Genetic avatars identify new therapy for liver cancer

Researchers have discovered that a new combination of drugs can almost completely eradicate the most common type of liver cancer, hepatocellular carcinoma, in mice replicating cancer-causing DNA alterations.

Cancer is often caused by changes in our DNA, which cause cells to grow too quickly or in the wrong place.  

Led by Professor Tom Bird of the Institute for Regeneration and Repair and the Cancer Research UK Scotland Institute in Glasgow, the researchers replicated these cancer-causing DNA alterations in mice, creating genetic ‘avatars’ of hepatocellular carcinoma. 

These avatars were then treated with a range of drugs to identify the most effective treatment strategy.  

The team showed that cladribine, an existing cancer drug commonly used to treat leukaemia and multiple sclerosis, reduced the number of tumours in the avatar mice. However, it was most effective when combined with another drug called lenvatinib, completely eradicating almost all tumours. 

 Cladribine helps to stimulate the body’s own immune system to clear tumours, but had never been used for liver cancer before.  

 A clinical trial over a period of years is now required to confirm the results in liver cancer patients over time. 

Liver cancer in the UK

There are around 6,600 new liver cancer cases in the UK every year. Liver cancer incidence rates are significantly higher in Scotland than the UK average. 

Diagnosis of liver cancer is often late. Many patients are diagnosed only when already receiving treatment for existing diseases such as cirrhosis or fatty liver disease. This makes liver cancers hard to treat as, due to the function of the liver, the disease often responds poorly to chemotherapy drugs.

In the UK, fewer than half of those diagnosed with liver cancer survive their disease for a year or more, which makes finding new ways to tackle this disease vital. 

 This new research offers potential for more complex treatment regimes, known as precision medicine. These approaches treat patients’ individual liver cancers, improving their chances of success.  

This exciting discovery provides new hope for the thousands of people living every day with a liver cancer diagnosis. 

Finding new and effective ways to combine and use treatments already approved for other cancers may be a faster way to achieve successful outcomes for future patients. 

Taking a precision approach to treatment by tailoring therapies to the particular types of tumours based upon their genetic alterations, has the potential to transform how we understand, and treat, cancer.

Professor Tom Bird Lead Researcher

There are lots of exciting things happening in cancer research and I hear about it every time I am in for a check-up. I’ve been told that if the treatment I’m on stops working, there are other options, so I’m delighted to hear of new developments like this.

John O’Donnel Participant in an advanced liver cancer clinical trial

We are delighted to have funded this exciting research which could lead to new treatments and improved outcomes for patients with liver cancer. 

Liver cancer is a difficult cancer problem – it’s the fastest rising cause of cancer death in the UK and it can be hard to diagnose it at an early stage when treatment can be more effective. 

That’s why research like this is so important – it lays the foundations for improved cancer treatment, driving us towards a time when no one fears cancer.

Dr Sam Godfrey Cancer Research UK’s Science Engagement Lead

This research was published in the journal Nature in an article entitled "Human-correlated genetic models identify precision therapy for liver cancer".

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