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Patients with rare cancers given hope by UK-led ‘drug-match’ trial

Lisa Park was 48 when she was admitted to A&E in 2019 with worsening bouts of abdominal pain. Her eventual diagnosis was unexpected. She was told she was suffering from gallbladder cancer, a rare but serious condition that usually only affects people in old age.

Surgery and chemotherapy eradicated the tumours, but after a few months the cancer returned. “I was given a year to live,” said Park. It was a grim diagnosis, yet her prospects have recently taken an unexpected turn for the better.

Park – who is married and lives in Edinburgh – was recently asked to join a revolutionary UK research programme known as Determine. It has has been set up to target individuals with rare tumours in the hope that drugs licensed for more common cancers could be appropriated as a new treatment.

“By definition, rare cancers are uncommon,” said one of the project’s leaders, oncologist Matthew Krebs of Manchester University. “However, there are so many of them that in total they represent around 20% of all cancers that are diagnosed globally each year – more than any single type of cancer including lung, breast and colorectal cancer.

Rare cancers’ total impact on the health service is therefore significant but there are few treatments currently available to help patients. Determine has been set up to find if drugs that are already prescribed by doctors for more common types of cancer could benefit patients with rare cancers for which they have not been licensed.

The study is led by Manchester University, sponsored by the Cancer Research UK Centre for Drug Development, and involves several other leading medical centres in the UK. The ultimate aim is to match old drugs for new cancers.

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The Christie NHS Foundation Trust in Manchester where Lisa Park’s rare cancer was treated.The Christie NHS Foundation Trust in Manchester where Lisa Park’s rare cancer was treated. Photograph: John B Hewitt/Shutterstock

The crucial point is that, with recent advances in DNA analysis, scientists have discovered some very rare tumours possess abnormalities that could make them susceptible to treatments developed for common cancers. “We will be able to fast-track the approval of any promising drugs, opening the door to treatments for patients who have historically been left with limited options,” said Iain Foulkes, the executive director of research and innovation at Cancer Research UK.

The approach has already produced promising results for Lisa Park. She has been given a treatment at the Christie NHS Foundation Trust in Manchester that had been developed for some breast cancers, and these have reduced her tumours, according to recent scans. “I know I still have a journey ahead of me but my cancer has been reduced to manageable tumours, and that has been a very welcome outcome,” said Park.

Nor is she alone. Another patient, Simon Robey from Nottingham, was recently diagnosed with the rare cancer of the salivary gland. Despite surgery and radiotherapy, the cancer spread, so he was asked to join Determine. “After tests, I was given a drug that was usually used to treat breast cancer and in my case the response has been superb, with scans showing great improvements. It is like a fairytale. This drug is keeping me alive.”

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Krebs told the Observer that it is hoped that several hundred people – from very young cancer sufferers to the elderly – can benefit from Determine.

“It is not just this country that could benefit,” added Krebs. “We might find five patients of a certain rare cancer in the UK that have all done really well with the targeted treatment. That is only five patients but it might be that the Netherlands or Norway or France also have a handful of patients from the same subgroup that have responded to the same treatment, and we want to pull those data all together.

“This could have a major international impact in the end.”