Amyloidosis, a rare disease that develops when certain abnormal proteins accumulate in the body, can be treated with chemotherapy, targeted therapy, and sometimes organ transplantation. Which treatments your doctor recommends will depend on the type of amyloidosis you have, how severe your symptoms are, and which organs are 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, managing symptoms, and treating organ damage.
Several medications can be used to treat the different types of amyloidosis. You may receive one or a combination of these drugs. Most of these medications are not officially approved by the U.S. Food and Drug Administration (FDA) to treat amyloidosis, but clinical trials have shown they can be helpful for people with this disease.
Chemotherapy medications can be used to treat amyloid light-chain (AL) amyloidosis. Although this disease is not itself a cancer, medications used to treat blood cell cancers can sometimes be effective. AL amyloidosis, also called primary amyloidosis or immunoglobulin light-chain amyloidosis, develops from plasma cells. When these cells make abnormal light-chain proteins — which are normally used to produce antibodies for fighting infection — the proteins form amyloid fibrils that build up throughout the body. Researchers have found that drugs designed to treat multiple myeloma, a cancer of the plasma cells, can also attack abnormal plasma cells that cause AL amyloidosis.
One of the longest-used chemotherapy drugs for AL amyloidosis is Evomela (melphalan). This medication is often given along with Dextenza (dexamethasone), a steroid. Alternatively, some people get high-dose melphalan along with a stem cell transplantation. Other chemotherapy drugs used to treat AL amyloidosis include Cytoxan (cyclophosphamide) and Treanda (bendamustine).
Chemotherapy drugs may come with side effects, including tiredness, nausea, and vomiting. Some medications, such as melphalan, have been linked to an increased risk of developing leukemia. Talk to your doctor about any concerns you may have regarding your treatment plan.
Targeted therapies are medications that can seek out and block the growth of specific abnormal proteins or cells. These drugs are more likely to leave healthy cells alone, and therefore may cause fewer side effects. There are several categories of drugs that can help treat AL amyloidosis:
You may receive a combination of targeted therapies and other medications. Most people with AL amyloidosis are treated with Cytoxan (cyclophosphamide), Velcade, Dextenza (dexamethasone), and Darzalex Faspro (daratumumab plus hyaluronidase).
Most of these medications are used off-label — that is, they are approved to treat other diseases like multiple myeloma, but are not approved for amyloidosis. Part of the reason for this is that amyloidosis is a rare disease, and it can be difficult to enroll enough participants in clinical trials to study new therapies. However, some studies are underway. In January, Darzalex Faspro was approved by the FDA to treat AL amyloidosis.
In recent years, researchers have also developed medications specifically designed to target amyloidosis-related genes or proteins. Kiacta (eprodisate) can help prevent amyloid deposits from forming in people with AA amyloidosis. However, this drug has not yet been approved by the FDA. Tegsedi (inotersen) and Onpattro (patisiran) block mutant transthyretin (TTR) genes, which are responsible for creating amyloid proteins in hereditary forms of amyloidosis. These treatments are FDA-approved to treat people diagnosed with hereditary ATTR amyloidosis who have peripheral neuropathy (nerve damage). Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) help prevent the TTR protein from forming amyloid deposits. They are approved to treat people who have cardiomyopathy (heart disease).
AA amyloidosis develops when long-term inflammation occurs in the body. This can happen when people have diseases like rheumatoid arthritis, ongoing infections, and inflammatory bowel disease. Treating these inflammatory diseases is the most important part of treating AA amyloidosis. For example, if you have an infection such as tuberculosis or osteomyelitis, antibiotics can help the body clear the infection. Anti-inflammatory medications can help control conditions like arthritis. Reducing inflammation often leads to lower levels of amyloid proteins and fewer symptoms.
Different types of amyloidosis may require specific treatments, depending on which parts of the body are affected. These treatments don’t remove amyloid deposits or prevent the body from forming amyloid proteins, so they do not treat amyloidosis itself. Instead, they treat the symptoms of amyloidosis and help prevent and manage complications.
Kidney damage often develops in people with amyloidosis, and it is particularly a problem for people with the AA subtype. 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, or medications that help keep your bones strong.
Wild-type ATTR amyloidosis and hereditary ATTR amyloidosis can lead to heart failure. If you have heart symptoms, drugs like blood thinners can help support heart health. However, medications such as beta-blockers that are often used to treat other types of heart failure can be harmful for people with amyloidosis. If you have heart symptoms, it’s important to see a specialist who has experience treating people with amyloidosis.
Medications may be effective at slowing down amyloidosis and managing symptoms. However, sometimes surgery or other medical procedures may be needed when amyloid deposits damage a particular organ or organ system.
Your health care team may recommend an autologous stem cell transplant, or bone marrow transplant, in combination with the chemotherapy drug melphalan to treat AL amyloidosis. During this procedure, your blood stem cells are removed from your body. Then, you are given a high dose of melphalan, and about three days later, your stem cells are reintroduced to your body. This procedure may help reduce amyloidosis symptoms for a while, but it is not a cure.
A stem cell transplant comes with some risks and side effects, and it often requires a long recovery time. You may have to stay in the hospital for a few weeks following this procedure.
When amyloid deposits build up in the kidneys and these organs can no longer remove waste from the body, dialysis may help. Dialysis is a procedure in which an external machine filters wastes and toxins out of the blood. A kidney transplant may be a good option for some people with AA amyloidosis who have gotten their underlying inflammatory disease under control, but have experienced significant kidney damage.
People with dialysis-related amyloidosis typically have high levels of abnormal beta-2 microglobulin protein in the blood. 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 has been FDA-approved for dialysis-related amyloidosis.
For amyloidosis that affects the heart, sometimes called cardiac amyloidosis, you may need a heart transplant if the damage is severe. Hereditary ATTR amyloidosis is sometimes treated with a combination of a heart and liver transplant.
For people with amyloidosis, bone marrow, kidney, or heart transplants can treat the disease’s symptoms. However, for people with some forms of amyloidosis, a liver transplantation can actually target the disease itself. Hereditary ATTR amyloidosis develops when the liver makes abnormal TTR proteins. Getting a liver transplant and removing the dysfunctional liver may slow down or even completely stop the formation of new amyloids for some people with this subtype.
Clinical trials are underway to find additional treatments for amyloidosis. These studies can help researchers learn more about whether existing drugs can treat amyloidosis and allow them to better understand the effects of new drugs. Several clinical trials within the past few years have helped experts identify new treatment options. If you have amyloidosis, you may be able to participate in a clinical trial. Talk to your health care provider to learn more.
Amyloidosis Condition Guide