A gathering of supporters for the Murdoch Children’s Research Institute (MCRI) in New York on Thursday night turned into the stage for a striking medical announcement: researchers say they have developed a gene therapy that not only prevented heart disease in animal studies but reversed cases where the disease had already taken hold.
The event, held at Old Mates, the Australian-style pub in Manhattan co-owned by actor Hugh Jackman, brought together MCRI supporters for a night of Australian food — sausage rolls, oysters Kilpatrick and pavlova — alongside a scientific presentation from Professor Enzo Porrello, MCRI’s Theme Director of Stem Cell Medicine and head of its Heart Regeneration Group.
Porrello told the crowd that pediatric heart disease is far more common than many people realize. “Heart disease in children is pretty common,” he said. “It affects one in 100 children… that’s about 40,000 children born in the US every year.”
His research focuses on the most severe cases, where children’s hearts fail entirely. “The only treatment for heart failure is a heart transplant,” Porrello said. “And transplants are not a cure. It’s really a last resort. And we really need better therapies — more targeted therapies — they get to the root cause of disease.”
A single injection, tested in animals
Speaking on a panel alongside Old Mates co-owner Nicholas Stone, a founder of Bluestone Lane who also sits on MCRI’s Global Advisory Board, and Sarah Murdoch, MCRI Co-Chair and Chair of its Global Advisory Board, Porrello described a gene therapy delivered as a single-shot injection that has shown remarkable results in animal studies.
“We’ve shown in animal studies that not only can this gene therapy prevent the development of heart disease,” Porrello said. “It can actually completely reverse established heart disease… which is incredible. We’ve never seen anything like that in our sort of 15 to 20 years of doing this work.”
Porrello said the next step is moving the therapy out of the laboratory. “We’re super excited now to take that gene therapy out of the lab and get it into clinical trials,” he said. “So our focus now is, ‘How do we move this as fast as possible.'” He also walked the audience through the steps required to bring the therapy toward human trials.
Honoring the institute’s founder
Sarah Murdoch used her remarks to highlight the legacy of MCRI co-founder Dame Elisabeth Murdoch. “She was an extraordinary woman,” Murdoch said, noting that when the institute was founded, genetic science was far less advanced. “We had not mapped the human genome, we didn’t know about genetics the way we do today. And we know that almost every disease has a genetic course. Fast forward to now, we’re top three in the world. It’s extraordinary. I think that what makes it special is that her determination… her legacy lives on deeply through the institute.”
Murdoch also credited researchers like Porrello for being positioned close to patients. “You need researchers like Enzo… located in the hospital where they see the children unwell” to “find answers,” she said.
Goldman Sachs returned as presenting sponsor of the event for a second consecutive year, with organizers thanking the firm for its continued support. Asena Advisors, Bondi Partners and Fabletics also sponsored the gathering, which included a raffle and a preview of a new animated short, “For All. Forever,” marking MCRI’s 40th anniversary. The animation will be narrated by Jackman.
