Research funded by Shake It Up Australia Foundation and the Michael J. Fox Foundation uncovers a promising new therapy for Parkinson’s.
This ground-breaking research, published in Science Translational Medicine was conducted at The University of Queensland.
The study demonstrated a key target in microglia, called the NLRP3 inflammasome, is highly activated in Parkinson’s. The same signals produced by inflammasome activation in human brains were seen in different pre-clinical models of Parkinson’s disease.
UQ Faculty of Medicine researcher Associate Professor Trent Woodruff said the team found that a small molecule, MCC950, stopped the development of Parkinson’s in several animal models.
“MCC950, given orally once a day, blocked NLRP3 activation in the brain and prevented the loss of brain cells, resulting in markedly improved motor function.”
There are no medications on the market that prevent brain cell loss in Parkinson’s patients, with current therapies focusing on managing symptoms rather than halting the disease.
UQ Institute for Molecular Bioscience researcher Professor Matt Cooper said drug companies had traditionally tried to treat neurodegenerative disorders by blocking neurotoxic proteins that build up in the brain and cause disease.
“We have taken an alternative approach by focusing on immune cells in the brain called microglia that can clear these toxic proteins,” he said.
“With diseases of ageing such as Parkinson’s, our immune system can become over-activated, with microglia causing inflammation and damage to the brain.
“MCC950 effectively ‘cooled the brains on fire’, turning down microglial inflammatory activity, and allowing neurons to function normally.”
Prof. Matt Cooper, Co-founder and CEO of Inflazome, commented: “We are indebted to The Michael J. Fox and Shake It Up Australia Foundations for supporting this work.
Prof. Cooper added “ We look forward to progressing this research through to clinical trials using our proprietary, improved drug candidates”.
Kuldip Dave, PhD, Director of Research Programs at The Michael J. Fox Foundation said, “Inflazome has validated a promising new target for Parkinson’s therapeutics and translated that finding into a potential drug to treat Parkinson’s disease. This is a key aspect of The Michael J. Fox Foundation’s research strategy, and we look forward to their candidate drug’s continued development.”
“Shake It Up is proud and excited to be a part of the collaboration’s funding team, allowing highly-talented researchers an opportunity to create world leading breakthroughs that have the opportunity to be a game changer for people with Parkinson’s,” said Clyde Campbell, Founder and CEO of the Shake It Up Foundation.
These results are very promising but as they are pre-clinical data the next step is human testing, in which the therapy goes through a rigorous (and lengthy) process to determine whether it is safe, tolerable and efficacious. The therapy needs to go through several steps (and many years) of testing before it may be ready and valid for human use. We will bring you updates as they become available.