Centre for Amyloidosis and Acute Phase Proteins
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Centre for Amyloidosis and Acute Phase Proteins
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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 amyloid protein associated diseases, and also for cardiovascular and inflammatory diseases. All the drug discovery and development work is now conducted within the Wolfson Drug Discovery Unit established in the Centre in 2011, with funding from the Wolfson Foundation, when Sir Mark retired as Head of Medicine at the Royal Free Campus. The Unit receives its core funding from the National Institute of Health Research (NIHR) via the UCL/UCLH Biomedical Research Centre (BRC).
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. The first phase 1 clinical trial in systemic amyloidosis started in late 2013 and is showing very excitingly encouraging results. The Clinical Proof of Mechanism milestone has been achieved, Orphan Drug status has been awarded by the European Medicines Agency and the programme has been adopted as a flagship project for adaptive licensing, intended to make the treatment available for patients earlier than would otherwise have been possible. A separate novel therapeutic approach for amyloidosis devised in the Unit is also being developed with GlaxoSmithKline and has lately achieved its Experimental Proof of Mechanism milestone.
Preparatory work for the DEpletion of Serum Amyloid P Component In Alzheimer's Disease (DESPIAD) clinical trial of our drug (CPHPC), which depletes serum amyloid P component from the brain, is funded by the NIHR via the BRC and the trial, to be run in the Leonard Wolfson Experimental Neurology Centre by Professor Martin Rossor FMedSci and colleagues at the UCL Institute of Neurology, will start in 2015. Initial clinical studies of CPHPC in cerebral amyloid angiopathy and in traumatic brain injury are also planned.
Our proof of concept clinical trial of CPHPC mediated serum amyloid P component depletion for the enhancement of DNA vaccination against HIV is funded by the Medical Research Council’s Developmental Clinical Studies Programme and is currently in progress.
The Centre’s drug development work targeting C‑reactive protein (CRP) for treatment of cardiovascular and inflammatory diseases, which was enabled by MRC funding since 1979, is now being supported by the British Heart Foundation.
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 02 sep 14 15:31 by E A (Beth) Jones