50 Million euro investment for LUMC spin-off RougeTx advances therapies for inherited bleeding disorders.
Congratulations to reNEW Melbourne PIs Professor Enzo Porrello, Professor Andrew Elefanty and AI Dr Jessica Vanslambrouck, based at Murdoch Children’s Research Institute, who have each received two-year Stem Cell Therapies Mission grants from the Medical Research Future Fund (MRFF), worth a total of $3 million.
Professor Porrello and his team are internationally recognized for their ability to create human heart muscle cells (cardiomyocytes) from stem cells at a scale that permits screening of tens of thousands of candidate drugs.
Using these human cardiac tissues, the team will identify candidate drugs that promote human cardiomyocyte proliferation; an approach that may reduce the need for transplantations in patients with heart failure.
Importantly, using these human cardiac tissues allows the team to identify compounds that have any safety concerns much earlier in the drug development pipeline than traditional approaches which is a leading cause of drugs failing to reach market.
“Developing improved treatments for heart failure is critical; the disease affects 64 million people and is the leading cause of death globally,” Professor Porrello said.
Professor Elefanty and his team’s work is based on their recent discovery, which enables the differentiation of human induced pluripotent stem cells (iPSCs) in the laboratory to form blood stem cells (termed iHSCs).
This breakthrough will generate personalised stem cell-based therapies with the potential to transform clinical care.
“While our initial findings have been promising, we need to refine this process through further lab testing before it can be offered as a potential treatment in the future,” Professor Elefanty said.
“This stem cell technology could one day ensure leukemia patients and those with serious blood disorders don’t have to wait for a bone marrow transplant or fear organ rejection-related symptoms.”
Dr Vanslambrouck will combine living kidney cells with mechanical blood filtration as a proof-of-concept for future bioartificial kidney devices.
Such a device could be a potential solution to donor kidney shortages.
“Chronic kidney disease (CKD) is a leading cause of death and illness with limited treatment options. About four million people globally currently rely on regular regimes of blood dialysis (hemodialysis) and only a third of waitlisted patients will receive a donor kidney.” Dr Vanslambrouck said.
“We are working towards a game-changing method that could greatly improve quality of life and longevity for more patients with CKD”.
The Medical Research Future Fund (MRFF) is a $22 billion long-term investment by the Australian Federal Government, to support Australian health and medical research.
