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 in human, phase 1, clinical trial in systemic amyloidosis that started on 1 June 2013 and ended in December 2015. The results have been excitingly encouraging, with unprecedented clearance of visceral amyloid deposits, improvement in organ function and acceptable tolerability. The initial patients were published in the New England Journal of Medicine, 2015, and later studies in abstract form in Blood, 2015. 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.
The DEpletion of Serum Amyloid P Component In Alzheimer's Disease (DESPIAD) phase 2b clinical trial of our drug (CPHPC), which depletes serum amyloid P component from the brain 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 September 2017. An initial clinical study of CPHPC in cerebral amyloid angiopathy is also planned.