New treatments have greatly expanded therapy options for people with transthyretin amyloid cardiomyopathy (ATTR-CM), but more research is needed to understand which therapy offers the greatest benefit, how best to tailor treatment to each patient and how to measure the long-term benefits of treatment, according to a recent review in the European Heart Journal.
ATTR-CM happens when a protein called transthyretin (TTR) loses its shape and forms amyloid deposits in the heart. Over time, these deposits make the heart stiff, and reduce its pumping ability.
The review’s authors explain that ATTR-CM develops when the body produces amyloid faster than it can clear it away. Because the body removes amyloid very slowly, these deposits gradually build up in the heart and cause it to weaken. They emphasize that effective treatments should not only slow the formation of new amyloid but also help the body remove the deposits that have already formed.
Several new types of medications are now available. TTR stabilizers like tafamidis and acoramidis keep the TTR protein in its normal shape and slow amyloid buildup. TTR silencers like vutrisiran (which was approved by the FDA this year) and eplontersen work by reducing production of the transthyretin (TTR) protein in the liver and helping to slow amyloid buildup in the heart. Gene-editing therapy with nexiguran ziclumeran offers a potential one-time treatment that permanently turns off the TTR gene.
“However, these approaches do not enhance the body’s limited capacity to clear existing amyloid deposits, underscoring the need for novel agents that accelerate amyloid removal,” the review’s authors said.
Read more about treatment and care for ATTR-CM
The good news is that new treatments called monoclonal antibodies are being developed for ATTR-CM. These treatments were designed to clear pre-existing amyloid deposits from the heart. Early studies suggest they might slow down the disease and improve heart function.
While they note that these developments are promising, the authors emphasize that more research is needed to understand the best way to use these treatments, who will benefit most and how to measure if they’re working.
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