hATTR Amyloidosis Treatment: A Glimpse at Available and Future Options
Promising therapies aim to treat hATTR and improve quality of life
Medically reviewed by Stella Bard, MD
Hereditary transthyretin amyloidosis, or hATTR, is a rare disease that causes proteins to build up in the body, forming amyloid deposits. This causes serious, wide-ranging symptoms, including heart disease, kidney failure, and nerve damage.
Amyloidosis treatment involves addressing the symptoms, including those of ATTR cardiac amyloidosis, while also stopping the protein production that makes symptoms worse. Promising treatments have been approved and more are undergoing clinical trials, including ATTR-CM treatments.
Continue reading to learn more about hATTR amyloidosis treatments that can slow the progression of the disease and improve your quality of life.
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hATTR Amyloidosis Drug Treatment
Drugs used to treat amyloidosis work in the following two ways:
They can stop the TTR protein from developing amyloid deposits, or protein buildup.
They can stop the production of the TTR protein
They can address the symptoms caused by hATTR. These are known as supportive therapies.
Here’s an overview of the type of amyloidosis medications in each category.
TTR Stabilizers
TTR stabilizers are drugs that stop the TTR protein from creating amyloid deposits. These deposits are what cause many of the symptoms of hATTR. The two types of TTR stabilizers are:
Diflunisal: This nonsteroidal anti-inflammatory drug (NSAID) binds to the TTR protein in the blood, stopping it from causing amyloid buildups. In addition to treating hTTR, Diflunisal is a medication that treats arthritis. It’s taken daily.
Vyndaqel and Vyndamax (tafamidis): These drugs are used to treat hATTR and ATTR cardiac amyloidosis. They bind to the TTR protein, to stop it from a process called folding and, therefore, building up in organs. The drugs are taken as a pill daily.
Another type of TTR stabilizer, acoramidis, is currently being studied in clinical trials.
TTR Silencers
The second class of medications are gene silencers. They stop TTR production by silencing the gene that causes the protein to form and build up. The three TTR silencers approved to treat hATTR are:
Onpattro (patisiran): Onpattro acts on the RNA that causes TTR production. The drug, which was approved in 2018, is given every three weeks intravenously (by IV).
Tegsedi (inotersen): Tegsedi is a once-a-week shot that patients with nerve damage from hATTR can give themselves at home. The medication acts of RNA, stopping the production of TTR in the liver.
Wainua (eplontersen): Wainua works in a similar way as Tegsedi. It also is a shot that patients can give themselves at home, but Wainua is administered once a month.
Supportive Therapies
In addition to treating hATTR, your healthcare provider may recommend medications to address its symptoms, including neuropathy and heart disease. These are called supportive therapies, because they support you in living a healthier, more comfortable lifestyle.
The supportive therapies for hATTR often include:
Medications to treat diarrhea or constipation
Always tell your healthcare provider about any other medications or supplements you’re taking before starting a new medication.
hATTR Amyloidosis Surgeries and Organ Transplant
Before 2018, the only treatment available for hATTR was a liver transplant. With the development of new medications, people with hATTR have been able to control their symptoms without a liver transplant.
However, a liver transplant is still an option for patients who don’t respond to treatments. Because the TTR protein is produced in the liver, a transplant may help stop production. Yet, some patients continue to have irregular TTR production even with a new liver.
Cardiac disease is one of the main symptoms of hATTR, some patients with the disease will need a heart transplant. Heart disease sometimes continues after surgery for patients with hATTR, so it’s very important to talk with your healthcare provider about the benefits and risks when considering this major surgery.
Over-the-Counter Medications to Manage Symptoms
Because hATTR causes many symptoms, some symptoms experienced with the condition hATTR may not seem related. It’s tempting to reach for over-the-counter (OTC) treatments like laxatives or diarrhea medicine without knowing the root cause.
Before you do that, talk with your healthcare provider. They can help you understand whether OTC medications could interact with prescription medication, and how they might work for you given the unique circumstances that hATTR presents.
Your healthcare provider can also suggest lifestyle changes, such as provider-approved exercise, that might help manage symptoms. Working with a physical therapist or dietitian familiar with hATTR and your symptoms might also help.
Complementary and Alternative Medicine (CAM) Therapies
Many people with chronic diseases find relief through CAM treatments, like acupuncture, massage, and holistic healing. Integrated medicine can be impactful, especially in addition to traditional medical treatment.
Be sure that any alternative providers you see understand hATTR, and talk with your medical healthcare provider about which alternative treatments might be helpful and which you should avoid.
Mental Health and hATTR Amyloidosis
Living with a life-threatening condition like hATTR can be very stressful, and can take a toll on your mental health. Consider seeing a counselor, or joining a support group for people with amyloidosis. Self-care can play an important role too.
hATTR Amyloidosis Treatment Breakthroughs
Since 2019, there have significant breakthroughs in the treatment of hATTR, and even more are on the horizon.
As of 2024, a medication that could remove protein buildups is in clinical trials. So are additional medications that block TTR production by targeting RNA.
Participating in clinical trials can open up new treatment options for patients with hATTR. Talk with your healthcare provider about the benefits and risks of participating in clinical trials.
Specialists and hATTR Amyloidosis Hospitals
You’ll need a group of healthcare providers to treat hATTR, including a hematologist (blood doctor), cardiologist (heart doctor), and neurologist (nerve doctor). The Amyloidosis Research Consortium has a tool that can help you find healthcare providers in your area that are familiar with the disease.
Many major university medical schools, including John Hopkins University, Boston University, and the University of Chicago, have amyloidosis treatment programs.
Summary
Hereditary transthyretin amyloidosis can be fatal, but there have been marked improvements in treatment in recent years. Medications and injections can stop or slow protein buildup, potentially sparing patients from more serious symptoms like heart disease and organ failure.
Working with a healthcare provider who is familiar with hATTR is crucial, since your treatment will likely involve many different medications and lifestyle adjustments.
Read the original article on Verywell Health.
