Nikhil Prasad Fact checked by:Thailand Medical News Team Oct 25, 2025 4 months, 3 weeks, 2 days, 3 hours, 45 minutes ago
Medical News: Experimental Drug Offers New Hope for Common Form of Heart Failure
Researchers from the Max Delbrück Center in Berlin, the University of Arizona College of Medicine in Tucson, and the University of Missouri have discovered a promising new treatment that could restore elasticity to aging and stiffened hearts. Their breakthrough may help millions suffering from a common and hard-to-treat form of heart failure known as heart failure with preserved ejection fraction, or HFpEF.
New Therapy Helps Aging Hearts Stay Flexible and Prevent Failure
As people age, the heart muscle naturally loses flexibility, making it harder for the heart to relax and fill properly with blood between beats. Unlike other types of heart failure where pumping ability is impaired, in HFpEF the heart still pumps efficiently but becomes too stiff to expand. Currently, there is no medication that effectively reduces death rates in patients with this condition. According to Professor Michael Gotthardt from the Max Delbrück Center, this
Medical News report highlights a potential therapy that directly targets the stiffness at the molecular level.
Restoring the Hearts Natural Elasticity
Gotthardt and his long-term collaborator, Professor Henk Granzier from the University of Arizona, developed a drug called RBM20-ASO. It is a small synthetic molecule known as an antisense oligonucleotide (ASO) that alters the production of a key protein in the heart muscle called titin. Titin functions like a spring, helping the heart expand and contract. In HFpEF, the body produces a stiffer version of titin. RBM20-ASO works by reducing the activity of a gene regulator called RBM20, allowing the heart to produce more of the flexible, youthful form of titin.
In mouse models of HFpEF, treatment with RBM20-ASO significantly improved the heart’s ability to fill with blood. The researchers observed that the mice’s hearts became more compliant, with reduced thickening of the heart muscle and improved blood flow during the relaxation phase.
Optimizing the Dose for Safety
Dr. Mei Methawasin of the University of Missouri, the study’s first author, led efforts to fine-tune the dosage to ensure safety. The team found that reducing RBM20 levels by about 50% was enough to restore healthy heart function without weakening its pumping ability. Even in animals with conditions like obesity and high blood pressure—common in HFpEF patients—the drug improved heart performance and reduced stiffness.
Importantly, side effects were minimal, and the team believes they can further minimize immune responses by adjusting the dosing intervals. Their findings suggest that this therapy could be developed as a standalone or complementary treatment for HFpEF, addressing the underlying mechanical cause rather than just the symptoms.
A New Frontier in Heart Failure Treatment
The research teams, supported by the Deutsches Zentrum für Herz-Kreislauf-Forschung and the Germ
an Research Foundation, plan to test RBM20-ASO in larger animal models before moving to human trials. If successful, this approach could represent a revolutionary shift in the treatment of heart failure in the elderly, potentially offering a way to restore youthful flexibility to aging hearts and prevent further organ damage.
The study findings were published in the peer-reviewed journal: Cardiovascular Research.
https://academic.oup.com/cardiovascres/advance-article/doi/10.1093/cvr/cvaf171/8286279
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