randomised open-label Phase II trial jointly funded by the National Institute for Health Research and the Medical Research Council that will evaluate whether the existing type 2 diabetes drug metformin can prevent or delay the emergence of cancer in people with LFS.
LFS is a rare inherited disorder that currently impacts around 600 people in the UK but, with increasing use of self-testing and genetic sequencing in diagnostic practice, this number is expected to rise. LFS is caused by mutations in the TP53 tumour suppressor gene that encodes an important protein that prevents cancer. People with LFS have a 70-90% lifetime risk of developing a wide range of cancers, particularly cancers of the brain, breast, blood and soft tissue.