Systemic amyloidosis.

Abstract

Amyloidosis describes a heterogeneous group of diseases in which normally soluble plasma proteins are deposited in the extracellular space in an abnormal insoluble fibrillar form. These diseases can affect virtually any organ system and often present a major diagnostic and management challenge. Current therapies centre on reducing the supply of the respective amyloid fibril precursor protein, combined with supportive treatment. Better understanding of the mechanisms underlying amyloid formation has led to the development of novel treatment strategies aimed at inhibiting fibrillogenesis or destabilizing existing amyloid deposits. It is hoped that some of these developments might contribute to effective treatment not only of systemic amyloidosis, which is relatively rare, but also of the much more common type II diabetes and Alzheimer's disease, in which local amyloid formation is thought to play a role.