Professor Sir Mark Pepys was elected to an Honorary Fellowship of Trinity College, Cambridge, in 2014.

First reports on the phase I clinical trial of the obligate therapeutic partnership of CPHPC and anti-SAP antibodies in patients with systemic amyloidosis

Our novel approach to direct targeting of systemic amyloid deposits for accelerated clearance from the tissues was invented in 2005.  Early successful preclinical experiments led to patent applications and awards to Pentraxin Therapeutics Ltd, the UCL spin out company founded by Sir Mark in 2001 to hold and commercialise his intellectual property (IP). The amyloid treatment IP was licensed to GlaxoSmithKline (GSK) in 2009 and they have developed the unique small molecule and antibody combination for clinical testing and have run the first in human clinical trial.  The whole process has involved very close collaboration between GSK, Pentraxin, the Wolfson Drug Discovery Unit and the National Amyloidosis Centre.  It is a paradigm for academic-pharmaceutical company collaboration in drug discovery and development.

The treatment has been well tolerated so far and, encouragingly, it has demonstrated notable efficacy in removing amyloid deposits from the tissues.  The initial results were reported at the annual meeting of the Association of Physicians of Great Britain and Ireland, held in Sheffield in March 2015. The first formal publication was posted online in the New England Journal of Medicine on 15 July 2015.  The article was the subject of a report in the Financial Times on 16 July 2015.

The clinical trial is ongoing and continuing to deliver impressive positive results that will be reported in the near future.  Meanwhile planning of a phase II study, focussed on efficacy and patient benefit, is well advanced and is scheduled to start in 2016.

Creation of a new dedicated cardiac magnetic resonance imaging facility in the National Amyloidosis Centre

Radiolabelled SAP scintigraphy, invented by Sir Mark in 1987, was developed for routine clinical use by him and Professor Philip Hawkins in Hawkins’s PhD project in 1987-1990.  It has long been a crucial component of the unique clinical research and practice of the National Amyloidosis Centre.  Although a form of SAP scan can image cardiac amyloid effectively, it requires a much more active isotope than the very safe short half life 123I used routinely. This is prohibited for safety reasons but cardiac magnetic resonance (CMR) methods, developed in the past 10 years, now provide very sensitive and specific information about amyloid in the heart and its effects on cardiac function.  The National Amyloidosis Centre has been funded to obtain CMR tests on our patients but these have been limited by shortage of NHS CMR facilities.  Professor Hawkins has lately obtained NHS funds for installation of a dedicated CMR facility within the Centre for Amyloidosis and Acute Phase Proteins and construction work started in June 2015.  Magnificent fundraising for the UCL Amyloidosis Research Fund, largely through the sponsored bike ride from Land’s End to John O’Groats by Thirusha Lane and David Hutt, together with many friends and supporters, contributed significantly towards the cost.  When the new scanner comes online it will be a tremendous help in patient assessment and critical important for clinical development of our own and other novel treatments for amyloidosis.

Page last modified on 20 jul 15 11:54 by E A (Beth) Jones