University of Queensland (UQ) researcher Dr Margreet Ridder has been named the inaugural Dr Ian Davis OAM Award recipient for the 2024 top-ranked FightMND project.
Electrophysiologist Dr Ridder received a $285,000 FightMND IMPACT grant and the inaugural Dr Ian Davis OAM Award for the top-ranked grant in the 2024 round.
Based at UQ’s Queensland Brain Institute (QBI), Dr Ridder’s research explores using gene therapy to rescue motor neurons from hyperexcitability.
“Neurons are excitable cells, communicating with each other and muscles through chemical and electrical signals, and too much excitability (hyperexcitability) can cause neurons to die,” Dr Ridder explained.
“In amyotrophic lateral sclerosis (ALS), the most common form of motor neurone disease (MND), toxic hyperexcitability has been observed in motor neurons before they die, and as a universal feature of all forms of MND/ALS, it is an attractive target for developing treatments.
“Our gene therapy approach uses an engineered silencing receptor to turn down this hyperactivity in a mouse model for MND.
“This project builds on more than 12 years of research, and I would like to acknowledge Professor Pankaj Sah and Emeritus Professor Joe Lynch for bringing me to QBI to work on this.
“Not many people combine electrophysiology and gene therapy to develop treatments for MND, and it was challenging to get funding initially as everyone saw it as blue-sky research, but gene therapy for the treatment of MND has arrived.”
Dr Ridder explained that if the team can show that the silencing receptor is effective, it is already optimised for human use and activated by ivermectin, a safe FDA-approved drug.
“One of the first steps to bringing a new gene therapy to patients is to show its safety and effectiveness in preclinical trials.
“Our early data shows that our receptor can delay disease onset, increase survival in an MND mouse model, and preserve motor neurons in the early-stage disease.
“During this project, we aim to validate our preliminary data, bringing us a step closer to a human gene therapy,” Dr Ridder said.
Dr Ridder is one of 40 recipients identified through FightMND’s competitive grant round for a record investment of nearly $18.89 million into MND research and care initiatives for people living with MND.
At QBI, Associate Professor Adam Walker and Dr Rebecca San Gil also received FightMND grants to progress their MND research.
