First of all, the diagnosis of amyloidosis has to be absolutely accurate. For a safe treatment an exact diagnosis regarding amyloid form is absolutely necessary. Presently, in all types, the goal is to eliminate the production of the protein which subsequently forms amyloid. This can be done but differs fundamentally between the types.
In AL-amyloidosis, the plasma cells, which produce the abnormal light chain, are suppressed by cytostatic drugs. Stem cell transplantation is used in many centers with good results. Although these treatments not necessarily reduce the amyloid deposits immediately, new research has shown to smaller protein aggregates which are toxic to cells, e.g. in the heart, rapidly vanish. AL-amyloidosis, which earlier was regarded as almost impossible to treat, is today associated with much better prognosis than earlier.
The basic method to treat AA amyloidosis is by suppression of the underlying inflammatory disease, such as rheumatoid arthritis. In this way, the liver-production of SAA, which forms the amyloid goes down and no new amyloid is formed. It is known since decades that already formed AA amyloid then can be absorbed and the function of the organs is improved.
The treatment of familial amyloidosis varies and depends on type. The most frequent form, in Europe particularly common in Portugal and Sweden, is associated with mutation in the transthyretin gene. This systemic amyloidosis is now often treated with liver transplantation with good results. There are, however, other mutations in the same gene which are less suitable for this treatment.
New optionsDuring the last few years, new drugs have been developed which are directed towards the amyloid itself and some of them are now in clinical trials. The first results are very promising and it is very probable that we within very few years will have new treatment options for most or all forms of systemic amyloidosis.