Improving treatment for systemic amyloidosis

A potential new approach to treat systemic amyloidosis, invented at UCL and being developed by GlaxoSmithKline (GSK), marks the start of a successful and innovative academic-industry collaboration. The first in human clinical trial of a novel investigational drug intervention for patients with systemic amyloidosis has established proof of mechanism. Results in the first 15 patients treated with a therapeutic partnership of a small chemical molecule and a large biological molecule (an antibody) are published in the New England Journal of Medicine . Further clinical testing is in progress and a phase II trial to explore efficacy and safety is planned. Amyloid is an abnormal protein material that accumulates in the tissues, damaging their structure and function and causing a serious and usually fatal disease called systemic amyloidosis. It is a rare disease but an important unmet medical need. Present treatments can stabilise some patients and substantially prolong life but about 20% of patients still die within 6 months of diagnosis.