In early 2026, commercials featuring golfer Jack Nicklaus and actor Morgan Freeman discussing medications for cardiac amyloidosis began appearing on television and online. Before then, many people had probably never heard of this rare, progressive heart disease. Caused by misshapen proteins that build up in the 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 on the way. “These advances, along with improved recognition and earlier diagnosis, are helping to 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 and carries thyroid hormone and vitamin A, is one of the most common proteins involved in amyloidosis, which 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 that the disease is underdiagnosed in women,” says Dr. Cuddy. (Note: there is no known or direct link 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 difficult to control atrial fibrillationan unusually slow heart rate (bradycardia) and feeling dizzy when you stand up. As the disease progresses, blood pressure tends to drop, and people who have been taking blood pressure medications no longer need them, says Dr. Cuddy.
Amyloid deposits in the joints, nerves and other tissues can cause a number 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.
Diagnosis of cardiac amyloidosis
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 tests include blood and urine tests, 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 the heart muscle is recommended to confirm the diagnosis.
Treatment of cardiac amyloidosis
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 the 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 the progression of the disease, reduce hospitalizations and prolong life. Currently, vutrisiran (Amvuttra) is the only silencer approved for the treatment of ATTR-CM, although similar drugs are approved for the treatment of various types of amyloidosis.
“These therapies have really changed 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 drugs are well tolerated, with few side effects. And while their list prices range from about $250,000 to $475,000 annually, they’re covered by Medicare, and most people pay no more than about $2,000 a year, says Dr. Cuddy.
Another major area of interest is medication that not only stops 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 underway, with results expected within the year. Both drugs are monoclonal antibodies that bind to malformed TTR protein, or amyloid fibrils, triggering the body’s immune system to engulf and eliminate the amyloid deposits.
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