Treatment for amyloidosis a step closer

UCL scientists funded by the Medical Research Council (MRC) have devised a new approach to treatment of amyloidosis, according to new research . Systemic amyloidosis is a serious and usually fatal disease that can affect virtually any organ in the body. It is the cause of death in one per thousand of the population in developed countries. Amyloid is composed of abnormal protein fibres that are deposited in the body's tissues, damaging their structure and function. Diagnosis is often difficult and delayed so that, by the time the disease is recognised, most patients already have irreversible organ damage. The SAP protein from the blood accumulates in amyloid deposits and contributes to their formation and persistence.In work supported by the MRC spanning 30 years, Professor Mark Pepys FRS and his team from UCL discovered the role of SAP in amyloidosis and have been developing new treatments aimed at it. Initially they partnered with Roche to develop a small molecule drug, called CPHPC, which removes SAP from the blood but only partly clears it from amyloid deposits.