About Us

The Centre conducts world leading research in all aspects of the pentraxin family of plasma proteins, and in amyloidosis.  Studies range from molecular, genetic, biochemical, physiological and pathological investigations 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 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 introduced specific 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 covers treatments for amyloidosis and amyloid protein associated diseases.  All the drug discovery and development work is 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 therapeutic partnership between a small molecule drug and a fully humanised monoclonal antibody for treatment of systemic amyloidosis.  The intellectual property was licensed to GlaxoSmithKline in February 2009 and a highly synergistic collaborative clinical development programme led to the first phase I clinical trial in systemic amyloidosis that started on 1 June 2013 and has shown very excitingly encouraging results. The Clinical Proof of Mechanism milestone was achieved in July 2014 and Orphan Drug designation has been granted by the European Medicines Agency and the US Food and Drug Administration.  The programme has also been adopted by the EMA as an early candidate for adaptive licensing, an exploratory new approach intended to accelerate access to new treatments for unmet medical needs.  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 is on schedule 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.  The trial will be conducted in the Leonard Wolfson Experimental Neurology Centre by Professor Martin Rossor FMedSci and colleagues at the UCL Institute of Neurology and 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 in progress with a prospective end date in 2016.

Page last modified on 20 jul 15 13:38 by E A (Beth) Jones