New Advances in ALS and Alzheimer’s Treatments

Oak Brook, IL—SLAS Discovery’s Volume 40 spotlights four original studies that push drug discovery forward by engineering faster, more quantitative screening workflows. Across neurodegenerative disease, transporter biology, and protein–protein interactions, the issue highlights a unifying theme: turning complex biology into assay-ready, decision-making data at scale.

In the first study, researchers build a high-throughput platform using ALS patient iPSC-derived spinal motor neurons. They screen more than 6,000 compounds for changes in neurofilament light chain expression, a marker linked to disease state. Follow-up work refines the search to a lead candidate designed to avoid TGF-β inhibition, while also improving drug metabolism and pharmacokinetic properties—an example of how screening strategy and medicinal chemistry can be integrated early.

Another paper establishes an acoustic droplet ejection mass spectrometry platform tailored to solute carrier (SLC) targets. By quantifying a broad range of transported substrates, the method is validated across multiple SLC sub-families and scaled for hit identification. In a pilot campaign focused on SGLT2, the platform demonstrates how universal SLC analytics could accelerate the discovery of transporter modulators.

A third article tackles a particularly challenging drug target class: protein–protein interactions involving NOS1 and NOS1AP. Using a nanobit-based luminescence complementation assay, the team screens over 10,000 compounds and validates 19 hits, including three with potent inhibitory activity. The work underscores that sensitive signal engineering can make “undruggable” interfaces accessible to systematic screening.

Finally, a study employs affinity selection–mass spectrometry screening to identify a small-molecule binder for CAPON (NOS1AP). Screening roughly 10,000 compounds yields a lead candidate, MA32, with a dissociation constant of 74 µM. The result provides direct evidence of a druggable pocket on a target previously explored less deeply.

Together, these contributions position SLAS Discovery Volume 40 as a “technology-to-therapy” showcase—where assay innovation is not an accessory, but the engine of discovery. By combining throughput, quantitative readouts, and target-specific validation, the studies aim to reduce bottlenecks between early hits and meaningful chemical starting points.

Access to this volume and related journal information is available through SLAS Discovery.

News Publication Date: 1-Jun-2026

Web References: https://www.slas-discovery.org

References: High-Throughput Screening of ALS Patient iPSC-Derived Spinal Motor Neurons… (S2472-5552(26)00009-2) ; A Versatile Acoustic Droplet Ejection Mass Spectrometry Platform… (S2472-5552(26)00008-0) ; Development of a Nanobit-Based High-Throughput Screening Assay… (S2472-5552(26)00006-7) ; Identification of a Small Molecule CAPON Binder… (S2472-5552(26)00010-9)