Exploring a Vertigo Drug’s Potential in Early Parkinson’s - in top 3 research pick in 2024

There have been several summaries of what happened in 2024 in the world of Parkinson’s. Typically, the most comprehensive reviews come from the research community, as everything is well-documented. We’d like to draw your attention to research review by Dr. Simon Stott, Head of Research at Cure Parkinson’s. In his review, Dr. Stott goes month by month through last year, highlighting the most interesting scientific discoveries that bring us closer to better managing and ultimately ending Parkinson’s. Many of these findings have been covered in our PD News, but his review is excellent, and we highly recommend reading it. Dr. Stott highlights three major events, including the disappointing results of the Phase 3 trial of Exenatide, which we discussed extensively a couple of months ago. His second pick is a new way to analyse lysosomes (tiny cell "rubbish bins") using blood samples. This could be a major breakthrough for Parkinson’s, as lysosomes play a key role in the condition. This year also saw big progress in Parkinson’s biomarkers, including tests for alpha-synuclein (linked to disease stages), DOPA decarboxylase in spinal fluid, and mitochondrial DNA damage. Efforts like the AMP PDRD project aimed to combine these findings into larger datasets, helping researchers diagnose Parkinson’s more accurately, identify subtypes, track disease progression, and develop better treatments. His third pick is something quite intriguing. We recently discussed a small study on a vertigo drug available in France, which is being explored for potential repurposing in Parkinson’s treatment. The researchers looked into whether Acetyl-DL-leucine could slow the early stages of Parkinson’s. They tested it on two people with REM sleep behaviour disorder (RBD), a condition where people act out their dreams while asleep. RBD is often an early warning sign of Parkinson’s, appearing years before diagnosis. The two participants took the drug for three weeks, and their RBD symptoms improved, staying better for over 18 months with continued treatment. Interestingly, brain scans after 22 months showed changes that might suggest the drug could reverse some early Parkinson’s-related damage. However, there was a concern: both participants showed signs of declining memory and thinking skills during the study. For one person, this led to mild cognitive impairment. It’s unclear if this was caused by the drug or was simply part of their condition, so more research is needed. It’s worth noting this was a tiny study with just two people. Larger studies are necessary to confirm the results and understand how the drug works. While it’s too early to say if Acetyl-DL-leucine could help with Parkinson’s, these findings were fascinating, especially the brain scan results. It’s an interesting choice for Dr. Stott to include this discovery in his top three. We’re curious whether this means Cure Parkinson’s will be exploring this research further. We’ll have the opportunity to find out next week and ask Dr. Stott directly during his annual research review webinar with No Silver Bullet on 13th January at 7:30 PM (London time).