Doctors in the UK are hoping to transform the lives of people with deadly bile duct cancer.
A new study – which has shown promising early results – matches the patient’s tumour to one or more of seven key medicines to drive the disease back.
Some people on the drugs have seen their cancer go into remission, while others have had previously inoperable cancer become suitable for surgery.
Doctors say the study will lead to a set of new standards for treating the disease, which currently kills the majority of patients in around a year.
The UK arm of the international work is being run by University College London Hospitals (UCLH) and University College London (UCL), where around 40 people have so far received treatment.
Overall, the study aims to recruit 800 people from all over the world.
The cancer may not cause symptoms in the early stages but can lead to jaundice, changes in urine and stool colour, itchy skin and weight loss.
In the new study, patients diagnosed with the three main types of bile duct cancer (intrahepatic, perihilar or distal cholangiocarcinoma) or with cancer of the gallbladder could be eligible to take part in the SAFIR-ABC10 trial.
They will have their tumours genetically profiled and will then be offered one or more of seven different anti-cancer therapies best matched to their tumour.
UCLH consultant medical oncologist and UCL Cancer Institute clinical researcher Professor John Bridgewater said: “With the current standard of care, patients typically only live for one year after treatment begins.
“It has become increasingly more urgent for us to try to identify more innovative and effective alternative treatment options.
“This is the first precision medicine study for patients with cancer in the biliary tract and the first time this patient group will be offered these anti-cancer therapies.
“The SAFIR ABC10 study resolves this problem by providing seven different therapies which we can match up with the specific ‘targets’ found in each patient’s tumour.”
Prof Bridgewater said the outlook for bile duct cancer patients can be poor and people are often “extremely unwell” at the point their cancer is discovered.
He told the PA news agency that data suggests up to 50% of patients “get absolutely no treatment”.
However, he said that for “some of the patients that we will be able to identify as part of this study, the outlook can be incredibly good – several years, if not more, of extremely good quality of life survival, even if your cancer is incurable.
“That’s the goal for at least some of our patients and that’s what we’re aiming towards.”
Prof Bridgewater said he hopes the new study will show medics and patients that a lot can be done for this type of cancer.
While the drugs may extend lives, some of them have been shown in preliminary work to “offer long-term remission of five years or more, which, by Cancer Research UK guidelines is a cure,” he said.
“We know because we’ve done it in preliminary studies that, because some of these new treatments work so well, at least some of these patients are converted from being inoperable to operable.”
The new trial will build on earlier research to introduce the drugs at the point treatment starts.
At least two of the drugs have passed medical approval and the hope is the study will strengthen the case for more to be used in the NHS.
Prof Bridgewater said a key part of the study is genetic profiling before treatment begins, adding that the current NHS genetic profiling of tumours is “lamentable”.
The new trial will involve giving people chemotherapy and the drug durvalumab.
One or more of the following drugs will also be offered depending on the genetic profile of the tumour: futibatinib, ivosidenib, zanidatamab, trastuzumab, neratinib, encorafenib and binimetinib.
“There is a pressing need for new treatment avenues and, through SAFIR ABC-10, we’re proud to support one of the first precision medicine trials for biliary tract cancers.
“With trials like this, we’re accelerating the development of more personalised, genetically targeted treatments that are more likely to be effective against cancer, so that more people can live longer, better lives free from the fear of this disease.”
Around 5,000 to 6,000 people every year are diagnosed with bile duct cancer but medics suspect the true figure is higher.
The number of deaths each year is roughly the same.
Ronald Glover, 76, from Chislehurst, south-east London, began treatment on the trial in July under the care of Prof Bridgewater.
He said he suffered “no side-effects at all”, with his first scan showing the treatment had cut his tumour in size by 36%.
The second phase of treatment involved 20 weeks of the drug ivosidenib.
“After about six weeks, I had another CT scan which found that my tumour had shrunk down to 30mm, now almost 50% down from the initial measurement,” Mr Glover said.
“I am now eight months down the line, fit and well, and have another scan at the end of this month.”
