Diabetes Drug Offers Hope for Families with Rare Cancer-Causing Genetic Condition

Tom Broder

November 23, 2021

Researchers at Oxford University are undertaking the world’s first cancer prevention study in patients with Li Fraumeni syndrome (LFS), a rare genetic condition linked with a high lifetime risk of developing cancer.

Investigators hope that the widely-used diabetes drug metformin may be able to prevent or delay the emergence of cancer in people with LFS.

If successful, metformin would be the first effective intervention available for families with LFS.

Pan Pantziarka, whose son George died from an LFS-related cancer in 2011, explains: "Many LFS families have to deal with more than one person having cancer at a time. We have families where parents have put their own treatment on hold because one of their children gets cancer."

"A treatment that reduces that risk will make a world of difference to our families - it's something that we desperately need."

New Role for Metformin

Li Fraumeni syndrome affects around 1 in 20,000 people worldwide. It is caused by mutations in the TP53 tumour suppressor gene, which plays an important role in preventing cancer development in cells.

People with LFS have a 70% to 90% lifetime risk of developing a range of cancers, including cancers of the brain, breast, blood, and soft tissue. Currently the only option for people with LFS is intensive regular screening in the hope of catching emerging cancers early.

Metformin is a well-established and safe oral blood-sugar lowering drug that has been used to treat type 2 diabetes for over 60 years.

Previous experiments in mice have shown that metformin can inhibit mitochondrial metabolism to delay cancer occurrence. Researchers hope that these findings may translate to human models, providing an effective treatment for people with LFS.

"There is a huge unmet need in the LFS population," says Dr Sarah Blagden, Chief Investigator on the Oxford University study. "We hope that the outcomes of this trial will provide the first, and much needed, intervention option for families diagnosed with LFS."

International Collaboration

The £2 million Metformin in Li Fraumeni (MILI) trial is a randomised open-label Phase II trial funded by the National Institute for Health Research and the Medical Research Council. The organisers hope to recruit over 200 adults with LFS in the UK.

The trial is being run as part of a global collaboration, with parallel studies in Canada, Germany, and the US. Results will be pooled to provide the world’s largest data resource on LFS.

The George Pantziarka TP53 Trust, the UK's only charity dedicated to supporting families with Li Fraumeni syndrome, has been closely involved in the trial’s development. The charity will provide funding for travel costs for people taking part in the trial.

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