New pill spares women trauma of liver transplant

A NEW drug could save thousands of chronically ill British women from liver transplants.

In the UK, 20,000 people – 90 per cent of them women – suffer from primary biliary cholangitis, also known as primary biliary cirrhosis (PBC).

The illness sees the body’s immune system attack the bile ducts connecting the liver and the gut, leading to a build-up of bile in the liver which over time causes cirrhosis.

It has been said that it gives sufferers ‘the liver of an alcoholic’ even though they typically do not drink at all as they find alcohol intolerable.

The currently available drug treatment is only effective in 60 per cent of patients, which has led to PBC becoming the main cause of liver transplantation in British women.

A majority of patients are diagnosed aged 40 to 60, after showing initial symptoms of fatigue, dry eyes and mouth, arthritis, and pain in the upper stomach.

The cause of PBC is not clear, but it is believed to be an autoimmune disease triggered by infection, and has been linked to both genetics and pregnancy.

Until now, a drug called ursodeoxycholic acid (UDCA) has been the only one approved to treat PBC but 40 per cent of patients either do not respond to the drug or cannot take it.

These patients are more likely to have been diagnosed at a younger age, in their 30s or 40s. For them, liver transplantation has been the only alternative.

The new British-developed drug, Obeticholic acid, sold under the name Ocaliva, is the first treatment to be approved in the EU for patients with PBC in nearly 20 years.

It is being considered for use by the Scottish Medicines Consortium.

The £79.50-a-day drug will be given either in combination with UDCA or alone for patients unable to tolerate UDCA. The drug, which was approved by NICE for use in England only this month, works by activating a receptor in the liver and intestine which regulates bile acids. The most recent trial, published in August 2016, found that 50 per cent of PCB patients responded completely to the drug and 90 per cent had at least some benefit from it.

Collette Thain, chief executive of the PBC Foundation, said: ‘The approval of Ocaliva for treating people in the UK is extremely exciting news for PBC patients.

‘Liver transplantation is a distressing prospect for many people living with PBC, as nearly four in every ten patients do not respond to the current treatment. Ocaliva provides them with additional hope.

‘When I was diagnosed with PBC there was no alternative, or additional treatments, to the current therapy.

‘Fatigue doesn’t even begin to cover the symptoms: it was like walking through tar.

‘I had younger children and couldn’t even lift them. It would hurt when someone would touch me – imagine being in pain from hugging your child.’

Professor David Jones, director of UK-PBC Consortium who led the research said: ‘PBC affects thousands of ordinary people but it is rarely talked about, partly because there is a false notion that liver disease is linked to alcohol.

‘The 30 to 40 per cent of PBC patients who need Ocaliva are mostly those who have had an early presentation of the disease, and by default from suffering longer they are more likely to need a liver transplant.

‘This drug will make a difference to these people both because they will be getting better but also because they won’t face having a transplant – which in addition benefits the NHS.’