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 patents owned by Pentraxin Therapeutics Ltd, the UCL spin out company
founded by Sir Mark Pepys in 2001 to hold and commercialise his intellectual property
The amyloid treatment IP was licensed to GlaxoSmithKline (GSK) in 2009 and they have developed the unique small molecule and antibody partnership for clinical testing with recent successful completion of 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 collaboration in drug discovery and development between academic science and a major pharmaceutical company.
The treatment has been well
tolerated so far and, encouragingly, it has demonstrated notable efficacy in
removing amyloid deposits from the tissues, leading to clinical benefit.
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 and
appeared in print in September 2015. The article was the subject of a report in
the Financial Times on 16 July 2015. A phase 2 trial, focussed
on cardiac amyloidosis, is planned to start in 2016.
Creation of a new dedicated cardiac magnetic resonance imaging facility in the National Amyloidosis Centre
scintigraphy, invented by Sir Mark Pepys in 1986, 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. SAP scanning is not
suitable for imaging cardiac amyloidosis, for technical reasons. Fortunately, 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
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 therefore effectively raised NHS funds for
installation of a dedicated CMR facility within the Centre for
Amyloidosis and Acute Phase Proteins and it opened for patients
in December 2015.
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.
The new scanner is, and will continue to be, tremendously helpful for diagnosis and patient management, and also of critical important for clinical development of our own and other novel treatments for amyloidosis.