Amyloidosis: Treatments

Our comprehensive, multi-disciplinary approach to amyloidosis treatment involves hematologists, nephrologist, neurologists, cardiologists, pathologists, clinical and basic research experts. 

The many treatments for amyloidosis include:

Chemotherapy

Primary amyloidosis (AL) is usually treated with a chemotherapy-based regimen (similar to that used for multiple myeloma) to destroy the abnormal plasma cells that give rise to the amyloid proteins or plaque. Chemotherapy drugs used to treat amyloidosis include melphalan (Alkeran) and cyclophosphamide (Cytoxan, Neosar) combined with dexamethasone (multiple brand names) and prednisone (multiple brand names)

Monoclonal Antibodies

All established therapies in amyloidosis focus on the destruction of plasma cells and subsequently the stop of the production of light chains forming the amyloid. Hence new accumulation of amyloid will be prohibited but unfortunately existing amyloid will not be affected by the treatment resulting in continuous impairment of the organ function. Monoclonal antibodies targeting directly the amyloid fibrills and subsequently destroying the existing amyloid is a very new and exiting approach. Therefore our center focuses on the use of such monoclonal antibodies. CUMC is the only center worldwide that offers treatment with the monoclonal antibody 11-1F4 that was developed by Dr Alan Solomon at the University of Tennessee.

Proteasome Inhibitors

A new class of drugs called proteasome inhibitors may also be used in different combinations with chemotherapy drugs. These drugs affect the cell structure that regulates proteins involved in cell replication and survival. They essentially block the function of proteins that allow the myeloma cells to grow.

Stem Cell Transplantation (Blood)

Around 20% of patients with amyloidosis and an appropriate organ function are eligible for stem cell transplantation. If a patient with amyloidosis is in good condition (with fewer than three organs affected, with adequate cardiac function, and laboratory results), a bone marrow transplant specialist can perform the transplant. If the patient has more advanced disease, our team will pretreat the patient with a chemotherapy regimen to decrease the production of the amyloid plaque, and then perform the stem cell transplant once the patient is in a better performance status. Bone marrow transplant specialists at the HICCC have found this procedure to be very promising. More information on Stem Cell Transplantation can be found here.

Surgery: Heart Amyloidosis Treatment

Accumulation of amyloid protein in the heart reduces the ability of the heart to fill and can also result in damage to the heart’s electrical system. If left untreated, cardiac amyloidosis can lead to heart failure. Therefore, doctors at CUMC place a high priority on intervening early before this organ is affected.

If our cardiologists suspect amyloidosis after an electrocardiogram and echocardiogram, a definitive diagnosis can be made with a biopsy of the heart. The procedure is safe if performed at an experienced institution. Our physicians have significant expertise in endomyocardial biopsy. They routinely perform more than 20 heart biopsies each week at one of the largest heart failure and transplant program in the country.

Recently, our doctors have been successfully employing cardiac magnetic resonance imaging (MRI)—a less invasive method—to accurately diagnose cardiac amyloidosis.

Unfortunately amyloidosis can progress to the point where a heart transplant or a mechanical assist device is required. Founded over a quarter of a century ago, the heart transplant program at NewYork-Presbyterian Hospital/Columbia University Medical Center has long been the top cardiac transplant program in the US and a leader in the development and implantation of mechanical assist devices, also called VADs (Ventricular Assist Devices). These pumps can be used to support heart function and blood flow in people who have weakened hearts.

More information on Heart Transplantation can be found here.

More information in Mechanical Assist Devices can be found here.

Surgery: Kidney Transplant, Liver Transplant

Kidney Transplant Kidney amyloidosis inhibits the kidneys from properly removing waste from the body, and if left untreated can lead to kidney failure. Therefore, doctors at CUMC place a high priority on intervening early before the kidney is seriously affected. Our kidney transplantation program are the oldest in the region and the largest in the nation. More information on kidney transplantation for amyloidosis can be found here.

Liver Transplant The amyloid deposits in most cases of familial (hereditary) TTR amyloidosis are composed of the abnormally structured protein transthyretin (TTR), which is made in the liver. In those patients a liver transplant will cure the patients and will stop the production of an abnormal transthyretin protein.

The Center for Liver Disease and Transplantation at New York-Presbyterian Hospital is a multidisciplinary program that provides medical treatment, surgery, transplantation, and patient support.

More information on liver transplantation can be found here.