Physicians in Pursuit of the Self-Healing Heart
In the lab of Bernhard Kühn, MD, new findings offer the potential for use of an existing drug to treat children with heart disease by allowing the heart to heal itself.
As director of UPMC Children’s Pediatric Institute for Heart Regeneration and Therapeutics (I-HRT) and associate director of the Richard King Mellon Institute for Pediatric Research, Dr. Kühn investigates the inner workings of heart muscle cells (cardiomyocytes) to understand how these cells grow and regenerate. This information is enabling Dr. Kühn and his team to develop therapies to help the heart recover from a heart attack, or restore a congenital heart defect to normal cardiac function without requiring surgery.
Through extensive lab-based studies, Dr. Kühn and his team found that propranolol — a 60-year-old beta-blocker primarily used to treat high blood pressure — successfully generated new heart cells in animal models.
To determine whether propranolol could trigger increased heart muscle growth in infants, Dr. Kühn recently moved this project into a novel clinical trial that will begin enrolling patients this year. The first clinical trial of its kind, the study offers the promise of a new therapeutic approach that could delay the need for surgery and improve long-term health outcomes for patients with heart disease.
The trial investigates how propranolol might promote regeneration of heart muscle in babies with tetralogy of Fallot, a congenital heart condition that dramatically decreases healthy heart cell growth. The trial will enroll 40 babies with the condition. Physicians will provide standard of care to half of the infants, and half will receive propranolol in addition to standard of care.
Dr. Kühn and his team are furthering several additional projects, including research centered on defining how mature cells reproduce — which has the potential to advance regenerative therapies for heart disease, neurological diseases, and diabetes.
Prior to his groundbreaking work in the field, it was assumed that heart muscles stopped regenerating after birth. The Kühn Lab has demonstrated that healthy children typically generate new heart muscle cells up to age 20. “It became clear to me that pediatric heart regeneration could be more easily achieved,” Dr. Kühn notes in the 2018 UPMC Health Beat article, “The Self-Healing Heart.” “I’m a pediatric cardiologist, so I wanted to look at the patients who would be potential recipients of our therapies. That was a lot closer to my heart and mind.”
About the Richard King Mellon Institute for Pediatric Research
At the Richard King Mellon Institute for Pediatric Research, physician-researchers work every day to understand and develop treatments for some of the most debilitating illnesses in the world.
Founded in 2007 through a transformational gift from the Richard King Mellon Foundation, the Mellon Institute at UPMC Children’s supports catalytic scientific discovery centered on child health. With an emphasis on multidisciplinary collaboration, the Mellon Institute facilitates pioneering research that has the potential to transform the medical landscape.
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