Grant Allows Researchers to Test Gene Therapy on Personalized Brain Organoids

Rare Daily Staff

Ernst Wolvetang, professor at the Australian Institute for Bioengineering and Nanotechnology at The University of Queensland, has secured a $672,000 grant (AUS $1 million) to test gene therapies for children with a specific type of hereditary spastic paraplegia using brain organoids derived from patients’ cells.

HSP Type 56 (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. Wolvetang, though, hopes to test potential gene therapies on patient-derived organoids.

“The AIBN is aiming to lead the nation in the field of personalized medicine and this testing will help us create a pathway to faster, more accurate treatments for children with SPG56 and other forms of HSP,” 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.”

He said because there are hundreds of brain organoids from each individual patient growing in the dish, researchers have the luxury of systematically testing the best gene therapy approaches without risking harm to the patient.

Wolvetang’s 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. He said 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,” he 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.”

Photo: Ernst Wolvetang, professor at the Australian Institute for Bioengineering and Nanotechnology at The University of Queensland