Heart disease is one of the biggest killers in Australia, but for people living with conditions like Friedreich ataxia or diabetes, the situation is even more stark: there are no effective treatments for the heart complications that will eventually cut their lives short. Part of the reason progress has been slow is that most research relies on animal models or oversimplified lab experiments that do not behave the way the human heart actually does. Better models mean better science, and better science means better treatments.
Dr Lees grows tiny beating pieces of human heart tissue in the laboratory, made from stem cells donated by people living with the diseases he studies. These “mini-hearts” let his team watch disease unfold up close and test potential new medicines directly on human tissue.
His lab currently uses this approach to study Friedreich ataxia, a devastating inherited condition that robs young people of their mobility and their heart function, as well as diabetic heart disease. A newer program is growing heart and kidney tissue together in the same dish, mimicking the way these two organs affect each other when diabetes takes hold.
For Friedreich ataxia specifically, his team is pursuing several therapeutic strategies in parallel: developing mRNA-based therapies targeting the molecular drivers of the disease, investigating whether restoring protective fats that are depleted in affected heart cells can improve heart function, and contributing to a collaboration screening over 1,000 existing approved medicines to identify candidates that could be repurposed to treat the condition.