MCRI has been announced as the only non-European site to receive a prestigious Horizon2020 grant.
The researchers, led by VCGS' Professor Ravi Savarirayan, will receive AUD$8.4million to investigate a new treatment for Schmid dysplasia, a rare genetic disease characterised by short-limbed dwarfism and a waddling gait due to abnormalities in the legs and hips.
In mouse models the researchers have found an anti-epileptic drug works to reverse the disease. The Horizon2020 grant will support the project to move into human trials next year.
With an application process that spanned two years, Prof Savarirayan said the group's strong experience in clinical trials was what got it across the line.
"Normally Australia would not be included, but we received special dispensation because of the clinical trial track record of my group at MCRI," he said.
The treatment holds the potential to correct hip deformities in skeletal dysplasia patients, doing away with the need for surgery.
"This treatment would save children time in hospital and away from school," he said. "It is bringing precision medicine to patients and helping them grow better."
Centred in the European Union, Horizon2020 is the world's largest financial program for research and innovation, with 80 billion euros to be invested in projects between 2014 and 2020.
In 2017 Horizon2020 put out the call for projects focused on new therapies for rare diseases. The Schmid dysplasia trial will be led by Mike Briggs - professor of skeletal genetics at University of Newcastle upon Tyne, UK. The research involves repurposing the anti-epileptic drug carbamazepine to treat young patients with the condition.
For more information about this trial or genetic conditions and testing, email [email protected] or phone the VCGS Customer Care team on 1300 118 247.