SPG56 gene therapy the goal
Tiny brains grown in the lab could help University of Queensland (UQ) researchers find a treatment for SPG56 HSP and, in the process, unlock therapies for a range of other HSPs and different neurological disorders.
Professor Ernst Wolvetang and his team at UQ’s Australian Institute for Bioengineering and Nanotechnology (AIBN) have secured almost $1 million from the Medical Research Future Fund (MRFF) to test gene therapies for children with SPG56 HSP.
SPG56 is a very rare degenerative brain disease that normally begins in childhood and continuously worsens throughout life. Commonly, children with SPG56 lose the ability to sit, stand, walk or talk.
There is currently no cure or treatment for SPG56 but Professor Wolvetang hopes to find one by testing gene therapies on patient-derived organoids, which are tiny, synthetic organs grown from patients’ own cells.
“The AIBN is aiming to lead the nation in the field of personalised medicine and this testing will help us create a pathway to faster, more accurate treatments for children with SPG56 and other forms of HSP,” Professor Wolvetang said.
“We will test whether gene therapy is safe and effective in improving disease features in brain organoids that have the same genetic make-up as the patients we aim to help.
“Because we have hundreds of brain organoids from each individual patient growing in the dish, we have the luxury of systematically testing the best gene therapy approaches without risking harm to the patient.”
The SPG56 project will be the first time in Australia that brain organoids have been used to test the safety and efficacy of the gene therapy approach for HSP, and he says it may help foster change in the regulatory approval process that currently still requires extensive testing in animals.
“We hope that pre-clinical testing of the efficacy and safety of our methods in patient specific brain organoids is going to enable more rapid progress towards human trials,” Professor Wolvetang said.
“Once we demonstrate the power and accuracy of this approach for one genetic disease, it could open the door for testing of other gene therapies for a range of genetic neurological conditions.”
The Foundation contributed to the successful grant application, collaborating with Prof Wolvetang and others.
SOURCE: Australian Institute for Bioengineering and Nanotechnology, University of Queensland
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AIBN Communications, Alex Druce, 5 April 2023