Cardiac amyloidosis: Better detection and new treatments

In early 2026, commercials featuring golfer Jack Nicklaus and actor Morgan Freeman discussing drugs for cardiac amyloidosis started appearing on television and online. Before then, many people had probably never heard of this rare, progressive heart condition. Caused by misshapen proteins that build up in heart muscle (and, often, in other organs), cardiac amyloidosis causes the walls of the heart to become thick, stiff, and unable to pump normally.

Currently, three drugs are FDA-approved to treat the most common form of cardiac amyloidosis, and more are in the pipeline. “These advances, along with improved recognition and earlier diagnosis, are helping extend the lives of people with the condition,” says Dr. Sarah Cuddy, assistant professor of medicine at Harvard Medical School and director of the Cardiac Amyloidosis Program at Brigham and Women’s Hospital.

What is cardiac amyloidosis?

Amyloidosis is a group of diseases characterized by clumps of misshapen protein (amyloid) in tissues throughout the body. Transthyretin (TTR), a protein that travels through the bloodstream carrying thyroid hormone and vitamin A, is the one of the most common proteins involved in amyloidosis that affects the heart. Known as amyloid transthyretin cardiomyopathy (ATTR-CM), it occurs in two forms: inherited and non-inherited. The latter, also known as wild-type ATTR-CM, is age-related and accounts for the vast majority of cases.

“About 85% of the patients in our clinic with ATTR-CM are men. But we believe the disease is underdiagnosed in women,” says Dr. Cuddy. (Of note: there is no known or direct connection between cardiac amyloidosis and Alzheimer’s disease, which is characterized by brain deposits of beta-amyloid, a distinct type of amyloid protein.)

Cardiac amyloidosis symptoms

The symptoms of cardiac amyloidosis, such as fatigue and shortness of breath, especially during physical activity, often overlap with symptoms of heart failure. Other problems include hard-to-control atrial fibrillation, an unusually slow heart rate (bradycardia), and feeling lightheaded upon standing. As the disease advances, blood pressure tends to drop, and people who have been taking blood pressure drugs no longer need them, says Dr. Cuddy.

Amyloid deposits in the joints, nerves, and other tissues can cause an array of problems, including a torn biceps tendon in the upper arm, carpal tunnel syndrome (a pinched nerve in the wrist), and lumbar spinal stenosis (a narrowing of the space around the lower spine). Although these conditions have other, more common causes, any of them can be an early warning sign of cardiac amyloidosis even before the heart-related symptoms appear, says Dr. Cuddy.

Cardiac amyloidosis diagnosis

Tests to check for cardiac amyloidosis often start with a heart ultrasound (echocardiogram), which can reveal the thickened heart wall that characterizes the problem. Other testing includes blood and urine analyses, a cardiac MRI, and a specialized nuclear imaging scan that checks for amyloid protein inside the heart. Occasionally, a procedure to obtain a sample (biopsy) of heart muscle is recommended to confirm the diagnosis.

Cardiac amyloidosis treatment

Treatments for ATTR-CM fall into two categories: stabilizers and silencers. Stabilizers work by binding to the TTR protein, preventing it from breaking down and creating more amyloid. Stabilizers include tafamidis (Vyndamax) and acoramidis (Attruby). “Both of these oral drugs slow progression of the disease and help keep people out of the hospital and alive longer,” says Dr. Cuddy.

Silencers are small molecules given by injection that work by blocking the liver’s production of the TTR protein. Like stabilizers, they also slow progression of the disease, reduce hospitalizations, and lengthen life. Currently, vutrisiran (Amvuttra) is the only silencer approved to treat ATTR-CM, although similar drugs are approved to treat different types of amyloidosis.

“These therapies have really shifted our expectations for people with cardiac amyloidosis, with most of them now living well beyond the original life expectancy of three to five years after diagnosis,” says Dr. Cuddy. More good news: all of the drugs are well tolerated, with few side effects. And even though their list prices range from about $250,000 to $475,000 annually, they are covered by Medicare, and most people pay no more than about $2,000 a year, Dr. Cuddy says.

Another major area of interest is medication that doesn’t just stop TTR in its tracks but also removes amyloid that has already accumulated in the heart, says Dr. Cuddy. Currently, two late-stage trials of such drugs in people with ATTR-CM are under way, with results expected within the year. Both drugs are monoclonal antibodies that bind to misshapen TTR protein or amyloid fibrils, triggering the body’s immune system to engulf and eliminate the amyloid deposits.

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