Drug Therapies for Amyloidosis

Different therapy options may be used for patients with amyloidosis. Different combinations of drugs may be used to target the amyloidosis, as well as treat underlying symptoms caused by the disease. It’s not uncommon for patients to receive more than one drug in order to best manage their symptoms. The therapies chosen depends on each individual after conversations with their doctor and current health status. Learn more about the types of drugs that may be used below. 

Immunomodulatory drugs

Immunomodulating agents target the pathways that fuel amyloidosis. It’s not entirely clear how these agents affect the immune system, but they work to fight the cancerous cells in the body. Examples of immunomodulating agents are:

  • Thalidomide is a drug previously used in the 50s as a sleep aid and to treat motion sickness before being re-introduced to treat plasma cell disorders. Side effects include drowsiness, rash, constipation, and thought slowing.
  • Lenalidomide (revlimid) is an analog of thalidomide. It has the potential side effects of venous thrombosis, a drop in blood counts, and rash.
  • Pomalidomide (Pomalyst) is a derivative of thalidomide. The most common side effects include tiredness, weakness, constipation, shortness of breath, diarrhea, fever, back pain, and nausea.

Monoclonal antibodies

A monoclonal antibody attaches to a specific protein in the abnormal cells without affecting cells that don’t have that protein. Other monoclonal antibodies that directly targets the amyloid itself are currently in clinical trials as future options for patients. Currently approved options include:

  • Daratumumab (Darzalex) is given through an IV and attaches to the CD38 protein. It is thought to kill the cells directly and help the immune system fight them off. Symptoms for either form of the drug can include coughing, wheezing, trouble breathing, tightness in the throat, a runny or stuffy nose, feeling dizzy or lightheaded, headache, rash, and nausea.
  • Elotuzumab (Empliciti) attaches to the SLAMF7 protein, another protein found on myeloma cells, and attacks those cancer cells. It is used mainly in patients who have already had other treatments for their myeloma and is given through an IV. Symptoms include fever, chills, dizziness, rash, wheezing, tightness in the throat, or a runny or stuffy nose.

Proteasome inhibitors

Proteasome inhibition is a type of targeted therapy that targets specific enzymes called proteasomes that digest proteins in the cells. These drugs include:

  • Bortezomib (Velcade) was the first of these types of drugs to be approved. This medication needs to be administered through an IV and is especially helpful for patients that have kidney problems. Potential side effects include nausea, vomiting, tiredness, diarrhea, constipation, fever, decreased appetite, and lowered blood counts. More seriously, bortezomib can also cause nerve damage, known as peripheral neuropathy.
  • Carfilzomib (Kyprolis) can be used to treat patients who have already been treated with other drugs that didn’t work. It’s given through an IV and common side effects include tiredness, nausea, vomiting, diarrhea, shortness of breath, fever, and low blood counts.
  • Ixazomib (Ninlaro) is an oral proteasome inhibitor typically given after other drugs have been tried. Common side effects of this drug include nausea and vomiting, diarrhea, constipation, swelling in the hands or feet, back pain, and a lowered blood platelet count.

Investigational Agents

Research is ongoing to find new drugs or combinations of drugs that may be beneficial to patients with amyloidosis. The Weill Cornell Medicine Myeloma Center participates in these clinical trials to potential new therapies for our patients that have not yet been approved by the U.S. Food and Drug Administration (FDA). Patients may have the opportunity to participate in these studies after speaking to our physicians to discuss their treatment options, side effects, and overall health.

Chemotherapy may also be given to patients with amyloidosis in combination with other targeted therapies in order to destroy the abnormal cells in the blood. The most common types of chemotherapy given to amyloidosis patients include cyclophosphamide (Cytoxan, Neosar) and melphalan (Alkeran) combined with the steroid’s dexamethasone and prednisone.

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