Amyloidosis is a rare disease that develops when certain abnormal proteins accumulate in the body. Doctors treat amyloidosis with a combination of chemotherapy, targeted therapy, and organ transplantation, or a combination of these. The best treatment for your amyloidosis depends on:

• The type of amyloidosis you have
• The severity of your symptoms
• The organs affected

If amyloidosis is diagnosed early, treatments may be more likely to work. Treatment goals may include preventing or slowing the buildup of amyloid proteins. This will help you manage your symptoms and treat organ damage.

It’s important to work with your care team to improve your quality of life with amyloidosis. Doctors who treat amyloidosis include hematologists (blood specialists) and cardiologists (heart specialists).

Read on to learn more about eight types of treatments for amyloidosis.

1. Chemotherapy

Chemotherapy medications can be used to treat amyloid light-chain (AL) amyloidosis. Although this disease is not itself a cancer, it may be associated with a type of blood cancer known as multiple myeloma. For this reason, some medications for blood cell cancers work well.

AL amyloidosis is also called primary amyloidosis or immunoglobulin light-chain amyloidosis. It develops from plasma cells that normally make antibodies to fight infections. People with AL make abnormal light-chain proteins found in antibodies. These proteins form amyloid fibers that build up throughout the body.

Researchers have found that multiple myeloma drugs work well for treating AL amyloidosis. Multiple myeloma is a type of blood cancer affecting the plasma cells. Medications for this cancer can also attack abnormal plasma cells that cause AL amyloidosis.

One of the longest-used chemotherapy drugs for AL amyloidosis is melphalan (Evomela). This medication is often given along with the steroid drug dexamethasone. Alternatively, some people get high-dose melphalan along with stem cell transplantation. Other chemotherapy drugs used to treat AL amyloidosis include cyclophosphamide and bendamustine (Treanda).

Side effects of amyloidosis chemotherapy include:

• Tiredness
• Constipation or diarrhea
• Nausea and vomiting
• Mouth sores
• Loss of appetite

Some medications, like melphalan, raise the risk of developing leukemia. Talk to your doctor about any concerns you have about your treatment plan.

2. Targeted Therapy

Targeted therapies are medications that specifically block the growth of certain abnormal cells. These drugs are more likely to leave healthy cells alone and cause fewer side effects. There are several categories of drugs that can help treat AL amyloidosis. Some of these medications aren’t officially approved by the U.S. Food and Drug Administration (FDA) to treat amyloidosis. However, clinical trials have shown they can be helpful for people with this disease.

Proteasome Inhibitors

Proteasome inhibitors stop abnormal cells from growing uncontrollably. Examples include bortezomib (Velcade), ixazomib (Ninlaro), and carfilzomib (Kyprolis).

Monoclonal Antibodies

Monoclonal antibodies find abnormal plasma cells and tell the immune system to destroy these cells. Examples of monoclonal antibodies used to treat amyloidosis include daratumumab (Darzalex) and elotuzumab (Empliciti).

Immunomodulatory Drugs

Immunomodulatory drugs (IMiDs) encourage the immune system to fight abnormal cells. IMiDs used to treat amyloidosis include thalidomide (Thalomid), lenalidomide (Revlimid), and pomalidomide (Pomalyst).

Combinations With Targeted Medications

You may receive a combination of targeted therapies and other medications. Most people with a new AL amyloidosis diagnosis are treated with an FDA-approved combination of:

• Bortezomib
• Cyclophosphamide
• Daratumumab
• Dexamethasone

Side Effects of Targeted Drugs

Side effects of targeted therapies for amyloidosis include:

• Low blood cell counts
• Nerve damage
• Severe constipation
• Nausea and vomiting

Most of these medications are used off-label. This means they’re approved to treat other diseases like multiple myeloma but not for amyloidosis. This is partially because amyloidosis is a rare disease, so there haven’t been many clinical trials. It can be difficult to enroll enough participants in clinical trials to study new therapies. However, some new studies are underway.

TTR Gene Silencers

In recent years, researchers have also developed medications that target amyloidosis-related genes. Some work by blocking mutant transthyretin (TTR) genes. These genes create amyloid proteins in hereditary amyloidosis (hATTR amyloidosis). Examples include:

• Eplontersen (Wainua)
• Patisiran (Onpattro)
• Vutrisiran (Amvuttra)

These medications are all FDA-approved for treating hATTR amyloidosis and peripheral neuropathy (nerve damage). Common side effects include:

• Sore throat
• Joint pain
• Muscle pain and spasms
• Headache

Other medications help prevent the TTR protein from forming amyloid deposits. Tafamidis meglumine (Vyndaqel) and tafamidis (Vyndamax) received FDA approval in 2019 to treat people with cardiomyopathy (heart disease). It’s possible to experience an allergic reaction while taking tafamidis. Get medical attention right away if you begin experiencing any of the following:

• Trouble swallowing
• Hives or itchy skin rashes
• Tightness in your chest
• Fast heartbeat

3. Medications To Treat Organ Damage

Different types of amyloidosis may require treatments, depending on which body parts are affected. These treatments don’t remove amyloid deposits or prevent the body from forming amyloid proteins. This means they don’t treat amyloidosis itself. Instead, some medications treat amyloidosis symptoms and prevent and manage complications.

Kidney damage often develops in people with AA amyloidosis. To manage this condition, you may need medication to lower blood pressure, blood sugar, and cholesterol levels. You may also use drugs called diuretics that remove extra fluid from the body. Other medications help keep your bones strong.

Wild-type ATTR amyloidosis and hATTR amyloidosis can lead to heart failure. If you have heart symptoms, drugs like blood thinners can help support heart health. However, medications used to treat other types of heart failure can be harmful if you have amyloidosis. Examples include:

• ACE inhibitors
• Beta-blockers
• Calcium channel inhibitors

If you have heart symptoms, it’s important to see a specialist who has experience treating people with amyloidosis.

4. Stem Cell Transplantation

Medications may be effective at slowing down amyloidosis and managing symptoms. However, you may need surgery or a medical procedure if amyloid deposits damage any affected organs.

Your health care team may recommend an autologous stem cell transplant (bone marrow transplant). This procedure is usually combined with the chemotherapy drug melphalan to treat AL amyloidosis. During this procedure, a provider removes your blood stem cells from your body. They then give you a high dose of melphalan over one day. Around three days later, the provider reintroduces your stem cells to your body. This procedure may help reduce amyloidosis symptoms for a while, but it’s not a cure. Patients with AL amyloidosis may be eligible for a stem cell transplant if they are below 70 years of age and have adequate heart and lung function.

A stem cell transplant comes with some risks and side effects, and it takes some time to recover. You may have to stay in the hospital for a few weeks after this procedure.

5. Dialysis

Some people with amyloidosis develop amyloid deposits in the kidneys, causing kidney disease. When your kidneys don’t work properly, you may need dialysis to remove waste from your body. Dialysis is a procedure in which an external machine filters wastes and toxins out of your blood. A kidney transplant may be a good option for some people with AA amyloidosis who have significant kidney damage. You’ll likely need to control any underlying inflammation before a transplant.

People with dialysis-related amyloidosis typically have high levels of abnormal beta-2 microglobulin protein. Traditional dialysis procedures can’t remove this protein very effectively. However, a newer device called the Lixelle Beta 2-microglobulin apheresis column can clear this protein from the blood. This device is FDA-approved for dialysis-related amyloidosis.

6. Heart Transplant

Amyloidosis can also affect the heart — known as cardiac amyloidosis. You may need a heart transplant for severe damage. A combination of a heart transplant and a liver transplant is sometimes used to treat hATTR amyloidosis.

7. Liver Transplant

Some amyloidosis treatments address the disease’s symptoms. However, liver transplantation actually targets amyloidosis itself. When the liver makes abnormal TTR proteins, hATTR amyloidosis develops. A liver transplant to remove the dysfunctional liver may help treat the disease. It can slow down or even completely stop new amyloid formation.

8. Clinical Trials

Clinical trials are underway to find additional treatments for amyloidosis. These studies help researchers learn more about whether existing drugs can treat amyloidosis. Clinical trials also allow them to better understand the effects of new drugs.

For example, researchers are studying new antibodies targeting amyloid proteins. Other studies are also investigating CAR T-cell therapy. This treatment engineers immune cells (T cells) to treat diseases.

If you have amyloidosis, you may be able to participate in a clinical trial. Talk to your health care provider to learn more.

Talk With Others Who Understand

MyAmyloidosisTeam is the social network for people with amyloidosis and their loved ones. On MyAmyloidosisTeam, members come together to ask questions, give advice, and share their stories with others who understand life with amyloidosis.

Have you received treatment for amyloidosis? What was your experience like? Share your experiences in the comments below, or start a conversation by posting on your Activities page.