Core Day 2025 is our pit crew

The Starting Grid
The ALS Grand Prix: Racing Toward a Cure The fight against ALS is no longer a slow marathon; it’s a high-octane Grand Prix. We are moving off the practice tracks and onto the professional circuit, where precision, speed, and global teamwork are the only ways to take the checkered flag. The Starting Grid: Advancing the Foundation Every great race team needs a world-class garage. Core Day 2025 is our pit crew headquarters. By bringing together investigators from our Global Natural History Study and Postmortem Tissue Core, we are ensuring our "engine"—the data—is tuned to perfection. This collaboration allows us to review the telemetry (data collection) and evolve our resources so every researcher has the tools to compete at the highest level.

The Engineering Bay: Tissue & Genetics To win, you need to understand the mechanics of the vehicle. We are expanding access and equity in our tissue collections to ensure our research represents the entire global "driver" population. By using "Formula 1" level tech like long-read sequencing and spatial mapping, we are looking under the hood at the cellular level to fix the microscopic malfunctions that cause the engine to fail. Fueling the Race: Biofluids & The GNHS A race car is nothing without the right fuel and monitoring. Our Global Natural History Study (GNHS) has already "lapped" expectations, enrolling 229 participants with a goal of 1,000. With 23,000 samples of CSF, blood, and urine, we have a massive reservoir of biological "fuel" to analyze. Expanding the circuit to India and South Korea ensures that our data isn't just fast—it’s globally diverse.

The High-Speed Straightaway: Drug Discovery Most ALS cases (90%) are sporadic, meaning we’ve been racing without a map. While past treatments only patched up the bodywork, this new wave of therapies is designed to fix the ignition switch before the fire starts. Through our Industry-Led Collaborative Consortia, we are funding the "aerodynamics" of drug discovery, pushing programs out of the lab and onto the clinical trial track. The Podium Contender: QurAlis & QRL-101 One of our star "drivers," QurAlis, is making waves with QRL-101. This precision therapy targets neuronal hyperexcitability—the "overheating engine" of ALS. The Pit Report: Early trials show the drug hit its target and was well-tolerated. The Next Lap: They are now prepping an extended-release version, allowing the treatment to stay on the track longer and work more effectively for people living with ALS.

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