BDSRA Australia visits Batten Disease research team, QLD Institute Medical Research
30 May 2023
I recently enjoyed a visit to the QIMR Berghofer Medical Research Institute in Brisbane to meet with BDSRA Australia Research Grant recipient Associate Prof. Tony White (pictured left), and team members Dr Zachary Gerring (middle right) and Dr Lotta Oikari (far right). The team are currently undertaking a ‘drug repositioning’ project to identify potential treatments for Batten disease, utilising computational and bioinformatic approaches, combined with in vitro cell model research. We know that drug development for the treatment of brain disorders like Batten disease is a notoriously slow and expensive process, with very low rates of success. Few compounds discovered in the laboratory will ultimately gain regulatory approval for clinical use in human patients. More efficient approaches to drug discovery are urgently needed.
The White research group has applied computer-based algorithms to analyse genes and proteins involved in Batten disease and compared these to known drug actions in these specific pathways. Through this, the team has identified drugs already approved for treatment of other diseases that may, theoretically, also target key genes and proteins in Batten disease. These drugs will now be tested on cell models of Batten disease. Outcomes from this project may potentially provide a rapid pathway for identifying drugs that could then be tested in advanced clinical trials in patients with Batten disease.
Read more about the BDSRA-funded research here.