Murdoch Children's Research Institute (MCRI) Professor Enzo Porrello has been awarded funding to better understand a serious childhood heart condition that could pave the way for new treatments.
Professor Porrello has received $5 million from the National Health and Medical Research Council (NHMRC) to investigate the genetic causes of dilated cardiomyopathy (DCM), the leading cause of heart failure in children. Many young people with a DCM diagnosis may also require a heart transplant.
Image: Professor Enzo Porrello
The five-year Synergy Grant is designed to bring together a range of experts to solve complex problems.
The funding will enable Professor Porrello to integrate elements of genomics, proteomics (the study of proteins), stem cell modeling, patient data and AI tools together into a streamlined platform that could lead to new drug treatment options for DCM.
"As far as heart conditions go, DCM is fairly common and can lead to heart transplants, but despite this our current treatment options only target the symptoms and not the underlying causes of the disease," Professor Porrello said.
"This research will help us to better understand what issues occur in human heart tissue affected by DCM and how new or existing drugs may alleviate these problems."
Professor Porrello said that his team, including Dr Sean Humphrey, would use human heart tissue grown in the lab from stem cells to address current gaps in our understanding of DCM and to develop precision therapies.
Image: Dr Sean Humphrey
"Our cutting-edge stem cell model will help us find out if specific genetic mutations that cause DCM can be targeted with tailor-made therapies that could halt or reverse disease," he said.
"We hope the study will also inform future research and lead to precision medicine options for other forms of heart disease."
Researchers from the Garvan Institute of Medical Research and Gladstone Institutes will also contribute to the study.
Professor Porrello has also been recently named as a Fellow of the International Society for Heart Research. This accolade recognises Professor Porrello's significant contributions to the field of cardiac research and his commitment to advancing stem cell medicine through heart tissue regeneration.
