For AL (or primary) amyloidosis, the most commonly diagnosed form of the disease, extensive organ involvement is usual. Without treatment, the average survival rate is about 12-18 months, and only about 6 months for patients with severely impaired heart function Chemotherapy, either orally or intravenously, forms the cornerstone of treatment for AL amyloidosis. The goal is to
interrupt the growth of plasma cells producing the abnormal light-chain antibody proteins. For a number of years, therapies using melphalan (also known as Alkeran) or cyclophosphamide (Cytoxan) have been the treatment of choice.
Newer drugs that are used in treating multiple myeloma, such as bortezomib (Velcade), lenalidomide (Revlimid) or carfilzomib (Kyprolis), also have proved effective. These
therapies are often used in combination with dexamethasone, a steroid to help with the immune response. Undergoing chemotherapy may have side effects such as nausea, vomiting, hair loss, infection and extreme fatigue. If the side effects interfere with one’s quality of life, different regimens may be available. In carefully selected patients, chemotherapy is combined
with stem cell transplantation. Stem cells are found in the bone marrow, and they develop into several kinds of blood cells, including our plasma cells. Once the plasma cells are
destroyed using high doses of chemotherapy, the bone marrow is replenished with fresh stem cells from a patient’s own body (autologous transplant). With eradication of the faulty
plasma cells, amyloid production is slowed or stopped, and the bone marrow can become healthy again.
Chemotherapy followed by stem cell transplantation often achieves an excellent response, with significant improvement or stabilization of organ function. However, not all patients
can tolerate this aggressive regimen, particularly those with advanced heart problems. Given the complexity of the disease, it is recommended that treatment be performed in a medical center which has experience with amyloidosis (see the next section). Alternatively, patients can have an initial evaluation at such a center, with continued communication during treatment in his or her local community.
Another medicine being developed aims to target directly the light-chain amyloid deposits which have accumulated in the body. Currently in clinical trials, this treatment uses small molecules called monoclonal antibodies to find and attach specifically to the misfolded proteins of the amyloid fibrils. The monoclonal antibodies mimic the antibodies that our immune system naturally produces to protect us from disease. By targeting and destabilizing the amyloid deposits
in this way, one’s body can potentially identify and remove them more effectively. In the coming years, this promising drug may fulfill the final, missing piece of the treatment puzzle in helping to restore organ function and overall health.