£3 diabetes pill offers hope on rare cancer

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The first human trials are set to take place in Britain to see whether metformin can treat Li Fraumeni Syndrome [LFS]. The little-known condition is devastating, putting sufferers at a 90 per cent risk of developing cancer.

The £2million study – set to start in 2023 – will test whether the £3 blood sugar-lowering drug can delay or prevent onset.

If effective it would be the first fully-tested cancer prevention treatment for sufferers.

Chief researcher Sarah Blagden, associate professor of experimental cancer therapeutics at Oxford University, said: “There is a huge unmet need in the LFS population. It will be the largest study into LFS and the first cancer prevention study for this community.

“We hope the outcomes will provide the first, and much needed, intervention option for families diagnosed with LFS.”

It is a rare inherited disorder that affects around 600 people in the UK but, with increasing use of self-testing and genetic sequencing, this number is expected to rise.

Sufferers inherit a defective copy of a gene called TP53 that plays a key role in protecting cells from cancer. Without a working TP53 gene, those with LFS stand an almost certain chance of getting one or more cancers, particularly at a young age.

Sufferers stand a 70 to 90 per cent risk, particularly of developing cancer of the brain, breast, blood and soft tissue.

LFS families commonly suffer the tragedy of losing children or parents to cancer. The study will come too late for Pan Pantziarka.

He lost his wife Gina to ovarian cancer and within a year their son George was diagnosed with the first of three LFS-related cancers. He died aged 17.

Pan believes the metformin trials give hope to those whose lives have been wrecked by the condition. He is in regular contact with families through The George Pantziarka TP53 Trust, set up after George died.

He said: “Many LFS families have to deal with one family member after another becoming ill with cancer. In some cases, families are dealing with multiple cancers at the same time, and there is always the knowledge another may arise. Reducing the risk with a drug like this will make the biggest difference.”

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Currently sufferers rely on regular scans to tell them if they have cancer. 

The Metformin in Li-Fraumeni [MILI] trial, which will be jointly funded by the National Institute for Health Research and the Medical Research Council, will evaluate whether metformin can prevent or delay the emergence of cancer in people with LFS.

It is the latest repurposing trial testing the potential of a drug to treat a disease for which it was not originally intended.

Metformin, sold under brand names including Glucophage, is used to treat Type 2 diabetes, particularly for those who are overweight. It is also prescribed for polycystic ovaries. It lowers blood sugar levels by improving the way the body handles insulin.

In the trial, 200 people aged 16 and over will randomly receive metformin or a placebo, with all participants having regular scans, blood tests and assessments.

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