Amyloidosis

The following was written by Professor Pepys at the University College London.  The money I raise from walking across Spain will be going to the UCL Amyloidosis Research Fund, to assist with their search for a cure.

ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL 

UCL DIVISION OF MEDICINE (HAMPSTEAD CAMPUS) 

CENTRE FOR AMYLOIDOSIS AND ACUTE PHASE PROTEINS 

 

17 September 2007 

 

Research on amyloidosis at the UK NHS National Amyloidosis Centre in the Centre for Amyloidosis and Acute Phase Proteins of University College London 

Amyloidosis is a fatal disease caused by deposition of abnormal insoluble protein fibres in the tissues and organs of the body, damaging their structure and function.  Almost any part of the body can be affected so the clinical signs and symptoms vary greatly, making the diagnosis difficult for general physicians and even specialists who usually have not seen many cases.  Amyloidosis causes the death of about 1 per 1000 of all people dying in developed countries, so it is a rare but not vanishingly rare condition.  Without treatment most patients die within a year or so of diagnosis.  There is no specific and generally effective treatment although a range of different  approaches can significantly prolong survival. 

The National Amyloidosis Centre in the Centre for Amyloidosis and Acute Phase Proteins of University College Hospital, located at the Royal Free Hospital, London NW3 2PF, was founded by Professor Mark Pepys FRS FMedSci in 1999 with Professor Philip Hawkins FMedSci as the Clinical Director, and is funded by the Department of Health to provide diagnostic and management advisory services for the whole country.  The Centre builds on the research and clinical experience of Professor Pepys and his team, supported by the Medical Research Council since 1979, and now has expertise in all aspects of amyloidosis which is unparalleled elsewhere in the world.  The Centre currently sees over 2000 patients per year and the average survival of these patients now extends to 8 years or more. 

Research and development of new treatments for amyloidosis in the Centre have made enormous contributions to understanding of the disease and to improved outcomes for patients.  These achievements have been recognised by the award of large research grants by the Medical Research Council, the Wolfson Foundation and the Wellcome Trust as well as by the generous funding of patient care by the NHS.  However there is always a need for flexible research funds which can be used whenever required for developments and to support new ideas.  Award of formal research grants takes many months and is always rather strictly tied to particular uses.  Availability of resources in the UCL Amyloidosis Research Fund which can be deployed immediately for whatever is needed, equipment, reagents, salaries or infrastructure, has been of enormous benefit to our efforts. 

There are currently several very promising lines of work in the Centre on treatment for amyloidosis: a large multicentre national clinical trial of different types of chemotherapy, design and development of new drugs  to treat and prevent a particular form of hereditary amyloidosis, and most excitingly a combination of a new  drug developed in the Centre with a novel antibody invented there.  This combination therapy cures amyloidosis in an experimental model which closely resemble the human disease and the highest research priority is to take this approach into patients as soon as possible. 

The resources provided by the UCL Amyloidosis Research Fund through the generosity of donors will undoubtedly make major contributions to alleviating the suffering and saving the lives of many thousands of individuals afflicted by a terrible and hitherto incurable disease. 

Professor M B Pepys FRS FMedSci 

Head, Department of Medicine (Hampstead Campus) 

Director, Centre for Amyloidosis & Acute Phase Proteins 

Centre for Amyloidosis and Acute Phase Proteins 

UCL Department of Medicine (Hampstead Campus) 

Rowland Hill Street  London  NW3 2PF  UK 

Tel: +44 (0)20 7433 2801  Fax: +44 (0)20 7433 2803 

m.pepys@medsch.ucl.ac.uk 

www.ucl.ac.uk/medicine/amyloidosis