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Patients praised the revolutionary work of the National Amyloidosis Centre (NAC) at the Royal Free Hospital ahead of World Amyloidosis Day last week.
The NAC hosted three patients who shared their stories of amyloidosis and their work for Amyloidosis UK, a patient-led charity aiming to support those who receive an amyloidosis diagnosis.
The rare condition is characterised by the body’s inability to break down the amyloid protein. This protein then accumulates around the organs and stops them functioning normally. Whilst amyloidosis cannot be cured, recent treatments can help slow the disease progression.
The specialist service at the NAC receives referrals from all over the world and is at the forefront of research into amyloidosis diagnosis and treatment. The centre has also become the second largest recruiter for research at the Royal Free London (RFL).
David Gregory, 64, is one of 6,000 patients seen by the NAC each year and is raising awareness of amyloidosis as secretary of Amyloidosis UK.
He said: “We’re a small charity and all of our trustees are patients. At Amyloidosis UK, we aim to help and support newly-diagnosed patients in talking about their disease and are working on building a network of support groups. I’ve been diagnosed for seven years now, after my sister received a diagnosis of hereditary amyloidosis.
“Back then, there was minimal treatment available. But fortunately, over the last three to four years, new treatments have come along and mine is slowing my amyloidosis down – it's fantastic. We’ve had great treatment here at the RFL.”
Vince Nicholas, treasurer of Amyloidosis UK, has been visiting the NAC for a hereditary amyloidosis, ATTR amyloidosis, for over fifteen years.
He said: “My mum was one of the first people to be seen in the NAC in 1991. I received my diagnosis in 2009 after first showing symptoms. I’ve since lost several of my cousins and my twin brother to the disease – I feel very lucky to still be here. Receiving a diagnosis can take you to a very dark place. It's difficult, but having new treatments has given us a lot of hope.”
Before new drug treatments were developed, the most common treatment for ATTR amyloidosis was liver transplantation.
Vince shared: “I had a liver transplant in 2010 and have since seen the changes that having drug treatments available has made to those with amyloidosis. I've been a patient advisor for all the currently approved drugs for amyloidosis treatment, which is amazing. The support from the NAC has been fantastic. Without that, I don’t know what we would do – we wouldn’t be here.”
Paul Pozzo, 74, chair of Amyloidosis UK, shared his story: “I was on an open-top bus in America in 2015 when I first had symptoms – I felt breathless but it passed. Then, when I returned home to the UK, I couldn't breathe when I lay down to go to sleep. My wife phoned 111 and I went to hospital, where they found a litre and a half of fluid around my heart and lungs. Three months later, I visited the NAC and they diagnosed me with wild-type amyloidosis."
Paul took part in a drug trial and the treatment is slowing down the progression of his amyloidosis. He said: “Back in 2015 there was no drug to treat amyloidosis but here I am, telling the story. I believe there will be a cure for this terrible disease. I rely on, and have every faith in, the doctors here – they’re all marvellous.”
Professor Julian Gillmore, research lead at the NAC, said: “The range of drug treatments which have been developed in the last few years has revolutionised outcomes for patients. We’ve always been a very active research unit and I am proud to say that we have made important contributions to the advances in amyloidosis treatment over the last 20 years.
"We have over 1,000 patients who, in the last three months, have been prescribed the latest NICE-approved treatment for cardiac ATTR amyloidosis. It’s incredible to be able to offer patients treatment options and to see them doing well. For those with hereditary amyloidosis, it now means there’s hope for other family members as well. I feel very privileged to be a part of it all.”
Picture (left to right): Patients Vince Nicholas, David Gregory, Paul Pozzo and Professor Julian Gillmore.
