Amyloidosis: is a cure possible?

The amyloidoses are protein misfolding diseases in which different soluble proteins aggregate as extracellular insoluble fibrils. This process causes organ dysfunction and death, unless it is arrested by therapy. So far, 25 types of amyloidosis have been classified according to the protein forming the amyloid deposits. In AL amyloidosis, a usually small-sized bone marrow plasma cell clone produces toxic light chains that cause multiple organ damage and fibrillar deposits [1]. Only a minority of monoclonal light chains form amyloid fibrils, probably due to specific mutations that destabilize the protein favoring its misfolding and aggregation. In the last few years, a better understanding of the pathogenesis of the disease, new markers of prognosis and response and innovative therapies have improved the care of patients with AL amyloidosis, with a profound impact on the natural history of the disease.