Accelerating stem cell discoveries into tomorrow's medicine

The University of Melbourne and Stem Cells Australia have welcomed $3 million Federal Government funding to advance stem cell research towards new treatments.

The Accelerated Research program of the Medical Research Future Fund grant will be used to support national teams of experts to take stem cell research from the laboratory into the clinic.

The two initial projects will focus on using stem cells to test new ways to save the sight of children with rare genetic defects that slowly cause blindness, and to pioneer new approaches for the treatment of congenital heart disease.

Towards novel treatments for genetic causes of blindness:

This project aims to save the sight of children with rare genetic defects that slowly cause blindness. This would ultimately involve modifying the mutation within the patient’s eye using gene editing tools. The safety and effectiveness of this approach must be tested before moving to clinical trial. It is possible to build a model of a human eye from patient stem cells. Using this approach, researchers will carry out a unique human-based approach to determining whether this treatment is likely to work. The team includes clinical ophthalmologists with experience in taking novel treatments into eye and the charity Genetic Cures Australia. If successful, this innovative technology will have widespread application in many genetic and congenital diseases that affect millions of people.

Human heart tissue for understanding heart disease:

This project aims to repair and reconstruct tissues for kids with heart defects. More than 2000 children are born with major heart defects in Australia each year. While many can be saved via early and invasive surgical intervention, in many instances life span is limited and quality of life restricted. It is possible to generate human heart muscle from pluripotent stem cells. This provides the possibility of engineering contractile human tissue that may be able to improve the long term outcome for such children. There is a robust heart stem cell network nationally working right across the space of modelling heart disease, improving heart repair and potentially recreating heart tissue to treat kids with congenital heart disease. This group is well aligned with HeartKids and has strong links with the cardiac surgeons and cardiologists around Australia.

Stem Cells Australia Program Leader and University of Melbourne Professor Melissa Little said stem cell science has now advanced to the stage where it can impact future medical treatments.

“We can use stem cells to assess whether a new drug or gene therapy is safe and effective, as well as explore how to repair parts of the body through stem cell therapy,” Professor Little said.

“This funding will go a long way in helping our research.”

University of Melbourne Vice-Chancellor Professor Glyn Davis welcomed the announcement.

Stem Cells Australia’s expert team of doctors and scientists will be supported by partnerships with key Australian charities including Genetic Cures Australia and HeartKids.