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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 amyloidosis, 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 failure, heart palpitations (arrhythmias), fatigue, and swelling in the legs, 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 years 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.
Survival and quality of life improvements.
The HELIOS B study, officially named “Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”, was a phase 3, randomized, double-blind, placebo-controlled multicenter study. New England Journal of Medicine published the results.
The study randomly assigned 650 patients with ATTR-CM to one of two groups. The first group took vutrisiran for up to 36 months. Vutrisiran is delivered with an injection under the skin every three months. The second group received a placebo.
During the trial, another drug called tafamidis became the standard of care as the only currently FDA-approved treatment for ATTR-CM. Approximately 40% of trial participants, distributed evenly between groups, received background therapy with tafamidis.
When we analyzed the results, vutrisiran had clear benefits. Compared to the placebo group, participants assigned to this medication were:
- More likely to survive to the end of the study
- More likely to have a better quality of life
- Less likely to need hospitalization for reasons related to their heart
Our research observed no unwanted side effects from this medication. By all measures, this study succeeded, revealing an exciting new treatment option for patients.
How the study medication works.
Vutrisiran is an RNA inhibitor that blocks the production of the protein transthyretin, which can stick to heart tissue and result in cardiac amyloidosis. Vutrisiran degrades the messenger RNA molecule, a template for making transthyretin protein. By silencing the production of this misfolded protein, vutrisiran helps stop the progression of ATTR-CM.
Vutrisiran has already received approval from the U.S. Food and Drug Administration (FDA) for treating hereditary amyloid polyneuropathy—the nerve-affecting version of ATTR—and has been shown to improve patients’ quality of life and survival.
If vutrisiran is approved by the FDA for use in treating ATTR-CM, it will become the first RNA silencing treatment for this fatal disease. These treatments fall into one of two categories:
- Stabilizers: Tafamidis and another similar drug, acoramidis, stabilize transthyretin production. Acoramidis is currently being evaluated by the FDA as a treatment for ATTR-CM.
- Silencers: Vutrisiran stops the production of transthyretin
In addition, MedStar Health and other centers are currently studying other potential treatments, including the gene-editing technology CRISPR and the use of an amyloidosis-depleting agent to give patients and providers other powerful options to treat ATTR-CM.
Related reading: Why the Tiny Lp(a) Molecule is Big News for Heart Disease.
Research leadership from MedStar Health saves lives.
About ten years ago, ATTR-CM was a very different diagnosis for patients. There were very few available treatments, and most patients died less than four years after diagnosis. Things are very different now—thanks, in part, to research conducted here at MedStar Health.
These new treatments have turned ATTR-CM upside down, improving survival and patients’ ability to enjoy their lives. These treatments enhance functional capacity, making possible long walks, exercise, and other quality-of-life improvements.
MedStar Health is a leader in discovering and providing new treatments for this disease. We have the tools to identify patients with ATTR-CM who can benefit, and we have a center of excellence with top clinicians who can provide lifesaving and life-improving treatments.
At MedStar Health Research Institute, we’re proud to offer patients the opportunity to enroll in clinical trials that open access to tomorrow’s innovative treatments. Together, we are transforming care and making lifesaving treatment more equitable for all patients.
