Image of a heart with a DNA double helix in front of it to indicate cardiovascular disease and the impact of transthyretin amyloid cardiomyopathy (ATTR-CM) which can be treated with vutrisiran.
Credit: iStock/SvetaP DNA double helix: iStock/Kagenmi

A new partnership to study heart failure with preserved ejection fraction (HFpEF) is being launched by the National Institutes of Health and several private corporations. The Foundation for the National Institutes of Health (FNIH) and the National Heart Lung and Blood Institute (NHLBI) will work with partners that include Bayer US, Cytokinetics, Ionis Pharmaceuticals, Novartis AG, and Ultromics.

The goal of the Accelerating Medicines Partnership Heart Failure (AMP HF) program is to develop precision treatment strategies for HFpEF. It brings together the resources of eight partner organizations spanning the public and private sectors, with total commitments of over $37 million. The FNIH will provide project management for the effort over the next five years.

“Roughly half of all heart failure patients suffer from HFpEF. Understanding what it is, when it happens, and how to treat it remains the single largest unmet need in cardiovascular health. The AMP HF Program aims to close this gap in understanding, and ultimately improve the lives of patients everywhere,” said Norman Stockbridge, Director of the Division of Cardiology and Nephrology at the Office of Cardiology, Hematology, Endocrinology and Nephrology at the U.S. Food and Drug Administration (FDA).

Using cutting edge technologies, including digital measurements and artificial intelligence analytic methods, the new program is designed to find novel proteins or genes that could mitigate this disease when altered by therapeutics.

“We know that treatments that target the biological changes that drive disease are often most effective, but the challenge faced by researchers is finding the right targets,” said Lawrence A. Tabak, DDS, PhD, who is performing the duties of the NIH director. “AMP Heart Failure aims to improve the odds of hitting the mark earlier and faster.”

Although compelling progress has been made in the treatment of many forms of heart disease, death due to heart failure continues to rise nationally. In the United States alone heart failure directly contributes to about 45% of all cardiovascular disease deaths.

“The promise of precision treatments for heart failure is that we will have the opportunity to diagnose individuals much earlier and intervene, changing the course of this disease,” said NHLBI Director Gary H. Gibbons. “The AMP Heart Failure program—and the high caliber of the partnership at its core—will help us better understand and treat this common syndrome with the goal of ultimately benefitting millions.”

HFpEF is a common form of heart failure in which the ejection fraction—the percentage of blood ejected from the left ventricle with each heartbeat—is within the normal range. HFpEF is difficult to detect, because the left ventricle appears to be functioning normally, and is often deadly, with a five-year survival rate of just 35-40%. In addition to a high risk for mortality, patients with HFpEF live with declining quality of life and poor capacity to perform tasks of daily living.

The AMP HF Program, a public-private partnership facilitated by the FNIH, will advance our understanding of heart failure with preserved ejection fraction using two complementary and integrated research components: analyzing existing HFpEF datasets, sourced from public and private sector funded studies, and initiating a new clinical trial to confirm retrospective findings in an observational cohort with a goal to develop a framework for new precision treatments.

“HFpEF is clearly a major cause of heart failure hospitalizations and diminished quality of life for older patients. Up until now, developing effective therapeutic strategies to identify and treat HFpEF has eluded us. Through AMP HF, we will harness the valuable perspectives and expertise that collaborations bring to biomedical research, paving the way for a more hopeful outlook,” said Julie Gerberding, Chief Executive Officer at the FNIH.

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