Promising Progress in Parkinson’s Research: What’s New in 2025?

The latest edition of Cure InSight from Cure Parkinson’s brings some genuinely hopeful updates for people living with Parkinson’s. From major trials kicking off to new therapies being tested in the lab, here’s what’s happening right now in Parkinson’s research—without the fluff. 🚀 The ASPro-PD Trial Is Finally Underway One of the most anticipated trials in Parkinson’s research—the ASPro-PD trial—is officially up and running. After delays related to drug formulation, the first clinical site opened at University College London Hospital in February, with the first participant enrolled in March. This trial is testing ambroxol, a common cough medicine, to see if it can slow the progression of motor symptoms in Parkinson’s. Why ambroxol? It’s believed to boost the activity of an enzyme called glucocerebrosidase (GCase), which helps clear waste out of brain cells. Problems with this enzyme are thought to contribute to the build-up of toxic proteins in Parkinson’s. Half the participants in the study will have a specific gene variation (GBA1) linked to lower GCase activity, which makes them especially interesting to researchers. Over the rest of 2025, more sites across the UK will open. Recruitment is being done through the PD Frontline genetic screening platform. Cure Parkinson’s, Parkinson’s UK, and several research charities are funding the trial. 🔬 Smarter Trial Designs with SLEIPNIR The newsletter also introduces SLEIPNIR, a new type of clinical trial platform launching in Norway with support from Cure Parkinson’s. Named after Odin’s eight-legged horse, this trial will test up to three experimental therapies at once, using one shared placebo group. Led by Professor Charalampos Tzoulis, the goal is to quickly figure out whether the drugs actually reach the brain and interact with their intended targets. Samples of blood and cerebrospinal fluid will be collected to confirm this. It’s a more efficient approach than traditional trials and could help avoid expensive late-stage failures. The first round will include 120 participants. Promising drugs tested in SLEIPNIR could then move on to larger trials like EJS ACT-PD, another platform trial supported by Cure Parkinson’s. 🧪 From the Lab: New Projects You Should Know About Several new lab-based studies have been funded, targeting the roots of Parkinson’s progression: 1. iLCT Drug Screening at Sheffield Professor Heather Mortiboys is screening 100 drugs flagged through the International Linked Clinical Trials (iLCT) programme. Using neurons grown from the skin cells of people with Parkinson’s, her team will test how these compounds tackle three known drivers of Parkinson’s: poor energy production, waste build-up, and toxic proteins. 2. CP-6: Fighting Inflammation and Stress At the University of Pittsburgh, Dr Marco Fazzari is exploring a synthetic fatty acid called CP-6. It’s designed to reduce inflammation and oxidative stress—two major players in brain cell damage. This compound came recommended by iLCT reviewers for further study before human trials. 3. Small Molecules for Big Protection In Finland, Professor Mart Saarma is investigating four small molecules that mimic the effects of natural brain-protecting proteins like CDNF and MANF. These help neurons survive by easing stress inside the cell’s protein-folding machinery (called the endoplasmic reticulum). These molecules could slow the death of dopamine-producing cells in Parkinson’s. Why This Matters Parkinson’s affects more than 10 million people globally—and that number is expected to double by 2050. While existing treatments help manage symptoms, none yet slow or stop the disease. These trials and lab studies are part of a growing, global effort to change that. The takeaway? Real, targeted progress is being made. While science never moves as fast as we’d like, each of these studies brings us one step closer to the kind of therapies that could transform life with Parkinson’s.