Potential “best-in-class” therapeutics

Commencing January 2020, Dr Steven Petratos, Monash University, was awarded a 3-year Trish Translational Research Project Grant titled, “Developing a new drug to repair the brain in Multiple Sclerosis”.

Dr Petratos and his team have been looking to repurpose a drug previously approved for use in a different neurological condition.  He had previously shown that this drug may slow the progression of MS in a laboratory model of MS.

Despite the disruptions and challenges caused by COVID early in his work, Dr Petratos has made exceptional progress.

Dr Petratos and his group uncovered that in the brains of individuals living with progressive MS, there are protein changes that cause the death of brain cells. These proteins are in the cells that produce myelin. Importantly, the group has uncovered that a new class of medicines, known as small molecules, can access the brain to stop the death of these brain cells by protecting these proteins. This outcome has major implications in the protection of the myelin forming cells in the brain and can limit further damage imposed by the immune attack as seen in the progression of MS. Dr Petratos has been investigating whether the drug can also reverse the damage to the brain by activating stem cells to become myelin-forming cells. In experiments with the animal model of MS, his team has now shown that the drug can repair the damaged myelin through a process known as remyelination. These outcomes will allow entry into a future Phase 2 clinical trial in Australia to stop and reverse the damage to the brain that occurs in progressive MS. A service agreement has been now entered into with Monash University and the commercial company NeuOrphan Pty Ltd that will progress this drug toward the Clinical Trials.

Dr Petratos and his team have been collaborating with researchers at the University of Tasmania trialling the MCT-8-independent thyroid hormone analogue DITPA, as a potential therapeutic agent in demyelination to promote protection and repair of the brain during the immune attack against myelin. He is also the holder of a patent for the use of this drug for neurological conditions that has been granted in Australia, US, Canada and Europe.

The repurposing of the drug DITPA may have the potential to become a “first-in-class” therapeutic for individuals living with MS if clinical trials demonstrate that it can protect and potentially repair areas of the brain, spinal cord and optic nerve that have been damaged due to the disease. With further commercial funding on the horizon, this work will promote the development of potential “best-in-class” therapeutics that may promote substantial pharmaceutical industry interest in a series of new medications formulated for protection and repair of the brain during MS.

The Trish Foundation is proud and honoured to be contributing to the ground-breaking work of Dr Petratos, having first funded his research in our inaugural round of funding in 2002.

Dr Petratos, working with Professor Paul Stupple, The Monash Institute of Pharmaceutical Sciences, has been awarded a 2-year Trish Translational Research Project Grant commencing January 2023 titled, “Development of Small Molecules to Promote Remyelination in Multiple Sclerosis”.  The Foundation’s volunteer team and our generous Sponsors and donors look forward to learning of more advancement of Dr Petratos’ promising research.