The aim of the therapy is firstly to stop the organ damage and then to allow for improvement in the function of organs such as the heart or kidney. Recovery can be a slow process. At present, there are no specific drugs that can help the body to remove the amyloid deposits. Such drugs are in clinical trials and show great promise. Hence, the main treatment for amyloidosis is very similar to that for myeloma and relies on myeloma drugs. Both diseases have the same origin and several data show that these drugs have even better results in amyloidosis patients than in myeloma patients.

The options are either some form of chemotherapy or consideration of a stem cell transplant using stem cells taken from patients themselves. For amyloidosis patients who are relatively young and fit, a stem cell transplant (SCT) is a suitable option. SCT can provide long remission and very good results. However, if there is substantial organ function damage, especially heart involvement, then SCT may have unacceptably high risks. In addition, even the alternative chemo- immunotherapy may have severe toxicity even more than in myeloma patients.

For other patients, the alternative treatment option is chemotherapy and immunotherapy. The most important drugs for these patients are proteasome inhibitors (usually a drug called bortezomib, and more recently, newer variants of this called carfilzomib and ixazomib) that have shown outstandingly high efficacy in amyloidosis treatment. These drugs have essentially changed the landscape of this disease. They are used in combination with other agents and provide deep durable responses.

In addition to proteasome inhibitors, the current physician’s choice of treatment consists of chemotherapy drugs such as melphalan, cyclophosphamide and immunomodulators (such as thalidomide, lenalidomide and pomalidomide). Recently, daratumumab, an anti-CD38 antibody very effective in myeloma, was shown to be extremely effective in amyloidosis patients, with both durable responses alone or combined with bortezomib based treatments, and subsequently good organ responses regaining function.