Centre for Amyloidosis and Acute Phase Proteins
The Centre conducts world leading research in all aspects of the pentraxin family of plasma proteins, and in amyloidosis. Studies range from structural biology, through molecular, genetic, biochemical, physiological and pathological studies, to clinical diagnostics, patient management and new drug discovery. There are extensive collaborative links with scientists, clinicians and industry in many of these areas. The goal is to elucidate fundamental normal and pathobiological mechanisms in order to improve diagnosis, management and outcome of disease. Apart from all aspects of amyloidosis, for which the UK NHS National Amyloidosis Centre is located in this Department, there are particular interests in cardiovascular and inflammatory diseases, as well as the major common diseases associated with local amyloid deposits: Alzheimer’s disease and type 2 diabetes mellitus.
The National Amyloidosis Centre has
provided a diagnostic, staging, monitoring and management advisory service for
the national caseload of patients with amyloidosis since it was commissioned by
the NHS National Commissioning Group in 1999. The amyloid practice is the
world's largest and most diverse, and has lately introduced new services for
patients with hereditary and age-related cardiac amyloidosis. The Centre also
provides genetic, diagnostic and treatment services for patients with hereditary
periodic fever syndromes, and is home to the NHS Specialised Services CAPS Treatment
Service for children and adults with the cryopyrin associated periodic
syndrome. More information about the NHS CAPS Treatment Service and about the condition is available on the patient information site.
New inventions for treatment of disease made in the Centre are owned by Pentraxin Therapeutics Ltd, a UCL spin-out company founded and directed by Professor Sir Mark Pepys and Mr Cengiz Tarhan, Managing Director of UCL Business PLC. The Pentraxin Therapeutics Ltd portfolio comprises treatments for amyloidosis and for cardiovascular and inflammatory diseases. The most advanced project is the development of a unique small molecule and humanised monoclonal antibody combination for treatment of systemic amyloidosis. The I.P. was licensed to GlaxoSmithKline in February 2009 and a fully collaborative clinical development programme is in progress aiming for first patient studies in 2012. A second project, developing potential drugs to target transthyretin amyloidosis, was licensed to GSK in December 2010 and is making encouraging progress. The Centre’s drug targeting C‑reactive protein (CRP) for treatment of cardiovascular and inflammatory diseases, is being developed for clinical testing with the support of the Medical Research Council’s Developmental Clinical Studies scheme. Work is also progressing towards clinical testing of our drug which depletes serum amyloid P component (SAP) for treatment of Alzheimer’s disease. All the drug discovery and development work is now conducted within the newly formed Wolfson Drug Discovery Unit within the Centre.
The Laboratory for Protein Crystallography, headed by Professor Steve Wood, employs the techniques of X-ray and neutron diffraction and associated computational methods to define the three dimensional structures of a variety of proteins, including the pentraxins and their complexes with other proteins and low molecular weight ligands. In all cases we seek fundamental understanding of protein function at an atomic level and where appropriate to use this knowledge in the design of novel ligands with potential as drugs. The Genetics Unit, headed by Dr Paul Simons, conducts sophisticated genetic manipulations which underpin analysis of physiological and pathobiological processes in neuroscience, inflammation and amyloidosis as well as the effects of our novel therapeutic approaches.
Page last modified on 04 nov 13 13:28 by E A ( Beth ) Jones