Key Information
MedStar Health was the first and among the most prominent U.S. enrollers of participants in this important global Phase III clinical trial of a new drug to treat transthyretin amyloidosis with cardiomyopathy.
The groundbreaking HELIOS B clinical trial has demonstrated the effectiveness of a new treatment for one type of cardiac amyloidosisexternal link, opens in a new tab, a fatal condition that disproportionately impacts Black patients.
The MedStar Health Research team at MedStar Washington Hospital Center enrolled the first patient in the world—and was among the top enrolling research sites—in this study to explore the effectiveness of vutrisiran. This new medication may stop transthyretin amyloidosis with cardiomyopathy (ATTR-CM) progression in its tracks.
ATTR-CM causes instability and misfolding of transthyretin which results in the formation of insoluble fibrils known as amyloidosis. Usually, transthyretin transports vitamin A and thyroid hormone to tissues throughout the body. With ATTR-CM, these misfolded proteins join together and act like cement, attaching to heart tissues and causing cardiomyopathy (and neuropathy), which can result in heart failureexternal link, opens in a new tab, heart palpitationsexternal link, opens in a new tab (arrhythmias), fatigue, and swelling in the legsexternal link, opens in a new tab, and tingling in the hands and feet.
Over time, ATTR-CM may progress, leaving patients unable to perform daily tasks and impairing quality of life. Without treatment, survival related to ATTR-CM is limited with estimates of 2.5-3.5 yearsexternal link, opens in a new tab after diagnosis.
It is currently estimated that as many as 120,000 adults in the U.S. may have or be at risk for ATTR-CM. This condition can be caused by a genetic variation (hereditary ATTR-CM) that is passed down in families. The most common variant of hereditary ATTR-CM is found in 3-4% of all Black individuals in the U.S.
The HELIOS B study has revealed an effective new treatment for this once-fatal condition.