Our comprehensive multidisciplinary approach to amyloidosis treatment involves hematologists, nephrologists, neurologists, cardiologists, pathologists, and research experts.
The many treatments for amyloidosis include:
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 is “systemic” medicine—it interferes with all fast-dividing cells in your body. This is why it can cause side effects like hair loss.
Chemotherapy is often given through an IV in our infusion center, but it can sometimes be given with pills.
Targeted therapies work by targeting specific genes, proteins, or other aspects that contribute to the development of amyloidosis. These treatments limit damage to healthy cells while working to stop the growth and spread of abnormal cells.
Plasma cell directed antibody
One of the newest and most exciting new developments in therapy of AL amyloidosis is the development of CD38 antibody (Daratumumab), which works by binding to the malignant plasma cells and exposing them to the immune system of the patient. The drug is currently approved both for newly diagnosed as well as relapsed multiple myeloma. In addition, the drug has shown exceptional activity in AL amyloidosis. We currently use this drug routinely for therapy of our patients with systemic AL amyloidosis and have seen excellent outcomes.
Anti-amyloid fibril antibody (CAEL-101)
Currently most therapies in amyloidosis work by destroying the plasma cell and stopping the accumulation of the amyloid but unfortunately not reversing the buildup of an existing amyloid. Monoclonal antibodies that directly target the amyloid deposits such as this treatment are new and exciting treatments that have the potential to reverse the existing buildup of an amyloid in patients with amyloidosis. Our center focuses on these monoclonal antibodies and was the only center worldwide that initially offered therapy with the monoclonal antibody CAEL-101. The drug is currently being planned to be tested in a randomized phase 3 study.
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 (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 Columbia Cancer have found this procedure to be very promising.
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. Our doctors 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 such as ours. 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 programs 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 more than a quarter of a century ago, the heart transplant program at NewYork-Presbyterian Hospital/Columbia University Irving 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.
Kidney amyloidosis inhibits the kidneys from properly removing waste from the body and if left untreated can lead to kidney failure. Therefore, doctors at CUIMC place a high priority on intervening early before the kidney is seriously affected. Our kidney transplantation program is the oldest in the region and the largest in the nation.
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 stop the production of an abnormal transthyretin protein.
The Center for Liver Disease and Transplantation at NewYork-Presbyterian Hospital is a multidisciplinary program that provides medical treatment, surgery, transplantation, and patient support.