Breakthroughs boost liver disease prospects

Primary Biliary Cholangitis (PBC) causes misery to thousands in the UK but medical research and the campaigning work of the PBC Foundation are improving diagnosis, treatment and disease management


Promoted by PBC Foundation

Hope is on the horizon for people living with Primary Biliary Cholangitis (PBC), a long-term auto-immune condition that causes acute liver damage and reduced quality of life.
The condition, which results in bile acid inflaming, damaging and scarring the liver, has no therapeutic cure and symptoms are often overlooked or misdiagnosed, which has historically led to an average five-year wait before effective treatment.

But a series of clinical breakthroughs and campaigns by the PBC Foundation are encouraging drug development and improving condition management by raising awareness among the public and medical profession.

There are in excess of 20,000 people in the UK living with PBC, many of whom are believed to be undiagnosed. This is because their main symptoms, usually fatigue, an itch under the skin, joint and muscle pain, and brain fog, are often ascribed to other conditions.

“The result is that many people endure these symptoms unaware of PBC, and their personal and work lives can suffer when they could be getting the help they need,” says Robert Mitchell-Thain, CEO at the Edinburgh-based PBC Foundation. “Their quality of life is impaired significantly and it is still taking too long for patients to reach a diagnosis.

“PBC affects nine times more women than men and many are told all sorts of nonsense before reaching a diagnosis, such as ‘we are all tired’ or even that they are going through the menopause. We are committed to raising awareness among the medical profession so they can make swifter diagnoses and patients can take ownership of the condition and lead fuller lives.”

The Foundation is a leading force for education and research around the condition. It is collaborating with pharmaceutical companies and regulators to improve the prospects of clinical trials and accelerate treatments and a potential cure.

“We need to highlight to healthcare professionals [HCPs] the extent of symptom burdens that people living with PBC experience,” says Mitchell-Thain. “We also need to empower patients with the confidence to discuss their quality of life with HCPs so it can be prioritised in their treatment.”

HCPs look to other conditions first to explain the fatigue, brain fog, and joint and muscle pain that characterise PBC but, when they do consider it, the standard antimitochondrial antibody blood test is 95% accurate at detecting the presence of PBC.

“That all said, disease progression is very different to symptom burden and we absolutely must tackle both aspects of this incurable disease,” says Mitchell-Thain.

With no cure, an early diagnosis is essential in ensuring patients can try existing first- and second-line therapies, and develop an effective management of their condition. Early diagnosis and successful treatment can lead to a normal life expectancy, however if either of these aspects are missing it can lead to deterioration that can eat into both physical and emotional wellbeing.

The foundation is leading work to create a clinical methodology that measures symptoms in a way that works for PBC patients, clinicians and regulators. It involves adapting tests known as the Global Clinical Impressions (CGI) scale, which measure symptom severity and are approved for other conditions.

The research work could lead to a stronger clinical trials framework that could see therapies developed at pace to provide effective management of the condition and its symptoms from an early diagnosis.

The foundation has supported research across the disease space, including developing a quality of life measurement tool and focusing on potential triggers that confuse the body’s immune system and increase the impact of symptoms on quality of life.

“We have 15,000 patients from 80 countries using our services and, because of the unmet need, we have a huge responsibility and opportunity to make a difference to their and their families’ lives,” added Mitchell-Thain.

“GPs rarely encounter PBC and the time taken to reach a diagnosis can really impair the management of the progression of PBC, particularly in terms of their quality of life and the psychological impact. We are working hard to change that and, although there is a lot still to be done, the signs of change are promising.”

For more information please visit pbcfoundation.org.uk

This article has been sponsored by the ADVANZ PHARMA Group.
ADVANZ PHARMA recommended the potential authors for this article, however, with the exception of a factual accuracy check, ADVANZ PHARMA has had no editorial control over the content of this article.

UK-NP-PB-0801, Date of Prep: July 22


Promoted by PBC Foundation