Novel treatments for systemic amyloidosis

Systemic amyloidoses are potentially lethal diseases. Current treatment is directed against ongoing supply of precursor proteins. Treatment modalities include liver transplantation for ATTR amyloidosis, elimination of inflammation in AA amyloidosis and intensive chemotherapy in AL amyloidosis. These therapies often show substantial morbidity and mortality or insufficient effectiveness. Therefore the search for new more effective therapies without serious side-effects is still mandatory. Since cardiac involvement is vital in deciding on therapy, the introduction of N-terminal pro-brain natriuretic peptide (NT-pro-BNP) and troponin are of great importance. Determination of serum free light chains has become indispensable in the diagnosis and follow-up in AL amyloidosis. Manipulation of amyloid deposition and degradation might become possible in the future by the use of eprodisate in AA amyloidosis, 4’-iodo-4’-deoxydoxorubicin in AL amyloidosis, diflunisal in ATTR amyloidosis, and CPHPC in all types of amyloidosis. New potent precursor-lowering drugs, such as thalidomide, lenalidomide and bortezomib, need to find their place in the treatment protocols in AL amyloidosis.