Bile duct cancer study may pave way for new treatments

Patients with bile duct cancer could be helped by a new class of experimental drug, a study has shown.

Scientists have discovered that a key pathway – known as wnt – drives tumour growth in bile duct cancer.

Experimental drugs that block the wnt pathway are already being tested in patients with other cancers. This is the first indication that bile duct cancer may also be treatable in this way. Researchers found that the treatment prevented the growth of bile duct cancer cells in the lab and shrank tumours in animals with the disease.

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They are now planning to test whether these drugs will be effective in patients. The bile ducts are a network of tubes that drain toxins from the liver.

Bile duct cancer – known as cholangiocarcinoma – is often diagnosed at an advanced stage, which makes it very difficult to treat with surgery. Cholangiocarcinoma typically does not respond to chemotherapy. Fewer than one in twenty patients will survive for five years after diagnosis. Around 1000 patients are affected each year in the UK.

The study was led by the University of Edinburgh and is published in the Journal of Clinical Investigation. It was funded by Cancer Research UK, AMMF: The Cholangiocarcinoma Charity, and the Medical Research Council.

Professor Stuart Forbes, of the MRC Centre for Regenerative Medicine (CRM) at the University of Edinburgh, said:

“We are excited by these results because these drugs are already being tested in clinical trials for other types of cancer and could be beneficial for patients with cholangiocarcinoma.”

Dr Luke Boulter, former post-doc at CRM and now junior group leader at the MRC Human Genetics Unit at the University of Edinburgh, added:

Identifying the signals that control bile duct cancer’s growth will allow us to design better treatments that are urgently needed.

Helen Moremont, Chairman Trustee of AMMF, The Cholangiocarcinoma Charity, said:

“This is potentially very exciting. Cholangiocarcinoma is an under-researched, much neglected but truly devastating disease, so it is good to see progress being made in novel areas. We are hopeful that this research work with Wnt will provide a real step forward towards a clinical trial and some long awaited possible improvements in treatment.” 

Notes to editors

The study was published in the Journal of Clinical Investigation on 17 February 2015. Details here.  A 6 minute video describing the findings in more detail can be found on the JCI website.