Lab-grown ‘tiny hearts’ – developed with the support of the Phenomics Australia iPSC Derivation & Gene Editing Facility Node at Murdoch Children Research Institute (MCRI) and the Pipeline Accelerator scheme – have accelerated the way treatments are identified and tested for heart related disease.
Every 29 September, World Heart Day unites people globally to raise awareness of cardiovascular disease, the world’s leading cause of premature death.
Cardiovascular disease is a major cause of death in Australia, responsible for one in four of all deaths. Each year, cardiovascular disease (CVD) claims over 20.5 million lives. Up to 80% of premature CVD deaths are preventable through affordable access to care, early screening, and more opportunities to make healthy choices in nutrition and stay active.
Genetic heart disease and inherited heart conditions like cardiomyopathies, are incredibly difficult to understand and treat due to our limitations in disease modelling. Unlike cancer, cardiovascular disease, and heart failure, have very limited treatment options.
A collaboration between QIMR Berghofer, Murdoch Children’s Research Institute (MCRI) and the Royal Children’s Hospital has developed lab-grown ‘tiny hearts’ to address this gap and discover new therapeutics for patients. These ‘tiny hearts’ created by QIMR Berghofer’s Prof. James Hudson and fellow scientists mimic human heart muscle and behave like a genuine adult heart. With these organoids, researchers can model how the heart contracts and better understand how the heart changes with disease. The true breakthrough of this collaboration is the acceleration of the maturation process of the organoids, with the ability to make hundreds per week.
The Phenomics Australia Pipeline Accelerator played a fundamental role in assisting access to the iPSC Derivation and CRISPR Gene Editing Facility, a Phenomics Australia node, at Murdoch Children’s Research Institute. The early support of the voucher scheme has been instrumental in creating a good foundation for the team to translate their findings into larger drug discovery and disease modelling projects.
Professor James Hudson, Group Leader of the Cardiac Bioengineering Group at QIMR Berghofer, reflected:
“The support of the Voucher scheme enabled us to generate a novel line, which enabled us to perform lineage tracing of stromal cells in human cardiac organoids for the first time. This provided really important information to show that these cells resemble native cardiac cells. We hope to now extend these findings and discover new therapeutics targeting the stromal cells for heart failure.”
With these human cardiac organoids, researchers can be confident that the translation from petri dish to drug development will progress faster. Technologies like these allow researchers to screen new therapeutics and make it possible on a larger scale than previously done. The team has already had positive responses to various drugs that have known cardiac effects in the clinic and will begin larger screening projects to improve Australia’s ability to understand and treat cardiac disease in the future.
Phenomics Australia provides a national centre of expertise and service provision to deploy a more comprehensive and sophisticated range of in vitro Genome Engineering and Disease Modelling capabilities to understand the functional consequences of DNA sequence variation in the human genome for health and disease while maintaining the expertise for in vivo disease modelling and genome engineering. To meet the high demand for adaptable and scalable disease-modelling platforms for improved diagnosis, Precision Medicine for genetic disorders, and therapeutic development by both academia and the biopharmaceutical industry, Phenomics Australia offers In Vitro services through a collaborative consortium of ten laboratories and facilities across Australia, operating at ANU (ANU Centre for Therapeutic Discovery), Perkins (Translational Cancer Research Program in Oncology), Monash (Monash Organoid Program & Monash Genome Modification Platform), MCRI (iPSC derivation & Gene Editing Facility), Peter Mac (Victorian Centre for Functional Genomics), UMelb (Centre for Stem Cell Systems & Stem Cell Disease Modelling Laboratory), UQ (In vitro Genome Engineering and Disease Modelling Service), and VCCRI (Stem Cell Production Facility iPSC Reprogramming Service)
