Macrophage function in systemic amyloidosis; v1.0; 28 April 2016
Research type
Research Study
Full title
Role of monocytes and macrophages in patients with systemic amyloidosis
IRAS ID
199318
Contact name
Glenys Tennent
Contact email
Sponsor organisation
Royal Free London NHS Foundation Trust
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Amyloidosis is a fatal disease caused by abnormal protein deposits (amyloid) that accumulate in tissues and progressively damage organ function. The most prevalent forms of systemic amyloidosis are responsible for about one per thousand of all deaths in developed countries. There is no effective treatment to speed the body’s natural capacity to remove amyloid, and no way of identifying patients who are at high risk of developing amyloidosis. Our Centre has discovered much of what is known about amyloidosis and pioneered a diagnostic test, now incorporated into clinical practice, which showed that some individuals can slowly get rid of their amyloid. However, the mechanisms by which they do this are not fully understood. Nor is it known why amyloid goes away in some individuals but not in others. Recently, we developed a novel antibody therapy that completely and safely removes amyloid deposits in mice. Preliminary studies demonstrate similar effects in humans, but with wide variations in apparent efficacy. The cells responsible, and essential, for this clearance process are macrophages, cells that patrol throughout the body in order to remove foreign particles and tissue debris.
We now aim to characterise macrophages in amyloidosis patients, comparing those who do and do not get rid of their amyloid efficiently in response to treatment, and individuals at risk of amyloidosis who do and do not develop amyloid deposits. Our unique and extensive patient populations, expertise in amyloid and amyloid-macrophage interactions, and knowledge of all aspects of the disease, create this compelling opportunity to explore macrophage function in systemic amyloidosis. The results may, in due course, shed new light on clinically valuable determinants of individual susceptibility to amyloidosis, and enable the most effective and rational use of existing treatments and the very promising new antibody therapy which is about to undergo a Phase II clinical trial.
REC name
West Midlands - Solihull Research Ethics Committee
REC reference
16/WM/0227
Date of REC Opinion
19 May 2016
REC opinion
Further Information Favourable Opinion