How Is Amyloidosis Treated?

Amyloidosis is a rare disease in which an abnormal protein called amyloid builds up in the organs. The heart, kidneys, liver, spleen, nervous system, or digestive tract are all organs that may be impacted. Amyloidosis may affect only one organ or several. It may eventually lead to organ failure.^1,2

There are several types of amyloidosis. Some are passed down through families (genetic mutation), and others are caused by an underlying health condition. Sometimes the cause is unknown. The type of amyloidosis you have determines which treatment may be right for you.¹

Types of amyloidosis

Some of the most common types of amyloidosis are:^1-3

• AL or light-chain amyloidosis, the most common type that affects many organs
• AA amyloidosis, which mostly affects the kidneys and is typically caused by other conditions such as rheumatoid arthritis or inflammatory bowel disease
• ATTR or transthyretin amyloidosis, which includes the most common inherited form (hereditary or familial ATTR) and age-related ATTR (wild-type ATTR)

A closer look: ATTR-PN

A specific form of ATTR amyloidosis is called ATTR-PN (transthyretin amyloid polyneuropathy). This happens when the amyloid protein builds up in the peripheral nerves. These are the nerves that carry signals between your brain and the rest of your body, like your hands and feet.⁴

When these nerves are damaged, it can cause:⁴

• Numbness or tingling
• Pain or burning sensations
• Weakness in the legs or arms
• Balance problems

Other types of amyloidosis include dialysis-related, APOA1, gelsolin, fibrinogen, and lysozyme amyloidosis.

Treatment goals

Amyloidosis cannot be cured, so treatment goals focus on 3 things:^1,4

• To try and slow how much and how fast the disease advances
• To reduce symptoms
• To prolong life

Types of amyloidosis treatments

Inherited forms of amyloidosis and those caused by genetic mutations are treated differently than forms caused by underlying conditions. If your amyloidosis is caused by an underlying condition, your doctor will probably try to treat that condition as a way to control the amyloidosis.

Amyloidosis is a complex disease and treatment options often depend on a person's overall health as well as their type of amyloidosis. Being treated at a center specializing in the condition, or at least having your treatment plan created by a specialist, is recommended. Some people have a treatment plan built by a specialist and managed by their local doctor. Treatment options may include:^1,2

• Chemotherapy
• Bone marrow or stem cell transplant
• Drugs
• Clinical studies

Chemotherapy

Some of the same drugs that treat cancer may also be used for complications of AL amyloidosis. Two older drugs used include melphalan (Evomela®) and dexamethasone. Sometimes high-dose melphalan is given along with stem cell treatment. Some newer drugs include bortezomib (Velcade®), lenalidomide (Revlimid®), and daratumumab (Darzalex). In some cases, daratumumab may be used in combination with bortezomib, cyclophosphamide (Cytoxan®) and dexamethasone.²

Bone marrow transplant

Bone marrow transplant is also called stem cell transplant. This is another treatment option for AL amyloidosis, and is generally done in combination with chemotherapy. During a bone marrow transplant, healthy stem cells are removed from your blood or bone marrow.¹

You then get chemotherapy to kill the abnormal cells in your bone marrow (where blood cells are made). Then, your doctor will put the healthy stem cells back into your body. These healthy cells replace the unhealthy ones.¹

Heart medicines

Many times, amyloidosis attacks the heart. If your heart is affected, your doctor may prescribe blood thinners to reduce the risk of clots. You may also need drugs to control your heart rate or increase urination. These 2 types of drugs help ease stress on the heart. A well-balanced, low-salt diet may be necessary too.^2,5

ATTR and ATTR-PN treatments

There are special drugs used to treat ATTR. These work in 2 main ways:^2,4-6

• Gene Silencers – These "turn off" the gene that makes the problem protein. Drugs like patisiran (Onpattro®), vutrisiran (Amvuttra), and eplontersen (Wainua™) are used specifically for ATTR-PN to help protect the nerves.
• Stabilizers – These drugs, like tafamidis (Vyndamax®) and acoramidis (Attruby) act like "glue" to keep the protein from breaking apart and forming amyloid. This is often used when the heart is affected.

In some cases of hereditary ATTR, liver transplant may be an option when the disease is not too far advanced.^2,5,6

Other treatments

AA amyloidosis caused by an underlying condition may be treated in a number of ways. Kidney transplant may help some people with kidney disease due to AA amyloidosis. A medical device called Lixelle® is being studied as a treatment for a type of dialysis-related amyloidosis.

There are a few clinical studies looking at more drugs that may help people with different forms of amyloidosis.