World's first cancer prevention trial to test diabetes drug in patients with high-risk genetic condition
23/11/2021
This article is more than two years old.
Oxford researchers will lead a £2m national cancer prevention trial to assess the benefit a diabetes drug has in patients with Li Fraumeni Syndrome (LFS), a genetic condition that affects one in 20,000 people worldwide and puts them at a 70-90 percent lifetime risk of cancer.
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.
It is the world's first cancer prevention trial in adults with Li Fraumeni Syndrome (LFS), a rare inherited disorder that currently impacts around 600 people in the UK. 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 percent lifetime risk of developing a wide range of cancers, particularly cancers of the brain, breast, blood and soft tissue.
Currently, the only option for people with LFS is to undergo intensive regular screening in the hope of catching and treating emerging cancers as early as possible. However, this 'watch and wait' strategy can create a high level of anxiety among patients with LFS patients and their families.
"There is a huge unmet need in the LFS population and... we have designed the MILI trial to help meet this need. It will be the world's largest study into LFS and the first ever cancer prevention study for this community," said Dr Sarah Blagden, the Oxford University Hospitals Honorary Consultant Oncologist leading the trial.
"We hope that the outcomes of the trial will provide the first, and much needed, intervention option for families diagnosed with LFS."
Read more about the MILI trial.