Q1 2026 Research we Enable Update

March 31, 2026 Amy Easton


New Data Engine Release Now Live

A major update was deployed to the Target ALS Data Engine in February. The Global Natural History Study, Community-Based Pop-Up Outreach Study, and Stem Cell Core collections now feature harmonized column names and standardized column values across tables. This work is designed to make it easier to integrate data across collections and modalities while reducing friction in downstream analysis. A similar harmonization effort for the Postmortem Tissue Core collection is already underway and will be completed soon.

What’s new in this release:

By aligning column structures and enumerated values across studies, we are laying the groundwork for more seamless cross-cohort analyses and improved reproducibility. In addition, the inclusion of ingestion date information in each table makes it easier to identify, filter for, and track newly added data over time. You can log in and access the updated datasets through the Target ALS Data Engine at any time.  Users are provided access to Verily Workbench, where they can explore and analyze Target ALS data using Python (JupyterLab) or R (RStudio) across a range of configurable compute environments, including standard virtual machines, Spark-based clusters, and Nvidia-GPU-enabled platforms.  To learn more about working in Verily Workbench, please contact daniel.weatherill@targetals.org to sign up for a virtual workshop co-sponsored by Target ALS and Verily, taking place at 9:00 AM PDT on April 30, 2026 (space is limited).

Postmortem Tissue (PMT) Core

The Target ALS Postmortem Core has now collected 598 cases, making it the most comprehensive ALS tissue repository worldwide that is accessible with no-strings-attached to the global research community. This robust and expanding collection provides researchers with access to high-quality, well-annotated biospecimens essential for advancing understanding of ALS biology. The PMT Core is led by co-directors Dr. Brent Harris, Adjunct Prof. of Neurology, Georgetown University, and Director of Neuropathology, U. Colorado, and Dr. Bob Bowser, CSO Barrow Neurological Institute, and supported by expert neurologists and pathologists from Columbia University, Washington University, UCSD, Georgetown University, BNI, and Edinburgh University.

The Core remains a highly sought-after resource, with 46 tissue requests filled in 2025, setting a new annual record since the core’s inception in 2014.  Samples are used by researchers in academia and industry to directly examine disease-associated changes in cellular architecture, molecular pathways, and gene expression, yielding critical insights into the mechanisms that drive neurodegeneration.

Target ALS has a long history of investing in multi-modal data generation from every case. In 2025, nearly 385 samples had Short-Read RNA sequencing (RNA-seq), 64 samples had Long Read WGS, and 77 new samples were submitted for Short-Read Whole Genome Sequencing (SR-WGS). Other new datasets include pTDP43-stained and digitized images from 200 control, ALS, and FTD cases, supported by the Chan Zuckerberg Initiative, and spatial transcriptomic data from the frontal and motor cortex of 40 control, ALS, or ALS/FTD cases. Visium data were generated by Dr. Phatnani’s lab at the New York Genome Center. All data are available for viewing and mining in the Data Engine. Stay tuned for snRNAseq datasets in 2026.

Longitudinal Biofluid Core: Sustaining Discovery Over Time

In 2025, the Longitudinal Biofluid Core fulfilled 30 biofluid requests, distributing more than 4,000 samples to investigators to enable the development of biomarkers and support a growing body of longitudinal ALS research. Notably, many investigators returned for follow-on sample sets to extend promising initial findings, underscoring the value of repeated measures for biomarker validation. The longitudinal collection continues to expand, driven by improved patient adherence to the study protocol and sustained participant engagement. Rönnholm and team’s recent publication highlights the quality of these samples as used to validate TMT-based quantification of proteins in the CSF. Submit your request online through our Longitudinal Biofluid Core webpage.

Stem Cell Core: Building Robust Human Models for ALS

Through the Target ALS Stem Cell Core Consortium, we are making steady progress toward delivering a new generation of well-characterized, patient-derived iPSC lines to the ALS research community. This collaborative effort is focused on generating reproducible human models carrying key ALS mutations, each paired with CRISPR-corrected isogenic controls to enable precise disease versus control comparisons. Distribution of these lines will be managed by Jackson Laboratories and is expected to be available by June of 2026.   To learn more about the talented scientists behind this effort, see our Stem Cell Core page.

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