Researchers in Australia have found that a mysterious FDA-approved drug, called compound X, removes toxic alpha-synuclein proteins from the brains of mice with Parkinson's-like symptoms. The treatment improved the animals' balance and mobility by enhancing the brain's glymphatic waste disposal system. The findings were presented at a symposium in the UK.
Zhao Yan at Swinburne University of Technology in Melbourne tested compound X on mice exposed to misfolded alpha-synuclein via nasal drops weekly for four months, mimicking early-stage Parkinson's disease. After two months, half the mice received the drug four times a week alongside methylcellulose, while controls got only methylcellulose. Yan presented the results at the Oxford Glymphatic and Brain Clearance Symposium on 1 April, as first reported by New Scientist. 80% of treated mice successfully navigated a pole test, compared to 10% of controls. On a rotating rod, treated mice balanced for the full five minutes, while controls lasted about three minutes on average. Further tests showed compound X boosted slow brainwaves during deep sleep, increasing glymphatic fluid flow and reducing alpha-synuclein clumps in the motor cortex by 40% more than in controls. “We aim to put some intellectual property protection around the repurposing of compound X as it has shown significant findings so far, and could become the first disease-modifying treatment for Parkinson’s disease,” Yan said. Wenzhen Duan at Johns Hopkins University called the work important, noting current treatments only relieve symptoms without slowing progression. The team plans to seek approval for human trials in early-stage patients within a year, aiming for earliest intervention.
