Ex situ heart perfusion: A novel platform to test cardiovascular therapeutics in human hearts

Background: Explanted hearts from recipients undergoing cardiac transplantation may be utilized as a human model of cardiomyopathy. Ex-situ perfusion of hearts allows control of the physiological and biochemical conditions of perfusion. AZD8601 is a novel modRNA for VEGF-A that was shown to be safe...

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Main Authors: John Onsy Louca, Magnus Althage, Marco Öchsner, Aravinda Page, Joao Pedro Nunes, Catherine Wilson, Sanjay Sinha, Simon Messer, James Matt Bae, Mostin Hu, Nicole Asemota, Sarah Fielding, Sai Bhagra, Neil Henderson, Johnny Lindquist, Daniela Später, Anna Collén, Kaushik Sengupta, Benjamin Challis, Justin Perkins, Stephen Large
Format: Article
Language:English
Published: Elsevier 2025-08-01
Series:JHLT Open
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Online Access:http://www.sciencedirect.com/science/article/pii/S2950133425001314
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Summary:Background: Explanted hearts from recipients undergoing cardiac transplantation may be utilized as a human model of cardiomyopathy. Ex-situ perfusion of hearts allows control of the physiological and biochemical conditions of perfusion. AZD8601 is a novel modRNA for VEGF-A that was shown to be safe in a Phase IIa clinical trial – the EPICCURE trial. This proof-of-concept study aimed to demonstrate the potential utility of testing novel therapies on explanted recipient hearts using ex situ machine perfusion. Methods: In order to ascertain the expression of VEGF-A in a human model of cardiomyopathy, AZD8601 was injected at high- and low-dose into the mid-myocardium of the left ventricle of human hearts explanted at the time of cardiac transplantation and perfused on the m0rgan, a novel, normothermic organ perfusion machine. Hearts were perfused ex situ for 6 h. After which, injection sites were biopsied and divided into subendocardium, mid-myocardium and sub-epicardial myocardium. Immuno-analysis was performed to assess levels of VEGF-A protein. Results: There were elevated levels of VEGF-A protein in the subendocardium and mid-myocardium of injection sites which received AZD8601. Low-dose and high-dose AZD8601 resulted in a significantly higher concentration of VEGF-A protein in the myocardium. Conclusions: This study builds on the EPICCURE study, a phase IIa clinical trial which demonstrated safety of this mRNA in patients undergoing coronary artery bypass grafting. This study provides a novel model of diseased human heart for experimental studies utilizing ex situ heart perfusion.
ISSN:2950-1334