Amyloidosis Patient Information Site

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The National Amyloidosis Centre developed this website as a public service to provide patients and their family and friends with a comprehensive, accurate and up to date resource on all types of amyloidosis. We welcome your feedback.

Much information about amyloidosis is available on the internet.  Some of it is alarming and much of it can be confusing.  There are many different types of amyloidosis. Each type has different causes, different symptoms and different treatments.  In order to understand the disease you need to understand which type is present.

We suggest that first time visitors to this website start by reading the essentials section, then read the section about the type of amyloidosis that you or your relative has. The different types include AL ,  ATTR (which may be either hereditary or non-hereditary), AA and other, rarer hereditary types. All of these may be “systemic” which means that they affect many different parts of the body. Amyloidosis may also be “local” or “localised” which means that just a single organ or part of the body is affected. Local amyloidosis is usually of AL type and is a very different disease to systemic amyloidosis. Local amyloidosis may require no treatment or may be completely curable.

Although there is no cure for systemic amyloidosis at present there are several treatments which can be extremely helpful and prolong life for many years.  If you have been diagnosed with amyloidosis, or if a family member or friend has the condition, we hope that you will find all the information you need here.  The website is here to help you no matter what your stage of the disease.  New treatments are being researched and introduced more actively than ever before and are increasingly effective.  There are good reasons for optimism and it is important to remember that you are not alone.

Interview with Professor Sir Mark Pepys – 2018

Watch other interviews where Professor Sir Mark Pepys discusses some of his additional professional interests and achievements.

Interviews have been provided courtesy of Dr Anton Titov, Diagnostic Detectives

Update: changed and future roles in the Centre for Amyloidosis and Acute Phase Proteins

Professor Philip Hawkins stepped down as Director of the Centre on 31 July 2019 and was replaced by Professor Julian Gillmore.  Professor Hawkins returned in October to continue a central role, as one of the world’s most experienced amyloidosis physicians, expertly informing key aspects of the clinical work and clinical research in the Centre.  In particular, he will play a crucial role in maintaining the unique connection between the fundamental scientific discovery research in the Wolfson Drug Discovery Unit and the extraordinary clinical resources of the National Amyloidosis Centre (NAC).  This model of conjoined basic science and clinical practice, exemplified by the career of Professor Sir Mark Pepys himself, is the basis of the Centre Sir Mark established on coming to UCL in 1999.  It has been critical to our success in the past and will continue to be so in future.

Professor Julian Gillmore, whose MD project was supervised by Professor Hawkins and whose PhD project was supervised by Sir Mark, and who has been a senior member of staff in the NAC for nearly 20 years, is admirably equipped to lead the Centre and to continue and develop its unique attributes.  His ground breaking original work, particularly in transthyretin amyloidosis, places the NAC in a pivotal position in the rapidly advancing field of novel and effective drugs for this previously untreatable form of the disease.

Dr Marianna Fontana, who leads the dedicated magnetic resonance imaging facility of the NAC and is the world’s leading expert on cardiac magnetic resonance evaluation of amyloidosis, is the new Deputy Director of the Centre.

Sir Mark’s laboratory is known as the Wolfson Drug Discovery Unit in recognition of the £2 million award he received from the Wolfson Foundation in 2008.  The Unit’s core funding comes from the National Institute of Health via the UCL/UCLH Biomedical Research Centre, of which he is one of the Principal Investigators.  The current five year period of this funding, more than £1 million, ends on 31 March 2022 and Sir Mark intends to step down as Director of the Unit at that time, handing over to Professor Vittorio Bellotti.  Until then, and possibly afterwards, depending on progress in the next two years, Sir Mark will continue to run his drug development programmes.  These comprise the NIHR funded (£5.75 million) DESPIAD clinical trial of his drug, miridesap, for Alzheimer’s disease and two other major, fully funded programmes for new potential drugs invented by him and his team.

Professor Vittorio Bellotti was a post-doctoral research fellow in the Pepys laboratory in 1994 and has been a close collaborator ever since, becoming a world leader in amyloidosis research, especially its biochemical and biophysical aspects.  Sir Mark recruited Professor Bellotti to join the Wolfson Drug Discovery Unit in 2010, specifically as his chosen successor to lead the laboratory in due course.  Professor Bellotti rapidly established his own team in the Unit, working on his own projects, some in close collaboration with Sir Mark.  Professor Bellotti’s exciting and productive work has included a series of critically important new discoveries about the mechanisms of amyloid of formation that will underpin new therapeutic developments.

More about the NAC

More about Public and Patient Involvement at the NAC

Essential information

Publications authored by NAC doctors

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The Amyloidosis Awareness Programme of the NAC is generously supported by:
Akcea Therapeutics
Alnylam Pharmaceuticals
The Tufton Charitable Trust

General administration, web design and medical communications provided by:
Dr Miriam Vered, Vered Consulting